Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

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) (CALLISTO)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01805024
Recruitment Status : Completed
First Posted : March 5, 2013
Results First Posted : September 20, 2019
Last Update Posted : September 20, 2019
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Congenital Muscular Dystrophy
Intervention Drug: Omigapil
Enrollment 20
Recruitment Details  
Pre-assignment Details  
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
Hide 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
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
Hide 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 Number of Baseline Participants 4 4 4 8 20
Hide Baseline Analysis Population Description
Baseline characteristics per cohort
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 4 participants 4 participants 4 participants 8 participants 20 participants
8.9  (2.9) 10.2  (1.4) 9.3  (1.4) 11.2  (4.6) 10.2  (3.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 4 participants 8 participants 20 participants
Female
2
  50.0%
3
  75.0%
3
  75.0%
5
  62.5%
13
  65.0%
Male
2
  50.0%
1
  25.0%
1
  25.0%
3
  37.5%
7
  35.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 4 participants 4 participants 4 participants 8 participants 20 participants
Aisan
0
   0.0%
1
  25.0%
0
   0.0%
0
   0.0%
1
   5.0%
Black
0
   0.0%
0
   0.0%
0
   0.0%
1
  12.5%
1
   5.0%
Caucasian
4
 100.0%
3
  75.0%
4
 100.0%
7
  87.5%
18
  90.0%
1.Primary Outcome
Title Pharmacokinetic Profile of Omigapil:Maximum Observed Plasma Concentration (Cmax) of Omigapil
Hide Description [Not Specified]
Time Frame 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12
Hide Outcome Measure Data
Hide 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
Hide 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
Overall Number of Participants Analyzed 4 4 4 8
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng/mL
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%)
2.Primary Outcome
Title Pharmacokinetic Profile of Omigapil: Time at Which Cmax Was Apparent (Tmax) of Omigapil
Hide Description [Not Specified]
Time Frame 0.5, 1, 1.5, 2, 4 and 8 hours post-dose on Day 1, Week 4 and Week 12
Hide Outcome Measure Data
Hide 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
Hide 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
Overall Number of Participants Analyzed 4 4 4 8
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: h
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%)
3.Primary Outcome
Title Pharmacokinetic Profile of Omigapil Area Under the Plasma Concentration Versus Time Curve From Time Zero to 8h Post-dose (AUC0-8)
Hide Description [Not Specified]
Time Frame 0 to 8 hours post-dose on Day 1, Week 4, Week 12
Hide Outcome Measure Data
Hide 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
Hide 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
Overall Number of Participants Analyzed 4 4 4 8
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: mg.h/ml
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%)
4.Secondary Outcome
Title Summary of All Treatment-emergent Adverse Events (TEAEs)
Hide Description TEAEs reported during the treatment period
Time Frame 12 weeks
Hide Outcome Measure Data
Hide 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
Hide 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
Overall Number of Participants Analyzed 4 4 4 8
Measure Type: Number
Unit of Measure: number of events
Treatment-emergent TEAEs 41 40 29 75
Serious TEAEs 0 0 0 0
Fatal TEAEs 0 0 0 0
Time Frame Treatment-emergent AEs (TEAEs) collected during the 12 weeks of treatment
Adverse Event Reporting 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
Hide 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 (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/4 (0.00%)      0/4 (0.00%)      0/4 (0.00%)      0/8 (0.00%)    
Show Serious Adverse Events Hide 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 (0.00%)      0/4 (0.00%)      0/4 (0.00%)      0/8 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 3%
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 (100.00%)      4/4 (100.00%)      4/4 (100.00%)      8/8 (100.00%)    
Blood and lymphatic system disorders         
Anaemia   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Ear and labyrinth disorders         
Ear congestion   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Eye disorders         
Ocular hyperaemia   1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Gastrointestinal disorders         
Abdominal pain upper   0/4 (0.00%)  0 1/4 (25.00%)  2 2/4 (50.00%)  2 1/8 (12.50%)  1
Diarrhoea   1/4 (25.00%)  1 1/4 (25.00%)  1 0/4 (0.00%)  0 2/8 (25.00%)  2
Faeces discoloured   1/4 (25.00%)  2 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Nausea   1/4 (25.00%)  2 0/4 (0.00%)  0 0/4 (0.00%)  0 2/8 (25.00%)  2
Toothache   0/4 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/8 (0.00%)  0
Vomiting   2/4 (50.00%)  3 1/4 (25.00%)  1 0/4 (0.00%)  0 1/8 (12.50%)  1
General disorders         
Chest pain   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Fatigue   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Injection site pain   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Pyrexia   2/4 (50.00%)  3 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Hepatobiliary disorders         
Gallbladder polyp   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Immune system disorders         
Hypersensitivity   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Infections and infestations         
Influenza   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Nasopharyngitis   1/4 (25.00%)  2 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Pharyngitis streptococcal   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Sinusitis   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Stoma site cellulitis   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Upper respiratory tract infection   1/4 (25.00%)  1 0/4 (0.00%)  0 1/4 (25.00%)  1 3/8 (37.50%)  3
Injury, poisoning and procedural complications         
Arthropod bite   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 4/8 (50.00%)  5
Burns second degree   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Contusion   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Exposure to communicable disease   1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Head injury   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Procedural pain   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Road traffic accident   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Skin abrasion   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Investigations         
Blood cholesterol increased   2/4 (50.00%)  2 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Blood thyroid stimulating hormone increased   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Blood triglycerides increased   0/4 (0.00%)  0 1/4 (25.00%)  3 0/4 (0.00%)  0 0/8 (0.00%)  0
Granulocyte count increased   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Protein total decreased   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Pulmonary function test decreased   2/4 (50.00%)  2 1/4 (25.00%)  1 3/4 (75.00%)  3 4/8 (50.00%)  5
Red blood cells urine   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Ultrasound liver abnormal   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
White blood cells urine positive   0/4 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  1 0/8 (0.00%)  0
Metabolism and nutrition disorders         
Dehydration   0/4 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/8 (0.00%)  0
Hypercholesterolaemia   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 3/8 (37.50%)  4
Musculoskeletal and connective tissue disorders         
Arthralgia   1/4 (25.00%)  2 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Muscle spasms   1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Musculoskeletal pain   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Pain in extremity   1/4 (25.00%)  1 3/4 (75.00%)  3 0/4 (0.00%)  0 1/8 (12.50%)  1
Nervous system disorders         
Headache   1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 3/8 (37.50%)  12
Paraesthesia   0/4 (0.00%)  0 1/4 (25.00%)  2 0/4 (0.00%)  0 0/8 (0.00%)  0
Psychiatric disorders         
Anorexia and bulimia syndrome   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Anxiety   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Attention déficit / hyperactivity disorder   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Irritability   1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 2/8 (25.00%)  3
Renal and urinary disorders         
Ketonuria   0/4 (0.00%)  0 2/4 (50.00%)  2 0/4 (0.00%)  0 0/8 (0.00%)  0
Pollakiuria   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Proteinuria   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Reproductive system and breast disorders         
Amenorrhoea   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Dysmenorrhoea   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  2
Respiratory, thoracic and mediastinal disorders         
Cough   2/4 (50.00%)  3 2/4 (50.00%)  3 3/4 (75.00%)  8 2/8 (25.00%)  2
Epistaxis   1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Nasal congestion   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 4/8 (50.00%)  4
Oropharyngeal pain   1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  2
Rhinorrhoea   4/4 (100.00%)  6 1/4 (25.00%)  3 4/4 (100.00%)  9 2/8 (25.00%)  2
Sneezing   1/4 (25.00%)  1 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Tachypnoea   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Upper-airway cough syndrome   0/4 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 0/8 (0.00%)  0
Skin and subcutaneous tissue disorders         
Blister   1/4 (25.00%)  1 1/4 (25.00%)  1 0/4 (0.00%)  0 1/8 (12.50%)  1
Pruritus   0/4 (0.00%)  0 1/4 (25.00%)  1 1/4 (25.00%)  1 1/8 (12.50%)  1
Rash   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Skin burning sensation   0/4 (0.00%)  0 0/4 (0.00%)  0 0/4 (0.00%)  0 1/8 (12.50%)  1
Skin disorder   0/4 (0.00%)  0 1/4 (25.00%)  1 0/4 (0.00%)  0 0/8 (0.00%)  0
Vascular disorders         
Flushing   1/4 (25.00%)  2 0/4 (0.00%)  0 0/4 (0.00%)  0 0/8 (0.00%)  0
Hypotension   0/4 (0.00%)  0 0/4 (0.00%)  0 1/4 (25.00%)  1 1/8 (12.50%)  1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Cooperative Research and Development Agreement
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Roxana Drake
Organization: Santhera Pharmaceuticals
Phone: +41 61 906 89 29
EMail: roxana.drake@santhera.com
Layout table for additonal information
Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01805024     History of Changes
Obsolete Identifiers: NCT02326831
Other Study ID Numbers: SNT-I-015
FDA-OPD 5076 ( Other Identifier: FDA )
First Submitted: March 4, 2013
First Posted: March 5, 2013
Results First Submitted: April 3, 2019
Results First Posted: September 20, 2019
Last Update Posted: September 20, 2019