Trial Outcomes & Findings for 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) (NCT NCT01805024)

NCT ID: NCT01805024

Last Updated: 2021-09-24

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

20 participants

Primary outcome timeframe

0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12

Results posted on

2021-09-24

Participant Flow

Participant milestones

Participant milestones
Measure
Cohort 1 0.02 mg/kg/Day
Omigapil Treatment, oral administration once per day after breakfast
Cohort 2 0.08 mg/kg/Day
Omigapil Treatment, oral administration once per day after breakfast
Cohort 3a 0.04 mg/kg/Day
Omigapil Treatment, oral administration once per day after breakfast
Cohort 3b 0.06 mg/kg/Day
Omigapil Treatment, oral administration once per day after breakfast
Overall Study
STARTED
4
4
4
8
Overall Study
COMPLETED
4
4
4
8
Overall Study
NOT COMPLETED
0
0
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Cohort 1 0.02 mg/kg/Day
n=4 Participants
Omigapil treatment, oral administration once per day after breakfast
Cohort 2 0.08 mg/kg/Day
n=4 Participants
Omigapil treatment, oral administration once per day after breakfast
Cohort 3a 0.04 mg/kg/Day
n=4 Participants
Omigapil treatment, oral administration once per day after breakfast
Cohort 3b 0.06 mg/kg/Day
n=8 Participants
Omigapil treatment, oral administration once per day after breakfast
Total
n=20 Participants
Total of all reporting groups
Age, Continuous
8.9 years
STANDARD_DEVIATION 2.9 • n=5 Participants
10.2 years
STANDARD_DEVIATION 1.4 • n=7 Participants
9.3 years
STANDARD_DEVIATION 1.4 • n=5 Participants
11.2 years
STANDARD_DEVIATION 4.6 • n=4 Participants
10.2 years
STANDARD_DEVIATION 3.3 • n=21 Participants
Sex: Female, Male
Female
2 Participants
n=5 Participants
3 Participants
n=7 Participants
3 Participants
n=5 Participants
5 Participants
n=4 Participants
13 Participants
n=21 Participants
Sex: Female, Male
Male
2 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
3 Participants
n=4 Participants
7 Participants
n=21 Participants
Race/Ethnicity, Customized
Aisan
0 Participants
n=5 Participants
1 Participants
n=7 Participants
0 Participants
n=5 Participants
0 Participants
n=4 Participants
1 Participants
n=21 Participants
Race/Ethnicity, Customized
Black
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
1 Participants
n=4 Participants
1 Participants
n=21 Participants
Race/Ethnicity, Customized
Caucasian
4 Participants
n=5 Participants
3 Participants
n=7 Participants
4 Participants
n=5 Participants
7 Participants
n=4 Participants
18 Participants
n=21 Participants

PRIMARY outcome

Timeframe: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12

Outcome measures

Outcome measures
Measure
Cohort 1 0.02 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 2 0.08 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 3a 0.04 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 3b 0.06 mg/kg/Day
n=8 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil
Week 12
1.25 ng/mL
Geometric Coefficient of Variation 37.8
11.6 ng/mL
Geometric Coefficient of Variation 51.6
1.95 ng/mL
Geometric Coefficient of Variation 31.9
3.43 ng/mL
Geometric Coefficient of Variation 55.2
Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil
Day1
1.05 ng/mL
Geometric Coefficient of Variation 32.4
4.75 ng/mL
Geometric Coefficient of Variation 24.9
2.15 ng/mL
Geometric Coefficient of Variation 62.8
3.27 ng/mL
Geometric Coefficient of Variation 86.4
Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil
Week 4
1.10 ng/mL
Geometric Coefficient of Variation 55.1
10.5 ng/mL
Geometric Coefficient of Variation 57.3
1.98 ng/mL
Geometric Coefficient of Variation 31.2
2.90 ng/mL
Geometric Coefficient of Variation 96.1

PRIMARY outcome

Timeframe: 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12

Outcome measures

Outcome measures
Measure
Cohort 1 0.02 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 2 0.08 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 3a 0.04 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 3b 0.06 mg/kg/Day
n=8 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil
Day 1
0.8 h
Geometric Coefficient of Variation 3.9
0.8 h
Geometric Coefficient of Variation 1.5
0.8 h
Geometric Coefficient of Variation 3.9
0.5 h
Geometric Coefficient of Variation 4.0
Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil
Week 4
0.6 h
Geometric Coefficient of Variation 1.5
0.5 h
Geometric Coefficient of Variation 2.0
0.5 h
Geometric Coefficient of Variation 1.0
1.0 h
Geometric Coefficient of Variation 4.0
Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil
Week 12
1.00 h
Geometric Coefficient of Variation 1.6
0.9 h
Geometric Coefficient of Variation 1.4
0.8 h
Geometric Coefficient of Variation 1.0
1.0 h
Geometric Coefficient of Variation 4.0

PRIMARY outcome

Timeframe: 0 to 8 hours post-dose on Day 1, Week 4, Week 12

Outcome measures

Outcome measures
Measure
Cohort 1 0.02 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 2 0.08 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 3a 0.04 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 3b 0.06 mg/kg/Day
n=8 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8)
Week 4
3.47 mg.h/ml
Geometric Coefficient of Variation 48.2
29.7 mg.h/ml
Geometric Coefficient of Variation 64.1
8.28 mg.h/ml
Geometric Coefficient of Variation 15.7
11.0 mg.h/ml
Geometric Coefficient of Variation 77.8
Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8)
Day 1
3.37 mg.h/ml
Geometric Coefficient of Variation 23.0
15.7 mg.h/ml
Geometric Coefficient of Variation 38.5
7.12 mg.h/ml
Geometric Coefficient of Variation 34.4
10.8 mg.h/ml
Geometric Coefficient of Variation 69.5
Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8)
Week 12
3.79 mg.h/ml
Geometric Coefficient of Variation 47.9
37.5 mg.h/ml
Geometric Coefficient of Variation 31.0
6.91 mg.h/ml
Geometric Coefficient of Variation 18.5
12.5 mg.h/ml
Geometric Coefficient of Variation 91.6

SECONDARY outcome

Timeframe: 12 weeks

TEAEs reported during the treatment period

Outcome measures

Outcome measures
Measure
Cohort 1 0.02 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 2 0.08 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 3a 0.04 mg/kg/Day
n=4 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Cohort 3b 0.06 mg/kg/Day
n=8 Participants
Omigapil Treatment Oral Administration once per day after breakfast
Summary of All Treatment-emergent Adverse Events (TEAEs)
Treatment-emergent TEAEs
41 number of events
40 number of events
29 number of events
75 number of events
Summary of All Treatment-emergent Adverse Events (TEAEs)
Serious TEAEs
0 number of events
0 number of events
0 number of events
0 number of events
Summary of All Treatment-emergent Adverse Events (TEAEs)
Fatal TEAEs
0 number of events
0 number of events
0 number of events
0 number of events

Adverse Events

Cohort 1 0.02 mg/kg/Day

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Cohort 2 0.08 mg/kg/Day

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Cohort 3a 0.04 mg/kg/Day

Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths

Cohort 3b 0.06 mg/kg/Day

Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Cohort 1 0.02 mg/kg/Day
n=4 participants at risk
Omigapil Treatment, oral Administration once per day after breakfast
Cohort 2 0.08 mg/kg/Day
n=4 participants at risk
Omigapil Treatment, oral Administration once per day after breakfast
Cohort 3a 0.04 mg/kg/Day
n=4 participants at risk
Omigapil Treatment, oral Administration once per day after breakfast
Cohort 3b 0.06 mg/kg/Day
n=8 participants at risk
Omigapil Treatment, oral Administration once per day after breakfast
Blood and lymphatic system disorders
Anaemia
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Ear and labyrinth disorders
Ear congestion
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Eye disorders
Ocular hyperaemia
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Gastrointestinal disorders
Abdominal pain upper
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
50.0%
2/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Gastrointestinal disorders
Diarrhoea
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
2/8 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Gastrointestinal disorders
Faeces discoloured
25.0%
1/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Gastrointestinal disorders
Nausea
25.0%
1/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
2/8 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Gastrointestinal disorders
Toothache
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Gastrointestinal disorders
Vomiting
50.0%
2/4 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
General disorders
Chest pain
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
General disorders
Fatigue
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
General disorders
Injection site pain
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
General disorders
Pyrexia
50.0%
2/4 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Hepatobiliary disorders
Gallbladder polyp
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Immune system disorders
Hypersensitivity
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Infections and infestations
Influenza
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Infections and infestations
Nasopharyngitis
25.0%
1/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Infections and infestations
Pharyngitis streptococcal
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Infections and infestations
Sinusitis
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Infections and infestations
Stoma site cellulitis
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Infections and infestations
Upper respiratory tract infection
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
37.5%
3/8 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Injury, poisoning and procedural complications
Arthropod bite
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
50.0%
4/8 • Number of events 5 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Injury, poisoning and procedural complications
Burns second degree
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Injury, poisoning and procedural complications
Contusion
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Injury, poisoning and procedural complications
Exposure to communicable disease
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Injury, poisoning and procedural complications
Head injury
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Injury, poisoning and procedural complications
Procedural pain
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Injury, poisoning and procedural complications
Road traffic accident
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Injury, poisoning and procedural complications
Skin abrasion
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
Blood cholesterol increased
50.0%
2/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
Blood thyroid stimulating hormone increased
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
Blood triglycerides increased
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
Granulocyte count increased
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
Protein total decreased
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
Pulmonary function test decreased
50.0%
2/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
75.0%
3/4 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
50.0%
4/8 • Number of events 5 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
Red blood cells urine
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
Ultrasound liver abnormal
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Investigations
White blood cells urine positive
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Metabolism and nutrition disorders
Dehydration
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Metabolism and nutrition disorders
Hypercholesterolaemia
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
37.5%
3/8 • Number of events 4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Musculoskeletal and connective tissue disorders
Arthralgia
25.0%
1/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Musculoskeletal and connective tissue disorders
Muscle spasms
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Musculoskeletal and connective tissue disorders
Pain in extremity
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
75.0%
3/4 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Nervous system disorders
Headache
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
37.5%
3/8 • Number of events 12 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Nervous system disorders
Paraesthesia
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Psychiatric disorders
Anorexia and bulimia syndrome
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Psychiatric disorders
Anxiety
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Psychiatric disorders
Attention déficit / hyperactivity disorder
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Psychiatric disorders
Irritability
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
2/8 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Renal and urinary disorders
Ketonuria
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
50.0%
2/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Renal and urinary disorders
Pollakiuria
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Renal and urinary disorders
Proteinuria
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Reproductive system and breast disorders
Amenorrhoea
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Reproductive system and breast disorders
Dysmenorrhoea
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Respiratory, thoracic and mediastinal disorders
Cough
50.0%
2/4 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
50.0%
2/4 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
75.0%
3/4 • Number of events 8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
2/8 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Respiratory, thoracic and mediastinal disorders
Epistaxis
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Respiratory, thoracic and mediastinal disorders
Nasal congestion
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
50.0%
4/8 • Number of events 4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
100.0%
4/4 • Number of events 6 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 3 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
100.0%
4/4 • Number of events 9 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
2/8 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Respiratory, thoracic and mediastinal disorders
Sneezing
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Respiratory, thoracic and mediastinal disorders
Tachypnoea
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Respiratory, thoracic and mediastinal disorders
Upper-airway cough syndrome
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Skin and subcutaneous tissue disorders
Blister
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Skin and subcutaneous tissue disorders
Pruritus
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Skin and subcutaneous tissue disorders
Rash
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Skin and subcutaneous tissue disorders
Skin burning sensation
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Skin and subcutaneous tissue disorders
Skin disorder
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Vascular disorders
Flushing
25.0%
1/4 • Number of events 2 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/8 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Vascular disorders
Hypotension
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
0.00%
0/4 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
25.0%
1/4 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
12.5%
1/8 • Number of events 1 • Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment

Additional Information

Roxana Drake

Santhera Pharmaceuticals

Phone: +41 61 906 89 29

Results disclosure agreements

  • Principal investigator is a sponsor employee Cooperative Research and Development Agreement
  • Publication restrictions are in place

Restriction type: OTHER