Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
Study Details
Study Description
Brief Summary
The purpose of the study is to establish the pharmacokinetic profile of omigapil in paediatric and adolescent patients with CMD and to evaluate the safety and tolerability of omigapil.
Funding source - FDA OOPD
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 1 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Omigapil Omigapil treatment oral administration once per day after breakfast Cohort 1 Cohort 1 0.02 mg/kg/day Cohort 2 0.08 mg/kg/day Cohort 3a 0.04 mg/kg/day Cohort 3b 0.06 mg/kg/day |
Drug: Omigapil
Cohort 1 0.02 mg/kg/day Cohort 2 0.08 mg/kg/day Cohort 3a 0.04 mg/kg/day Cohort 3b 0.06 mg/kg/day
|
Outcome Measures
Primary Outcome Measures
- Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil [0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12]
- Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil [0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12]
- Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8) [0 to 8 hours post-dose on Day 1, Week 4, Week 12]
Secondary Outcome Measures
- Summary of All Treatment-emergent Adverse Events (TEAEs) [12 weeks]
TEAEs reported during the treatment period
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Ambulatory and non-ambulatory patients from age 5 - 16 years (5 years old and <17 years old) at time of screening with a clinical picture (see below) consistent with COL6-related dystrophy (COL6-RD) or LAMA2-related dystrophy (LAMA2-RD)
-
Under regular review at a neuromuscular center
-
On adequate double-barrier contraception (if of child-bearing potential)
-
Stable on any allowed concomitant medications for 1 month prior to run in Phase
-
Forced Vital Capacity (FVC) 30-80% of the predicted value and confirmed at Screening and Baseline visit(s)
For patients with Collagen VI-related dystrophy (COL6-RD)- required clinical picture:
• Muscle weakness: inability to walk or, if patient is still ambulatory, inability to run and > 5 s for 10 m walk
Genetic and Pathology:
• Molecular diagnosis of COL6-RD, defined by one dominant or two recessive mutation(s) in COL6A1, COL6A2 or COL6A3 known to cause the clinical picture,,
OR
• Histological diagnosis showing (i) absent or significantly decreased expression of collagen VI in muscle (overall reduction or basal lamina specific) or (ii) absent or significantly abnormal matrix in skin fibroblast culture
For patients with Laminin alpha 2 related dystrophy (LAMA2-RD) - required clinical picture:
• Muscle weakness: Inability to walk; if patient is still ambulatory, inability to run and
5 s for 10 m walk.
Genetics and Pathology:
• Either: 2 identified pathologic or probable pathologic mutations in LAMA2 gene
OR:
• 1 identified pathologic or probable pathologic mutation in LAMA 2 gene with evidence of decrease in laminin alpha 2 staining on muscle or skin biopsy
OR:
• Evidence of decrease in laminin alpha 2 staining on muscle or skin biopsy with matching clinical phenotype and no suspicion of alpha dystroglycanopathy (aDG-RD) (clinically or by staining on muscle biopsy)
Exclusion Criteria:
-
Use of any investigational drug other than the study medication within 12 weeks of study start.
-
Recurrent hospitalisation for chest infections in previous 2 years (≥2 per year)
-
Patients with respiratory parameters (eg: low pulmonary function test value i.e. <30% or need for brief course of daytime non-invasive ventilation) currently affected by short term medications, or acute illness/ conditions (conduct baseline assessments when the patient has recovered and no longer taking acute medication)
-
Any need for surgery (scoliosis, gastrostomy, other) in the preceding 24 weeks or foreseen during the course of the study.
-
Patient has an intercurrent significant medical condition or situation which in the opinion of the Investigator or the study Medical Monitor may put the patient at significant risk, confound the study results or interfere significantly with the patient's participation in the study
-
Failure to thrive, defined as:
-
Falling 20 percentiles (20/100) in body weight in the 12 weeks preceding Screening/Baseline (based on family report of weight loss and acquiring relevant medical records)
-
In patients below the 3rd percentile, any further drop in body weight percentile in the 12 weeks preceding Screening/Baseline (based on family report of weight loss and acquiring relevant medical records)
-
Weight less than 17kg at Baseline
-
Morbidly obese or grossly overweight (≥86 percentile BMI in children)
-
History of epilepsy or on antiepileptic medication at Screening/Baseline
-
Diabetes
-
On daytime Non Invasive Ventilation (NIV)
-
Intake of prohibited medication (as listed in Appendix I)
-
Anticipated need for anesthesia during the course of this study
-
Patients with renal impairment defined as urinary protein concentration ≥ 0.2 g/L
-
Patients with moderate to severe hepatic impairment
-
ALT ≥ 8x upper limit of normal (ULN) and total bilirubin 2x ULN (plus >35% 'direct' bilirubin), or
-
ALT ≥ 8x ULN and INR >1.5 or ALT >2x baseline levels, and total bilirubin > 2x ULN (plus >35% 'direct' bilirubin), or
-
ALT >2x baseline levels, and INR greater than 1.5,
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | NINDS | Bethesda | Maryland | United States | 20892 |
Sponsors and Collaborators
- Santhera Pharmaceuticals
Investigators
None specified.Study Documents (Full-Text)
More Information
Publications
None provided.- SNT-I-015
- FDA-OPD 5076
- NCT02326831
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day |
|---|---|---|---|---|
| Arm/Group Description | Omigapil Treatment, oral administration once per day after breakfast | Omigapil Treatment, oral administration once per day after breakfast | Omigapil Treatment, oral administration once per day after breakfast | Omigapil Treatment, oral administration once per day after breakfast |
| Period Title: Overall Study | ||||
| STARTED | 4 | 4 | 4 | 8 |
| COMPLETED | 4 | 4 | 4 | 8 |
| NOT COMPLETED | 0 | 0 | 0 | 0 |
Baseline Characteristics
| Arm/Group Title | Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day | Total |
|---|---|---|---|---|---|
| Arm/Group Description | Omigapil treatment, oral administration once per day after breakfast | Omigapil treatment, oral administration once per day after breakfast | Omigapil treatment, oral administration once per day after breakfast | Omigapil treatment, oral administration once per day after breakfast | Total of all reporting groups |
| Overall Participants | 4 | 4 | 4 | 8 | 20 |
| Age (years) [Mean (Standard Deviation) ] | |||||
| Mean (Standard Deviation) [years] |
8.9
(2.9)
|
10.2
(1.4)
|
9.3
(1.4)
|
11.2
(4.6)
|
10.2
(3.3)
|
| Sex: Female, Male (Count of Participants) | |||||
| Female |
2
50%
|
3
75%
|
3
75%
|
5
62.5%
|
13
65%
|
| Male |
2
50%
|
1
25%
|
1
25%
|
3
37.5%
|
7
35%
|
| Race/Ethnicity, Customized (Count of Participants) | |||||
| Aisan |
0
0%
|
1
25%
|
0
0%
|
0
0%
|
1
5%
|
| Black |
0
0%
|
0
0%
|
0
0%
|
1
12.5%
|
1
5%
|
| Caucasian |
4
100%
|
3
75%
|
4
100%
|
7
87.5%
|
18
90%
|
Outcome Measures
| Title | Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil |
|---|---|
| Description | |
| Time Frame | 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day |
|---|---|---|---|---|
| Arm/Group Description | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast |
| Measure Participants | 4 | 4 | 4 | 8 |
| Day1 |
1.05
(32.4)
|
4.75
(24.9)
|
2.15
(62.8)
|
3.27
(86.4)
|
| Week 4 |
1.10
(55.1)
|
10.5
(57.3)
|
1.98
(31.2)
|
2.90
(96.1)
|
| Week 12 |
1.25
(37.8)
|
11.6
(51.6)
|
1.95
(31.9)
|
3.43
(55.2)
|
| Title | Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil |
|---|---|
| Description | |
| Time Frame | 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day |
|---|---|---|---|---|
| Arm/Group Description | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast |
| Measure Participants | 4 | 4 | 4 | 8 |
| Day 1 |
0.8
(3.9)
|
0.8
(1.5)
|
0.8
(3.9)
|
0.5
(4.0)
|
| Week 4 |
0.6
(1.5)
|
0.5
(2.0)
|
0.5
(1.0)
|
1.0
(4.0)
|
| Week 12 |
1.00
(1.6)
|
0.9
(1.4)
|
0.8
(1.0)
|
1.0
(4.0)
|
| Title | Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8) |
|---|---|
| Description | |
| Time Frame | 0 to 8 hours post-dose on Day 1, Week 4, Week 12 |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day |
|---|---|---|---|---|
| Arm/Group Description | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast |
| Measure Participants | 4 | 4 | 4 | 8 |
| Day 1 |
3.37
(23.0)
|
15.7
(38.5)
|
7.12
(34.4)
|
10.8
(69.5)
|
| Week 4 |
3.47
(48.2)
|
29.7
(64.1)
|
8.28
(15.7)
|
11.0
(77.8)
|
| Week 12 |
3.79
(47.9)
|
37.5
(31.0)
|
6.91
(18.5)
|
12.5
(91.6)
|
| Title | Summary of All Treatment-emergent Adverse Events (TEAEs) |
|---|---|
| Description | TEAEs reported during the treatment period |
| Time Frame | 12 weeks |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day |
|---|---|---|---|---|
| Arm/Group Description | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast | Omigapil Treatment Oral Administration once per day after breakfast |
| Measure Participants | 4 | 4 | 4 | 8 |
| Treatment-emergent TEAEs |
41
|
40
|
29
|
75
|
| Serious TEAEs |
0
|
0
|
0
|
0
|
| Fatal TEAEs |
0
|
0
|
0
|
0
|
Adverse Events
| Time Frame | Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment | |||||||
|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | ||||||||
| Arm/Group Title | Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day | ||||
| Arm/Group Description | Omigapil Treatment, oral Administration once per day after breakfast | Omigapil Treatment, oral Administration once per day after breakfast | Omigapil Treatment, oral Administration once per day after breakfast | Omigapil Treatment, oral Administration once per day after breakfast | ||||
All Cause Mortality |
||||||||
| Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/4 (0%) | 0/4 (0%) | 0/4 (0%) | 0/8 (0%) | ||||
Serious Adverse Events |
||||||||
| Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/4 (0%) | 0/4 (0%) | 0/4 (0%) | 0/8 (0%) | ||||
Other (Not Including Serious) Adverse Events |
||||||||
| Cohort 1 0.02 mg/kg/Day | Cohort 2 0.08 mg/kg/Day | Cohort 3a 0.04 mg/kg/Day | Cohort 3b 0.06 mg/kg/Day | |||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 4/4 (100%) | 4/4 (100%) | 4/4 (100%) | 8/8 (100%) | ||||
| Blood and lymphatic system disorders | ||||||||
| Anaemia | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Ear and labyrinth disorders | ||||||||
| Ear congestion | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Eye disorders | ||||||||
| Ocular hyperaemia | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Gastrointestinal disorders | ||||||||
| Abdominal pain upper | 0/4 (0%) | 0 | 1/4 (25%) | 2 | 2/4 (50%) | 2 | 1/8 (12.5%) | 1 |
| Diarrhoea | 1/4 (25%) | 1 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 2/8 (25%) | 2 |
| Faeces discoloured | 1/4 (25%) | 2 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Nausea | 1/4 (25%) | 2 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 2/8 (25%) | 2 |
| Toothache | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/8 (0%) | 0 |
| Vomiting | 2/4 (50%) | 3 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| General disorders | ||||||||
| Chest pain | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Fatigue | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Injection site pain | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Pyrexia | 2/4 (50%) | 3 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Hepatobiliary disorders | ||||||||
| Gallbladder polyp | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Immune system disorders | ||||||||
| Hypersensitivity | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Infections and infestations | ||||||||
| Influenza | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Nasopharyngitis | 1/4 (25%) | 2 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Pharyngitis streptococcal | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Sinusitis | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Stoma site cellulitis | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Upper respiratory tract infection | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 3/8 (37.5%) | 3 |
| Injury, poisoning and procedural complications | ||||||||
| Arthropod bite | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 4/8 (50%) | 5 |
| Burns second degree | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Contusion | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Exposure to communicable disease | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Head injury | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Procedural pain | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Road traffic accident | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Skin abrasion | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Investigations | ||||||||
| Blood cholesterol increased | 2/4 (50%) | 2 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Blood thyroid stimulating hormone increased | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Blood triglycerides increased | 0/4 (0%) | 0 | 1/4 (25%) | 3 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Granulocyte count increased | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Protein total decreased | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Pulmonary function test decreased | 2/4 (50%) | 2 | 1/4 (25%) | 1 | 3/4 (75%) | 3 | 4/8 (50%) | 5 |
| Red blood cells urine | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Ultrasound liver abnormal | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| White blood cells urine positive | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 1/4 (25%) | 1 | 0/8 (0%) | 0 |
| Metabolism and nutrition disorders | ||||||||
| Dehydration | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/8 (0%) | 0 |
| Hypercholesterolaemia | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 3/8 (37.5%) | 4 |
| Musculoskeletal and connective tissue disorders | ||||||||
| Arthralgia | 1/4 (25%) | 2 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Muscle spasms | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Musculoskeletal pain | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Pain in extremity | 1/4 (25%) | 1 | 3/4 (75%) | 3 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Nervous system disorders | ||||||||
| Headache | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 3/8 (37.5%) | 12 |
| Paraesthesia | 0/4 (0%) | 0 | 1/4 (25%) | 2 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Psychiatric disorders | ||||||||
| Anorexia and bulimia syndrome | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Anxiety | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Attention déficit / hyperactivity disorder | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Irritability | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 2/8 (25%) | 3 |
| Renal and urinary disorders | ||||||||
| Ketonuria | 0/4 (0%) | 0 | 2/4 (50%) | 2 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Pollakiuria | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Proteinuria | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Reproductive system and breast disorders | ||||||||
| Amenorrhoea | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Dysmenorrhoea | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 2 |
| Respiratory, thoracic and mediastinal disorders | ||||||||
| Cough | 2/4 (50%) | 3 | 2/4 (50%) | 3 | 3/4 (75%) | 8 | 2/8 (25%) | 2 |
| Epistaxis | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Nasal congestion | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 4/8 (50%) | 4 |
| Oropharyngeal pain | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 2 |
| Rhinorrhoea | 4/4 (100%) | 6 | 1/4 (25%) | 3 | 4/4 (100%) | 9 | 2/8 (25%) | 2 |
| Sneezing | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Tachypnoea | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Upper-airway cough syndrome | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/8 (0%) | 0 |
| Skin and subcutaneous tissue disorders | ||||||||
| Blister | 1/4 (25%) | 1 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Pruritus | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 1/4 (25%) | 1 | 1/8 (12.5%) | 1 |
| Rash | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Skin burning sensation | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/8 (12.5%) | 1 |
| Skin disorder | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Vascular disorders | ||||||||
| Flushing | 1/4 (25%) | 2 | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 0/8 (0%) | 0 |
| Hypotension | 0/4 (0%) | 0 | 0/4 (0%) | 0 | 1/4 (25%) | 1 | 1/8 (12.5%) | 1 |
Limitations/Caveats
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
Cooperative Research and Development Agreement
Results Point of Contact
| Name/Title | Roxana Drake |
|---|---|
| Organization | Santhera Pharmaceuticals |
| Phone | +41 61 906 89 29 |
| roxana.drake@santhera.com |
- SNT-I-015
- FDA-OPD 5076
- NCT02326831