Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
Status:
Recruiting
Trial end date:
2022-04-01
Target enrollment:
Participant gender:
Summary
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.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Children's National Research Institute Children's Research Institute