Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy

Part of paid clinical trials in Washington D.C., District of Columbia.

Sponsor: Children's National Research Institute
Study ID: NCT03936894
Phase: PHASE1/PHASE2
Status: Unknown

Conditions

Duchenne Muscular Dystrophy

Eligibility Criteria

Sex: MALE
Age: 2 Years - N/A
Healthy Volunteers: Not accepted

Interventions

Canakinumab Injection [Ilaris] — DRUG
Subject receives subcutaneous injection of canakinumab 2 mg/ kg or 4 mg/kg

Study Details

Canakinumab is an anti-interleukin 1 beta (IL1β) antibody approved for use in young children with familial Mediterranean fever, systemic onset juvenile idiopathic arthritis and TNF-receptor associated periodic fever syndrome. This study is a pilot trial to investigate the effects of canakinumab on clinical safety and potential clinical efficacy as demonstrated by short-term changes in select serum biomarkers in a sample of young boys with DMD who are most likely to have high levels of muscle inflammation. Steroid naive DMD subjects aged greater than or equal to 2 years old to less than 6 years old will receive a single subcutaneous dose of canakinumab and undergo safety and serum biomarker monitoring for 30 days. The first 3 subjects will receive 2 mg/kg and if well tolerated, the second 3 subjects will receive 4 mg/kg.