Safety, Efficacy and Pharmacokinetic Study of Teduglutide in Infants 4 to 12 Months of Age With Short Bowel Syndrome
Study Details
Study Description
Brief Summary
The purpose of the study is to evaluate the safety, efficacy/pharmacodynamics (PD) and pharmacokinetics (PK) of teduglutide treatment in infants with short bowel syndrome (SBS) dependent on parenteral (PN) support.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Teduglutide Participants will receive 0.05 milligram per kilogram (mg/kg) subcutaneous (SC) injection of teduglutide into abdomen or into either the thigh or arm once daily (QD) in addition to standard medical therapy for 24 weeks. |
Drug: Teduglutide
SC injection of 0.05 mg/kg teduglutide will be administered QD into abdomen or into either the thigh or arm for 24 weeks.
Other: Standard Medical Therapy
Standard medical therapy will be administered for 24 weeks.
Device: Syringe
Teduglutide will be administered using syringe (510k number: K980987).
Device: Needle
Teduglutide will be administered using needle (510k number: K021475).
|
| Other: Standard of Care (SOC) Participants will receive standard medical therapy for 24 weeks. |
Other: Standard Medical Therapy
Standard medical therapy will be administered for 24 weeks.
Device: Syringe
Teduglutide will be administered using syringe (510k number: K980987).
Device: Needle
Teduglutide will be administered using needle (510k number: K021475).
|
Outcome Measures
Primary Outcome Measures
- Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
Secondary Outcome Measures
- Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7) [Baseline: Pre-dose,1, 4 hours post-dose, and 2 hours post-dose at Week 7]
Mean plasma concentration of teduglutide was reported.
- Number of Participants With Treatment-emergent Adverse Events (TEAEs) [From start of study treatment up to end of study (EOS) (up to Week 28)]
An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. TEAEs are defined as AEs that start or deteriorate on or after the date of the first dose of investigational product.
- Change From Baseline in Body Weight Z-score at Week 24 [Baseline, Week 24]
Body weight was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in body weight Z-score at Week 24 was reported.
- Change From Baseline in Length Z-Score at Week 24 [Baseline, Week 24]
Length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in length Z-score at Week 24 was reported.
- Change From Baseline in Head Circumference Z-Score at Week 24 [Baseline, Week 24]
Head circumference was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in head circumference Z-score at Week 24 was reported.
- Change From Baseline in Weight-for-Length Z-Score at Week 24 [Baseline, Week 24]
Weight-for-length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in weight-for-length Z-score at Week 24 was reported.
- Change From Baseline in Average Total Urine Output at Week 24 [Baseline, Week 24]
Average total urine output was recorded over a 48-hour period of nutritional stability at Week 24 was reported. Here, milliliter per kilogram per day is abbreviated as mL/kg/day.
- Change From Baseline in Fecal Output at Week 24 [Baseline, Week 24]
Change from baseline in the fecal output (average number of stools per day) at Week 24 was reported.
- Number of Participants With Positive Specific Antibodies to Teduglutide [Baseline, EOS (up to week 28)]
Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies.
- Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at Week 24 [Week 24]
Number of participants who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at Week 24 were reported.
- Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at End of Study (EOS) [EOS (up to Week 28)]
Number of participants who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at EOS (up to Week 28) were reported.
- Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Percent change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day).
- Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Percent change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Percent change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Percent change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Number of Participants Who Achieved At Least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Number of participants who achieved at least 20% increase from baseline in weight-normalized EN volume at EOT/ET was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
- Number of Participants Who Achieved At Least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) [Baseline, EOT/ET (up to Week 24)]
Number of participants who achieved at least 20% increase from baseline in weight-normalized EN caloric intake at EOT/ET was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Informed consent by the parent or legal guardian.
-
Male or female infant 4 to 12 months corrected gestational age at screening.
-
Weight at least 5 kilogram (kg) and weight-for-length Z-score greater than -2 at screening and baseline.
-
Short bowel syndrome with dependence on parenteral support to provide at least 50% of fluid or caloric needs.
-
Stable PN requirements for at least 1 month prior to screening, defined as a less than or equal to (<=) 10% change in the weight-normalized PN total fluid and caloric intake, despite attempts to wean PN, not withstanding transient instability for events such as sepsis or interruption of central venous access.
-
Parent or legal guardian understands and is willing and able to fully adhere to study requirements as defined in this protocol.
Exclusion Criteria:
-
Previous treatment with teduglutide.
-
Intestinal malabsorption due to a genetic condition, such as cystic fibrosis, microvillus inclusion disease, etc.
-
Severe, known dysmotility syndrome, such as pseudo-obstruction or persistent, severe, active gastroschisis-related dysmotility, that is the primary contributing factor to feeding intolerance and inability to reduce PN support, prior to screening. Dysmotility is defined as severe if it is expected to limit the advancement of enteral feeding.
-
Inability to advance oral or enteral feeding due to lack of access to the gut, such as oral aversion in the absence of a feeding tube.
-
Intestinal obstruction or clinically significant intestinal stenosis.
-
Major gastrointestinal surgical intervention, such as serial transverse enteroplasty or major intestinal resection or anastomosis, within 3 months prior to screening or planned during the study period.
-
Unstable cardiac disease.
-
Renal dysfunction, defined as estimated glomerular filtration rate less than (<) 50 milliliter per minute (mL/min) per 1.73 square meter (m^2).
-
Biliary obstruction, stenosis, or malformation.
-
Clinically significant pancreatic disease.
-
Severe hepatic dysfunction or portal hypertension, defined by at least 2 of the following parameters:
-
International normalized ratio (INR) greater than (>) 1.5 not corrected with PN vitamin K
-
Platelet count <100×10^3/ microliter (mcL) due to portal hypertension
-
Presence of clinically significant gastric or esophageal varices
-
Documented cirrhosis
-
Persistent cholestasis defined as conjugated bilirubin >4 milligram per deciliter (mg/dL) (>68 micromoles per liter [mcmol/L]) over a 2 week period.
-
More than 3 serious complications of intestinal failure (example [e.g.], catheter-associated bloodstream infections, interruption of nutrition due to feeding intolerance, catheter-associated thrombosis, severe fluid or electrolyte disturbances) within 1 month prior to or during screening.
-
A history of cancer or a known cancer predisposition syndrome, such as juvenile polyposis or Beckwith-Wiedemann syndrome, or first degree relative with early onset of gastrointestinal cancer (including hepatobiliary and pancreatic cancers).
-
Concurrent treatment with glucagon-like peptide-1 (GLP-1); glucagon-like peptide-2 (GLP-2); insulin-like growth factor-1 (IGF-1); growth hormone, somatostatin, or analogs of these hormones; or glutamine.
-
Participation in a clinical study using an experimental drug within 3 months or 5.5 half-lives of the experimental drug, whichever is longer.
-
Known or suspected intolerance or hypersensitivity to the investigational product, closely-related compounds, or any of the stated ingredients.
-
Any condition, disease, illness, or circumstance that, in the investigator's opinion, puts the participant at any undue risk, prevents completion of the study, or interferes with analysis of the study results.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Helsingin yliopistollinen keskussairaala | Helsinki | Finland | 00290 | |
| 2 | Groupe Hospitalier Pellegrin - Hôpital des Enfants | Bordeaux | Gironde | France | 33000 |
| 3 | Hopital Jeanne de Flandre - CHRU Lille | Lille | Nord | France | 59037 |
| 4 | Ospedale Pediatrico Bambino Gesù | Roma | Italy | 00165 | |
| 5 | Great Ormond Street Hospital for Children | London | Greater London | United Kingdom | WC1N 3JH |
| 6 | Royal Manchester Children's Hospital | Manchester | Greater Manchester | United Kingdom | M13 9WL |
| 7 | Alder Hey Childrens Hospital | Liverpool | Merseyside | United Kingdom | L12 2AP |
| 8 | Birmingham Children's Hospital | Birmingham | West Midlands | United Kingdom | B4 6NH |
Sponsors and Collaborators
- Shire
Investigators
- Study Director: Study Director, Shire
Study Documents (Full-Text)
More Information
Additional Information:
Publications
None provided.- SHP633-301
- 2017-003606-40
Study Results
Participant Flow
| Recruitment Details | The study was conducted at Finland and United Kingdom between 31 August 2018 (first participant first visit) and 24 September 2020 (last participant last visit). |
|---|---|
| Pre-assignment Detail | A total of 10 participants were enrolled into the study, of which 8 participants completed the study. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Period Title: Overall Study | ||
| STARTED | 5 | 5 |
| COMPLETED | 4 | 4 |
| NOT COMPLETED | 1 | 1 |
Baseline Characteristics
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) | Total |
|---|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. | Total of all reporting groups |
| Overall Participants | 5 | 5 | 10 |
| Age (Months) [Mean (Standard Deviation) ] | |||
| Mean (Standard Deviation) [Months] |
8.5
(3.09)
|
8.3
(2.65)
|
8.4
(2.71)
|
| Sex: Female, Male (Count of Participants) | |||
| Female |
1
20%
|
2
40%
|
3
30%
|
| Male |
4
80%
|
3
60%
|
7
70%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |||
| Hispanic or Latino |
0
0%
|
0
0%
|
0
0%
|
| Not Hispanic or Latino |
5
100%
|
5
100%
|
10
100%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
| Race (NIH/OMB) (Count of Participants) | |||
| American Indian or Alaska Native |
0
0%
|
0
0%
|
0
0%
|
| Asian |
1
20%
|
1
20%
|
2
20%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
| Black or African American |
0
0%
|
0
0%
|
0
0%
|
| White |
4
80%
|
4
80%
|
8
80%
|
| More than one race |
0
0%
|
0
0%
|
0
0%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
Outcome Measures
| Title | Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Intent-to-Treat (ITT) set consisted of all participants randomized in the study. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Diary Data: EOT/ET |
3
60%
|
1
20%
|
| Prescribed Data: EOT/ET |
3
60%
|
3
60%
|
| Title | Plasma Concentration of Teduglutide at Nominal Time Points (Baseline at Pre-dose, and 1 Hour and 4 Hours Post-dose; 2 Hours Post-dose at Week 7) |
|---|---|
| Description | Mean plasma concentration of teduglutide was reported. |
| Time Frame | Baseline: Pre-dose,1, 4 hours post-dose, and 2 hours post-dose at Week 7 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Pharmacokinetic (PK) set consisted of all participants who received at least 1 dose of teduglutide and had at least 1 evaluable and interpretable post-dose PK concentration value. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. Data was not planned to be collected and analyzed for SOC arm. |
| Arm/Group Title | Teduglutide (TED) |
|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. |
| Measure Participants | 4 |
| Baseline: At Pre-dose |
0.00
|
| Baseline: At 1 hour |
16.300
|
| Baseline: At 4 hours |
8.385
|
| Week 7: At 2 hours |
16.950
|
| Title | Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
|---|---|
| Description | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. TEAEs are defined as AEs that start or deteriorate on or after the date of the first dose of investigational product. |
| Time Frame | From start of study treatment up to end of study (EOS) (up to Week 28) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set consisted of all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Count of Participants [Participants] |
5
100%
|
5
100%
|
| Title | Change From Baseline in Body Weight Z-score at Week 24 |
|---|---|
| Description | Body weight was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in body weight Z-score at Week 24 was reported. |
| Time Frame | Baseline, Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set consisted of all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 4 | 3 |
| Mean (Standard Deviation) [Z-score] |
-0.408
(0.377)
|
-0.289
(0.278)
|
| Title | Change From Baseline in Length Z-Score at Week 24 |
|---|---|
| Description | Length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in length Z-score at Week 24 was reported. |
| Time Frame | Baseline, Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set consisted of all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 4 | 2 |
| Mean (Standard Deviation) [Z-Score] |
-0.274
(1.258)
|
-0.422
(0.384)
|
| Title | Change From Baseline in Head Circumference Z-Score at Week 24 |
|---|---|
| Description | Head circumference was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in head circumference Z-score at Week 24 was reported. |
| Time Frame | Baseline, Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set consisted of all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 4 | 2 |
| Mean (Standard Deviation) [Z-Score] |
-0.544
(0.446)
|
-0.167
(0.690)
|
| Title | Change From Baseline in Weight-for-Length Z-Score at Week 24 |
|---|---|
| Description | Weight-for-length was measured using Z-score. Z-score was calculated as (observed value - median value of the reference population)/standard deviation value of reference population. A negative Z-score indicates values lower than the mean while a positive Z-score indicates values higher than the mean. Change from baseline in weight-for-length Z-score at Week 24 was reported. |
| Time Frame | Baseline, Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set consisted of all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 4 | 3 |
| Mean (Standard Deviation) [Z-Score] |
-0.447
(1.042)
|
-0.058
(0.992)
|
| Title | Change From Baseline in Average Total Urine Output at Week 24 |
|---|---|
| Description | Average total urine output was recorded over a 48-hour period of nutritional stability at Week 24 was reported. Here, milliliter per kilogram per day is abbreviated as mL/kg/day. |
| Time Frame | Baseline, Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set consisted of all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 3 | 3 |
| Mean (Standard Deviation) [mL/kg/day] |
-0.61
(14.548)
|
10.25
(17.383)
|
| Title | Change From Baseline in Fecal Output at Week 24 |
|---|---|
| Description | Change from baseline in the fecal output (average number of stools per day) at Week 24 was reported. |
| Time Frame | Baseline, Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set consisted of all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and had undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 3 | 3 |
| Mean (Standard Deviation) [Average number of stools per day] |
-3.33
(3.547)
|
1.67
(1.756)
|
| Title | Number of Participants With Positive Specific Antibodies to Teduglutide |
|---|---|
| Description | Number of participants with positive specific antibodies to teduglutide were used to summarize the presence of antibodies. |
| Time Frame | Baseline, EOS (up to week 28) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Safety set: all participants who met the following criteria: Teduglutide treatment arm: participants who received at least 1 dose of teduglutide and undergone at least 1 post baseline safety assessment; Standard of care treatment arm: participants who had undergone at least 1 post baseline safety assessment. Here, number of participants analyzed signifies participants who were evaluable for this outcome measure. Data for this outcome was not planned to be collected and analyzed for SOC group. |
| Arm/Group Title | Teduglutide (TED) |
|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. |
| Measure Participants | 3 |
| Count of Participants [Participants] |
0
0%
|
| Title | Number of Participants Who Achieved At Least 20 Percent (%) Reduction From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Number of participants who achieved at least 20% reduction from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Diary Data: EOT/ET |
3
60%
|
1
20%
|
| Prescribed Data: EOT/ET |
3
60%
|
3
60%
|
| Title | Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at Week 24 |
|---|---|
| Description | Number of participants who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at Week 24 were reported. |
| Time Frame | Week 24 |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 4 | 4 |
| Count of Participants [Participants] |
0
0%
|
0
0%
|
| Title | Number of Participants Who Achieved 100 Percent (%) Reduction in Complete Weaning Off (Enteral Autonomy) Parenteral Support (PS) Volume at End of Study (EOS) |
|---|---|
| Description | Number of participants who achieved 100% reduction in complete weaning off (enteral autonomy) PS volume at EOS (up to Week 28) were reported. |
| Time Frame | EOS (up to Week 28) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 4 | 5 |
| Count of Participants [Participants] |
0
0%
|
0
0%
|
| Title | Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number analyzed signifies participants who were evaluable for this outcome at specific time points. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Diary Data: EOT/ET |
-21.54
(28.909)
|
-9.51
(7.497)
|
| Prescribed Data: EOT/ET |
-22.90
(26.940)
|
-14.90
(12.323)
|
| Title | Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Volume at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Percent change from baseline in weight-normalized PS volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number analyzed signifies participants who were evaluable for this outcome at specific time points. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Diary Data: EOT/ET |
-24.77
(34.723)
|
-16.75
(16.392)
|
| Prescribed Data: EOT/ET |
-27.28
(33.518)
|
-22.39
(17.198)
|
| Title | Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. Here, kilo-calories per kilogram per day was abbreviated as (kcal/kg/day). |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number analyzed signifies participants who were evaluable for this outcome at specific time points. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Diary Data: EOT/ET |
-16.14
(17.547)
|
-6.10
(10.386)
|
| Prescribed Data: EOT/ET |
-15.31
(17.839)
|
-20.40
(21.024)
|
| Title | Percent Change From Baseline in Weight-normalized Parenteral Support (PS) Caloric Intake at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Percent change from baseline in weight-normalized PS caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number analyzed signifies participants who were evaluable for this outcome at specific time points. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Diary Data: EOT/ET |
-27.00
(29.473)
|
-13.68
(21.873)
|
| Prescribed Data: EOT/ET |
-27.81
(30.777)
|
-38.86
(39.893)
|
| Title | Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 4 | 5 |
| Diary Data: EOT/ET |
16.14
(18.683)
|
-15.25
(31.496)
|
| Prescribed Data: EOT/ET |
-1.28
(2.563)
|
2.27
(22.232)
|
| Title | Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Percent change from baseline in weight-normalized EN volume at EOT/ET (up to Week 24) was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 2 | 4 |
| Diary Data: EOT/ET |
273.20
(246.784)
|
-44.25
(78.847)
|
| Prescribed Data: EOT/ET |
-16.40
(NA)
|
14.80
(69.834)
|
| Title | Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 4 | 5 |
| Diary Data: EOT/ET |
9.08
(10.662)
|
-9.38
(21.402)
|
| Prescribed Data: EOT/ET |
-1.15
(2.306)
|
3.11
(16.285)
|
| Title | Percent Change From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Percent change from baseline in weight-normalized EN caloric intake at EOT/ET (up to Week 24) were reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. Here, the number of participants analyzed signifies participants who were evaluable for this outcome measure and number analyzed signifies participants who were evaluable at specific time point. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 2 | 4 |
| Diary Data: EOT/ET |
207.06
(153.159)
|
-44.25
(78.847)
|
| Prescribed Data: EOT/ET |
-16.40
(NA)
|
24.18
(78.115)
|
| Title | Number of Participants Who Achieved At Least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Volume at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Number of participants who achieved at least 20% increase from baseline in weight-normalized EN volume at EOT/ET was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Diary Data: EOT/ET |
2
40%
|
0
0%
|
| Prescribed Data: EOT/ET |
0
0%
|
2
40%
|
| Title | Number of Participants Who Achieved At Least 20 Percent (%) Increase From Baseline in Weight-normalized Enteral Nutrition (EN) Caloric Intake at End of Treatment/Early Termination (EOT/ET) |
|---|---|
| Description | Number of participants who achieved at least 20% increase from baseline in weight-normalized EN caloric intake at EOT/ET was reported. EOT/ET was defined as the last available visit after the date of first dose (or randomization in standard of care treatment group) during the 24-week treatment period. |
| Time Frame | Baseline, EOT/ET (up to Week 24) |
Outcome Measure Data
| Analysis Population Description |
|---|
| ITT set consisted of all participants randomized in the study. |
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) |
|---|---|---|
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. |
| Measure Participants | 5 | 5 |
| Diary Data: EOT/ET |
2
40%
|
0
0%
|
| Prescribed Data: EOT/ET |
0
0%
|
2
40%
|
Adverse Events
| Time Frame | From start of study treatment up to end of study (EOS) (up to Week 28) | |||
|---|---|---|---|---|
| Adverse Event Reporting Description | ||||
| Arm/Group Title | Teduglutide (TED) | Standard of Care (SOC) | ||
| Arm/Group Description | Participants (infants) 4 to 12 months of corrected gestational aged received 0.05 milligram per kilogram (mg/kg) of teduglutide subcutaneous (SC) injection QD in addition to standard medical therapy for 24 weeks. | Participants (infants) 4 to 12 months of corrected gestational aged received standard medical therapy for 24 weeks. | ||
All Cause Mortality |
||||
| Teduglutide (TED) | Standard of Care (SOC) | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/5 (0%) | 0/5 (0%) | ||
Serious Adverse Events |
||||
| Teduglutide (TED) | Standard of Care (SOC) | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 4/5 (80%) | 3/5 (60%) | ||
| General disorders | ||||
| Pyrexia | 1/5 (20%) | 2 | 0/5 (0%) | 0 |
| Immune system disorders | ||||
| Immunisation reaction | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Infections and infestations | ||||
| Device related infection | 0/5 (0%) | 0 | 2/5 (40%) | 2 |
| Investigations | ||||
| Transaminases increased | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Metabolism and nutrition disorders | ||||
| Metabolic acidosis | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Nervous system disorders | ||||
| Ataxia | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Product Issues | ||||
| Device breakage | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Device leakage | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Device occlusion | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
Other (Not Including Serious) Adverse Events |
||||
| Teduglutide (TED) | Standard of Care (SOC) | |||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 5/5 (100%) | 5/5 (100%) | ||
| Blood and lymphatic system disorders | ||||
| Anaemia | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Iron deficiency anaemia | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Gastrointestinal disorders | ||||
| Abdominal discomfort | 1/5 (20%) | 2 | 0/5 (0%) | 0 |
| Abdominal distension | 1/5 (20%) | 3 | 0/5 (0%) | 0 |
| Abnormal faeces | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Constipation | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Diarrhoea | 2/5 (40%) | 3 | 0/5 (0%) | 0 |
| Faeces discoloured | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Flatulence | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Frequent bowel movements | 2/5 (40%) | 2 | 0/5 (0%) | 0 |
| Gastrointestinal sounds abnormal | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Mucous stools | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Retching | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Teething | 0/5 (0%) | 0 | 2/5 (40%) | 3 |
| Vomiting | 3/5 (60%) | 8 | 1/5 (20%) | 2 |
| General disorders | ||||
| Pyrexia | 1/5 (20%) | 1 | 1/5 (20%) | 1 |
| Secretion discharge | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Infections and infestations | ||||
| Gastroenteritis norovirus | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Hand-foot-and-mouth disease | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Medical device site infection | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Nasopharyngitis | 2/5 (40%) | 2 | 0/5 (0%) | 0 |
| Respiratory tract infection viral | 1/5 (20%) | 2 | 0/5 (0%) | 0 |
| Upper respiratory tract infection | 1/5 (20%) | 1 | 2/5 (40%) | 2 |
| Viral infection | 1/5 (20%) | 1 | 1/5 (20%) | 1 |
| Injury, poisoning and procedural complications | ||||
| Contusion | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Lip injury | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Skin abrasion | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Investigations | ||||
| Alanine aminotransferase increased | 1/5 (20%) | 2 | 0/5 (0%) | 0 |
| Blood iron decreased | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Faecal volume increased | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Respiratory rate increased | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Serum ferritin decreased | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Transaminases increased | 0/5 (0%) | 0 | 1/5 (20%) | 2 |
| Metabolism and nutrition disorders | ||||
| Decreased appetite | 1/5 (20%) | 2 | 0/5 (0%) | 0 |
| Hypoglycaemia | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Hypophagia | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Product Issues | ||||
| Device breakage | 1/5 (20%) | 1 | 1/5 (20%) | 1 |
| Device occlusion | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Psychiatric disorders | ||||
| Irritability | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Sleep disorder | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Respiratory, thoracic and mediastinal disorders | ||||
| Cough | 1/5 (20%) | 1 | 1/5 (20%) | 1 |
| Rhinorrhoea | 1/5 (20%) | 1 | 1/5 (20%) | 1 |
| Skin and subcutaneous tissue disorders | ||||
| Dermatitis diaper | 1/5 (20%) | 2 | 0/5 (0%) | 0 |
| Eczema | 1/5 (20%) | 1 | 0/5 (0%) | 0 |
| Rash papular | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
| Vascular disorders | ||||
| Hypertension | 0/5 (0%) | 0 | 1/5 (20%) | 1 |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
Results Point of Contact
| Name/Title | Study Director |
|---|---|
| Organization | Shire |
| Phone | +1 866 842 5335 |
| ClinicalTransparency@shire.com |
- SHP633-301
- 2017-003606-40