PatientCrossroads CMD


           

CALLISTO Omigapil Study (Collagen VI & LAMA2)

Study Start:  January 2015
 
Study Name
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)

Purpose
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. 
 
 
Study Details
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.
 
Eligibility

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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)

Contact 
Gilberto V. Averion, BSN, RN
Clinical Research Nurse
Neurogenetics Branch/NINDS
National Institutes of Health
10 Center Drive
Building 10, Room 12N210, MSC 1477
Bethesda, MD 20892-1477
Phone: 301-594-2760
Fax: 301-480-3007
Email: This e-mail address is being protected from spambots. You need JavaScript enabled to view it

Study Sponsor
Santhera Pharmaceuticals (Switzerland) Ltd.

ClinicalTrials.gov ID NCT01805024