THRIVE: Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Sponsor
Horizon Therapeutics, LLC (Industry)
Overall Status
Terminated
CT.gov ID
NCT01948427
Collaborator
(none)
203
1
77
2.6
Study Details
Study Description
Brief Summary
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years. As an observational study, enrolled patients will not be required to make any additional office visits or take any medicine outside of normal care.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
|
Detailed Description
UCDs disproportionately affect children and females: depending on the severity of the defect, a UCD can manifest shortly after birth or later in life. This study will track long-term outcomes in UCD patients and effects of ammonia-scavenging agents on neuropsychological functions of UCD patients.
This is a non-interventional, multi-center registry to be conducted in patients with UCDs. Investigators will prescribe treatments based on usual clinical practice, and there will be no restrictions on the use of commercially available medications. As an observational study, this study will not change the patient/ healthcare provider relationship, nor influence the healthcare provider's drug prescription or the therapeutic management of the patient.
Patients with UCDs will be recruited and invited to attend a Baseline visit. After eligible patients are enrolled, retrospective and baseline data will be collected. Patients will be followed for up to 10 years, during which time they will be assessed by their healthcare provider. Patients and healthcare provider will be asked to report episodes of hyperammonemic crisis, available ammonia values, and other information.
Study Design
Study Type:
Observational [Patient Registry]
Actual Enrollment
:
203 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Long-Term Registry of Patients With Urea Cycle Disorders (UCDs)
Actual Study Start Date
:
Sep 25, 2013
Actual Primary Completion Date
:
Feb 24, 2020
Actual Study Completion Date
:
Feb 24, 2020
Outcome Measures
Primary Outcome Measures
- Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication [12 months prior to enrollment (retrospective), Baseline, Day 7 to 30, Month 6, Month 12, Month 18, Month 24, Month 30, Month 36, Month 42, Month 48, Month 54, Month 60, Month 66, Month 72]
Retrospective is defined as the 12 months preceding enrollment.
- Median Blood Ammonia Levels Over Time, by Last-Known Ammonia-Scavenging Medication [12 months prior to enrollment (retrospective), Baseline, Day 7 to 30, Month 6, Month 12, Month 18, Month 24, Month 30, Month 36, Month 42, Month 48, Month 54, Month 60, Month 66, Month 72]
Retrospective is defined as the 12 months preceding enrollment.
- Percentage of Participants With Hyperammonemic Crisis (HAC) by Baseline Ammonia-Scavenging Medication, Retrospective Values [12 months prior to enrollment (retrospective)]
Percentage of participants experiencing HAC (reported for the 12 months preceding enrollment).
- Percentage of Participants With Hyperammonemic Crisis (HAC), Post-Baseline by Last Known Ammonia-Scavenging Medication [From enrollment through the end of study (mean overall duration on study was 1187.7 days).]
Percentage of participants experiencing HAC (post-Baseline).
- Number of Participants With Serious Adverse Events (SAEs) [From enrollment through the end of study (mean overall duration on study was 1187.7 days).]
An SAE is an adverse event that: is fatal or life-threatening; results in persistent or significant disability or incapacity. Disability is defined as a substantial disruption of a person's ability to conduct normal life functions; requires inpatient hospitalization or prolongation of an existing hospitalization; is a congenital anomaly/birth defect; any other important medical event that may jeopardize the patient and may require medical or surgical intervention to prevent one of the outcomes listed above.
Eligibility Criteria
Criteria
Ages Eligible for Study:
N/A
and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
-
Confirmed or suspected diagnosis of UCD
-
Signed informed consent/Health Insurance Portability and Accountability Act (HIPAA) Authorization and medical records release
Exclusion Criteria:
- Any other reason that, in the Investigator's opinion, makes the patient unsuitable to participate in this study.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | United BioSource Corporation | Blue Bell | Pennsylvania | United States | 19422 |
Sponsors and Collaborators
- Horizon Therapeutics, LLC
Investigators
None specified.Study Documents (Full-Text)
More Information
Additional Information:
Publications
None provided.Responsible Party:
Horizon Therapeutics, LLC
ClinicalTrials.gov Identifier:
NCT01948427
Other Study ID Numbers:
- HPN-100-014
First Posted:
Sep 23, 2013
Last Update Posted:
Feb 18, 2021
Last Verified:
Feb 1, 2021
Keywords provided by Horizon Therapeutics, LLC
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|
| Arm/Group Description | Participants with a confirmed or suspected diagnosis of urea cycle disorder (UCD) receiving sodium phenylbutyrate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving Ravicti at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving sodium benzoate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving carglumic acid at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving other (not specified) ammonia-scavenging medication at Baseline. |
| Period Title: Overall Study | |||||
| STARTED | 49 | 89 | 4 | 1 | 60 |
| COMPLETED | 42 | 72 | 4 | 1 | 46 |
| NOT COMPLETED | 7 | 17 | 0 | 0 | 14 |
Baseline Characteristics
| Arm/Group Title | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other | Total |
|---|---|---|---|---|---|---|
| Arm/Group Description | Participants with a confirmed or suspected diagnosis of UCD receiving sodium phenylbutyrate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving Ravicti at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving sodium benzoate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving carglumic acid at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving other (not specified) ammonia-scavenging medication at Baseline. | Total of all reporting groups |
| Overall Participants | 49 | 89 | 4 | 1 | 60 | 203 |
| Age (years) [Mean (Standard Deviation) ] | ||||||
| Mean (Standard Deviation) [years] |
10.9
(13.5)
|
16.7
(15.1)
|
9.3
(8.7)
|
2.0
(NA)
|
14.7
(16.9)
|
14.5
(15.3)
|
| Sex: Female, Male (Count of Participants) | ||||||
| Female |
33
67.3%
|
54
60.7%
|
1
25%
|
0
0%
|
33
55%
|
121
59.6%
|
| Male |
16
32.7%
|
35
39.3%
|
3
75%
|
1
100%
|
27
45%
|
82
40.4%
|
| Ethnicity (NIH/OMB) (Count of Participants) | ||||||
| Hispanic or Latino |
13
26.5%
|
16
18%
|
3
75%
|
0
0%
|
7
11.7%
|
39
19.2%
|
| Not Hispanic or Latino |
36
73.5%
|
73
82%
|
1
25%
|
1
100%
|
53
88.3%
|
164
80.8%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Race (Count of Participants) | ||||||
| White |
30
61.2%
|
63
70.8%
|
4
100%
|
1
100%
|
48
80%
|
146
71.9%
|
| Black or African-American |
9
18.4%
|
9
10.1%
|
0
0%
|
0
0%
|
4
6.7%
|
22
10.8%
|
| Asian |
1
2%
|
2
2.2%
|
0
0%
|
0
0%
|
3
5%
|
6
3%
|
| Native Hawaiian or Other Pacific Islander |
1
2%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
1
0.5%
|
| Other, Not Specified |
8
16.3%
|
15
16.9%
|
0
0%
|
0
0%
|
5
8.3%
|
28
13.8%
|
Outcome Measures
| Title | Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication |
|---|---|
| Description | Retrospective is defined as the 12 months preceding enrollment. |
| Time Frame | 12 months prior to enrollment (retrospective), Baseline, Day 7 to 30, Month 6, Month 12, Month 18, Month 24, Month 30, Month 36, Month 42, Month 48, Month 54, Month 60, Month 66, Month 72 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants with a Baseline visit and an assessment at given time point. Participants are grouped by last-known ammonia-scavenging medication at given time point (participants may have been on more than 1 therapy during the study). |
| Arm/Group Title | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|
| Arm/Group Description | Participants with a confirmed or suspected diagnosis of UCD receiving sodium phenylbutyrate. | Participants with a confirmed or suspected diagnosis of UCD receiving Ravicti. | Participants with a confirmed or suspected diagnosis of UCD receiving sodium benzoate. | Participants with a confirmed or suspected diagnosis of UCD receiving carglumic acid. | Participants with a confirmed or suspected diagnosis of UCD receiving other (not specified) ammonia-scavenging medication. |
| Measure Participants | 40 | 67 | 6 | 2 | 5 |
| Retrospective |
86.5
(87.13)
|
43.8
(27.59)
|
23.0
(NA)
|
13.0
(NA)
|
963.7
(721.39)
|
| Baseline |
64.5
(52.32)
|
49.1
(43.58)
|
48.0
(29.73)
|
21.0
(NA)
|
|
| Day 7 to 30 |
64.0
(20.53)
|
79.6
(72.23)
|
55.0
(NA)
|
143.5
(98.29)
|
|
| Month 6 |
48.7
(53.77)
|
45.0
(30.26)
|
190.3
(257.80)
|
48.0
(25.46)
|
129.4
(110.54)
|
| Month 12 |
42.4
(20.87)
|
39.4
(24.43)
|
51.0
(16.97)
|
20.0
(NA)
|
79.0
(57.98)
|
| Month 18 |
54.5
(53.89)
|
45.9
(28.26)
|
29.0
(11.79)
|
13.0
(NA)
|
106.0
(110.31)
|
| Month 24 |
53.9
(38.49)
|
52.8
(78.14)
|
43.3
(31.26)
|
25.0
(NA)
|
|
| Month 30 |
42.0
(18.18)
|
47.5
(33.27)
|
79.8
(85.85)
|
114.5
(127.99)
|
|
| Month 36 |
36.2
(18.90)
|
45.9
(34.95)
|
72.0
(NA)
|
51.5
(34.65)
|
|
| Month 42 |
40.2
(14.39)
|
43.3
(27.30)
|
41.5
(0.71)
|
||
| Month 48 |
61.4
(57.78)
|
54.2
(42.95)
|
36.0
(NA)
|
22.0
(NA)
|
|
| Month 54 |
55.6
(8.59)
|
45.0
(29.44)
|
66.0
(0.00)
|
292.0
(NA)
|
|
| Month 60 |
57.3
(22.37)
|
45.2
(32.78)
|
8.9
(NA)
|
||
| Month 66 |
62.0
(NA)
|
31.0
(11.05)
|
|||
| Month 72 |
55.0
(NA)
|
45.5
(43.13)
|
| Title | Median Blood Ammonia Levels Over Time, by Last-Known Ammonia-Scavenging Medication |
|---|---|
| Description | Retrospective is defined as the 12 months preceding enrollment. |
| Time Frame | 12 months prior to enrollment (retrospective), Baseline, Day 7 to 30, Month 6, Month 12, Month 18, Month 24, Month 30, Month 36, Month 42, Month 48, Month 54, Month 60, Month 66, Month 72 |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants with a Baseline visit and an assessment at given time point. Participants are grouped by last-known ammonia-scavenging medication at given time point (participants may have been on more than 1 therapy during the study). |
| Arm/Group Title | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|
| Arm/Group Description | Participants with a confirmed or suspected diagnosis of UCD receiving sodium phenylbutyrate. | Participants with a confirmed or suspected diagnosis of UCD receiving Ravicti. | Participants with a confirmed or suspected diagnosis of UCD receiving sodium benzoate. | Participants with a confirmed or suspected diagnosis of UCD receiving carglumic acid. | Participants with a confirmed or suspected diagnosis of UCD receiving other (not specified) ammonia-scavenging medication. |
| Measure Participants | 40 | 67 | 6 | 2 | 5 |
| Retrospective |
46.0
|
38.0
|
23.0
|
13.0
|
1360.0
|
| Baseline |
50.0
|
37.0
|
46.5
|
21.0
|
|
| Day 7 to 30 |
60.0
|
61.5
|
55.0
|
143.5
|
|
| Month 6 |
33.0
|
34.0
|
44.0
|
48.0
|
96.0
|
| Month 12 |
42.0
|
34.5
|
51.0
|
20.0
|
79.0
|
| Month 18 |
36.0
|
45.0
|
26.0
|
13.0
|
51.0
|
| Month 24 |
47.0
|
34.5
|
48.0
|
25.0
|
|
| Month 30 |
43.0
|
35.0
|
52.0
|
114.5
|
|
| Month 36 |
38.0
|
34.0
|
72.0
|
51.5
|
|
| Month 42 |
40.5
|
38.0
|
41.5
|
||
| Month 48 |
39.0
|
40.0
|
36.0
|
22.0
|
|
| Month 54 |
57.0
|
35.8
|
66.0
|
292.0
|
|
| Month 60 |
62.0
|
29.0
|
8.9
|
||
| Month 66 |
62.0
|
27.5
|
|||
| Month 72 |
55.0
|
45.5
|
| Title | Percentage of Participants With Hyperammonemic Crisis (HAC) by Baseline Ammonia-Scavenging Medication, Retrospective Values |
|---|---|
| Description | Percentage of participants experiencing HAC (reported for the 12 months preceding enrollment). |
| Time Frame | 12 months prior to enrollment (retrospective) |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants with a baseline visit. |
| Arm/Group Title | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|
| Arm/Group Description | Participants with a confirmed or suspected diagnosis of UCD receiving sodium phenylbutyrate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving Ravicti at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving sodium benzoate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving carglumic acid at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving other (not specified) ammonia-scavenging medication at Baseline. |
| Measure Participants | 49 | 89 | 4 | 1 | 60 |
| Number [percentage of participants] |
44.9
91.6%
|
15.7
17.6%
|
25.0
625%
|
100.0
10000%
|
8.3
13.8%
|
| Title | Percentage of Participants With Hyperammonemic Crisis (HAC), Post-Baseline by Last Known Ammonia-Scavenging Medication |
|---|---|
| Description | Percentage of participants experiencing HAC (post-Baseline). |
| Time Frame | From enrollment through the end of study (mean overall duration on study was 1187.7 days). |
Outcome Measure Data
| Analysis Population Description |
|---|
| Participants with a baseline visit and at least 1 measurement of this assessment while on therapy. Participants are grouped by last-known ammonia-scavenging medication; participants may have been on more than 1 therapy during the study and the number of participants analyzed may be greater than the overall number of participants. |
| Arm/Group Title | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|
| Arm/Group Description | Participants with a confirmed or suspected diagnosis of UCD receiving sodium phenylbutyrate. | Participants with a confirmed or suspected diagnosis of UCD receiving Ravicti. | Participants with a confirmed or suspected diagnosis of UCD receiving sodium benzoate. | Participants with a confirmed or suspected diagnosis of UCD receiving carglumic acid. | Participants with a confirmed or suspected diagnosis of UCD receiving other (not specified) ammonia-scavenging medication. |
| Measure Participants | 61 | 118 | 10 | 2 | 22 |
| Number [percentage of participants] |
42.6
86.9%
|
37.3
41.9%
|
40.0
1000%
|
0
0%
|
72.7
121.2%
|
| Title | Number of Participants With Serious Adverse Events (SAEs) |
|---|---|
| Description | An SAE is an adverse event that: is fatal or life-threatening; results in persistent or significant disability or incapacity. Disability is defined as a substantial disruption of a person's ability to conduct normal life functions; requires inpatient hospitalization or prolongation of an existing hospitalization; is a congenital anomaly/birth defect; any other important medical event that may jeopardize the patient and may require medical or surgical intervention to prevent one of the outcomes listed above. |
| Time Frame | From enrollment through the end of study (mean overall duration on study was 1187.7 days). |
Outcome Measure Data
| Analysis Population Description |
|---|
| All participants who attended a Baseline visit. Due to data collection complexities in this observational study (e.g., switching of medications, incomplete data on medications, and missing SAE dates) AEs could not be reliably summarized according to a participant's treatment at the time of SAE, and was therefore summarized by treatment at baseline. |
| Arm/Group Title | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|
| Arm/Group Description | Participants with a confirmed or suspected diagnosis of UCD receiving sodium phenylbutyrate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving Ravicti at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving sodium benzoate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving carglumic acid at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving other (not specified) ammonia-scavenging medication at Baseline. |
| Measure Participants | 49 | 89 | 4 | 1 | 60 |
| Count of Participants [Participants] |
30
61.2%
|
40
44.9%
|
2
50%
|
0
0%
|
16
26.7%
|
Adverse Events
| Time Frame | From enrollment through the end of study (mean overall duration on study was 1187.7 days). | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | Per protocol, non-serious adverse events were not collected in this observational registry study. Due to data collection complexities in this observational study (e.g., switching of medications, incomplete data on medications, and missing SAE dates) AEs could not be reliably summarized according to a participant's treatment at the time of SAE, and was therefore summarized by treatment at baseline. | |||||||||
| Arm/Group Title | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other | |||||
| Arm/Group Description | Participants with a confirmed or suspected diagnosis of UCD receiving sodium phenylbutyrate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving Ravicti at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving sodium benzoate at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving carglumic acid at Baseline. | Participants with a confirmed or suspected diagnosis of UCD receiving other (not specified) ammonia-scavenging medication at Baseline. | |||||
All Cause Mortality |
||||||||||
| Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 3/49 (6.1%) | 2/89 (2.2%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
Serious Adverse Events |
||||||||||
| Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 30/49 (61.2%) | 40/89 (44.9%) | 2/4 (50%) | 0/1 (0%) | 16/60 (26.7%) | |||||
| Blood and lymphatic system disorders | ||||||||||
| Autoimmune Haemolytic Anaemia | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Immune Thrombocytopenic Purpura | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Cardiac disorders | ||||||||||
| Acute Myocardial Infarction | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Cardiac Arrest | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Congenital, familial and genetic disorders | ||||||||||
| Arginase Deficiency | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Eye disorders | ||||||||||
| Eye Swelling | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Gastrointestinal disorders | ||||||||||
| Abdominal Pain | 0/49 (0%) | 3/89 (3.4%) | 1/4 (25%) | 0/1 (0%) | 0/60 (0%) | |||||
| Constipation | 1/49 (2%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Diarrhoea | 3/49 (6.1%) | 1/89 (1.1%) | 1/4 (25%) | 0/1 (0%) | 0/60 (0%) | |||||
| Diarrhoea Haemorrhagic | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Intestinal Perforation | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Nausea | 1/49 (2%) | 0/89 (0%) | 1/4 (25%) | 0/1 (0%) | 0/60 (0%) | |||||
| Pancreatitis | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Small Intestinal Obstruction | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Stomatitis | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Vomiting | 7/49 (14.3%) | 5/89 (5.6%) | 1/4 (25%) | 0/1 (0%) | 2/60 (3.3%) | |||||
| General disorders | ||||||||||
| Death | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Medical Device Site Pain | 0/49 (0%) | 0/89 (0%) | 1/4 (25%) | 0/1 (0%) | 0/60 (0%) | |||||
| Pneumatosis | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Pyrexia | 3/49 (6.1%) | 2/89 (2.2%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Hepatobiliary disorders | ||||||||||
| Bile Duct Stenosis | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Hepatic Artery Thrombosis | 2/49 (4.1%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Hepatic Function Abnormal | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Immune system disorders | ||||||||||
| Anaphylactic Reaction | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Infections and infestations | ||||||||||
| Appendicitis Perforated | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Bronchiolitis | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Cellulitis | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Clostridium Difficile Infection | 2/49 (4.1%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Corona Virus Infection | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Croup Infectious | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Ear Infection | 0/49 (0%) | 0/89 (0%) | 1/4 (25%) | 0/1 (0%) | 0/60 (0%) | |||||
| Enterobacter Sepsis | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Escherichia Infection | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Gastroenteritis | 1/49 (2%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Gastroenteritis Viral | 2/49 (4.1%) | 4/89 (4.5%) | 0/4 (0%) | 0/1 (0%) | 3/60 (5%) | |||||
| Gastrointestinal Viral Infection | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Human Herpesvirus 6 Infection | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Influenza | 2/49 (4.1%) | 2/89 (2.2%) | 0/4 (0%) | 0/1 (0%) | 2/60 (3.3%) | |||||
| Medical Device Site Cellulitis | 0/49 (0%) | 0/89 (0%) | 1/4 (25%) | 0/1 (0%) | 0/60 (0%) | |||||
| Metapneumovirus Infection | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Otitis Media Acute | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Pneumonia Respiratory Syncytial Viral | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Pyelonephritis | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Respiratory Syncytial Virus Bronchiolitis | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Salmonellosis | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Subcutaneous Abscess | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Upper Respiratory Tract Infection | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 2/60 (3.3%) | |||||
| Urinary Tract Infection | 1/49 (2%) | 0/89 (0%) | 1/4 (25%) | 0/1 (0%) | 0/60 (0%) | |||||
| Viral Infection | 0/49 (0%) | 2/89 (2.2%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Viral Upper Respiratory Tract Infection | 2/49 (4.1%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Injury, poisoning and procedural complications | ||||||||||
| Accidental Overdose | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Femur Fracture | 1/49 (2%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Procedural Vomiting | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Road Traffic Accident | 1/49 (2%) | 2/89 (2.2%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Investigations | ||||||||||
| Ammonia Increased | 2/49 (4.1%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Norovirus Test Positive | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Streptococcus Test Positive | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Transaminases Increased | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Metabolism and nutrition disorders | ||||||||||
| Dehydration | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Failure To Thrive | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Feeding Intolerance | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Fluid Overload | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Hyperammonaemia | 19/49 (38.8%) | 26/89 (29.2%) | 0/4 (0%) | 0/1 (0%) | 2/60 (3.3%) | |||||
| Hyperammonaemic Crisis | 8/49 (16.3%) | 3/89 (3.4%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Hypernatraemia | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Hypoglycaemia | 1/49 (2%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Hypokalaemia | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Hypophagia | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Metabolic Acidosis | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Metabolic Disorder | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Musculoskeletal and connective tissue disorders | ||||||||||
| Costochondritis | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Joint Swelling | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Muscular Weakness | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Pain In Extremity | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Scoliosis | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Nervous system disorders | ||||||||||
| Altered State Of Consciousness | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Cerebral Venous Thrombosis | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Chorea | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Dizziness | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Encephalopathy | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Epileptic Encephalopathy | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Febrile Convulsion | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Guillain-Barre Syndrome | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Headache | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Hyperammonaemic Encephalopathy | 1/49 (2%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Incoherent | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Ischaemic Stroke | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Metabolic Encephalopathy | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Motor Developmental Delay | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Seizure | 0/49 (0%) | 2/89 (2.2%) | 1/4 (25%) | 0/1 (0%) | 0/60 (0%) | |||||
| Pregnancy, puerperium and perinatal conditions | ||||||||||
| Delivery | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Device Occlusion | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Product Issues | ||||||||||
| Device Dislocation | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Psychiatric disorders | ||||||||||
| Aggression | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Confusional State | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Conversion Disorder | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Delirium | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Mental Status Changes | 2/49 (4.1%) | 3/89 (3.4%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Renal and urinary disorders | ||||||||||
| Acute Kidney Injury | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Renal Failure | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Reproductive system and breast disorders | ||||||||||
| Adnexal Torsion | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Respiratory, thoracic and mediastinal disorders | ||||||||||
| Acute Respiratory Failure | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Dyspnoea | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Pleural Effusion | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Respiratory Disorder | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Respiratory Distress | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Respiratory Failure | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Sleep Apnoea Syndrome | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
| Vocal Cord Disorder | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Social circumstances | ||||||||||
| Treatment Noncompliance | 1/49 (2%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Surgical and medical procedures | ||||||||||
| Spinal Fusion Surgery | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Vascular disorders | ||||||||||
| Deep Vein Thrombosis | 0/49 (0%) | 1/89 (1.1%) | 0/4 (0%) | 0/1 (0%) | 0/60 (0%) | |||||
| Thrombophlebitis | 0/49 (0%) | 0/89 (0%) | 0/4 (0%) | 0/1 (0%) | 1/60 (1.7%) | |||||
Other (Not Including Serious) Adverse Events |
||||||||||
| Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other | ||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/0 (NaN) | 0/0 (NaN) | 0/0 (NaN) | 0/0 (NaN) | 0/0 (NaN) | |||||
Limitations/Caveats
Due to data collection complexities in this observational study (e.g., switching of medications, incomplete data on medications, and missing SAE dates) AEs could not be reliably summarized according to a participant's treatment at the time of SAE, and was therefore summarized by treatment at baseline.
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
Horizon requests that any investigator/institution that plans on presenting/publishing results provide written notification of their request 60 days prior to their presentation/publication. Horizon requests that no presentation/publication will be instituted until 12 months after a study is completed, or after the first presentation/publication whichever occurs first. A delay may be proposed of a presentation/publication if Horizon needs to secure patent or proprietary protection.
Results Point of Contact
| Name/Title | Horizon Therapeutics, LLC |
|---|---|
| Organization | Horizon Therapeutics, LLC |
| Phone | 866-479-6742 |
| clinicaltrials@horizontherapeutics.com |
Responsible Party:
Horizon Therapeutics, LLC
ClinicalTrials.gov Identifier:
NCT01948427
Other Study ID Numbers:
- HPN-100-014
First Posted:
Sep 23, 2013
Last Update Posted:
Feb 18, 2021
Last Verified:
Feb 1, 2021