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)
To understand how fast and well children’s bodies can process a new medicine called Omigapil in patients with Congenital Muscular Dystrophy (CMD) and what might be suitable tests for future clinical research studies with CMD patients using this new medicine.
There are 6 study visits required over 6 months, which will be conducted at the NINDS/NIH in Bethesda, MD. If you need to travel, the research study will pay for your travel.
- 5-16 years of age
- Diagnosed with COL6-related dystrophy or LAMA2–related dystrophy
- Forced Vital Capacity (FVC) 30-80% of the predicted value on Pulmonary Function Tests (PFTs)
- Patients who can walk or who are in a wheelchair are eligible to participate.
- Recurrent hospitalization for chest infections
- Weight less than 17kg (37 lbs), BMI > 85th percentile
- History of epilepsy or on antiepileptic medication
- Diabetes, renal impairment, hepatic impairment
- On daytime Non Invasive Ventilation (NIV)
Gilberto V. Averion, BSN, RN
Clinical Research Nurse
National Institutes of Health
10 Center Drive
Building 10, Room 12N210, MSC 1477
Bethesda, MD 20892-1477
Santhera Pharmaceuticals (Switzerland) Ltd.
ClinicalTrials.gov ID NCT01805024