Overview
Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
Status:
Recruiting
Recruiting
Trial end date:
2022-04-01
2022-04-01
Target enrollment:
0
0
Participant gender:
Male
Male
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 2Accepts Healthy Volunteers?
NoDetails
Lead Sponsor:
Children's National Research Institute
Children's Research InstituteCollaborator:
Foundation to Eradicate DuchenneTreatments:
Antibodies, Monoclonal
Criteria
Inclusion Criteria:- Subject's parent or legal guardian has provided written informed consent/HIPAA
authorization prior to any study-related procedure
- Subject has a diagnosis of DMD
- Subject is ≥ 2 years of age at time of enrollment in the study
- Subject is naïve to treatment with glucocorticoids for DMD
- Subject is ambulatory
- Clinical laboratory test results are within the normal range at the Screening Visit,
or if abnormal, are not clinically significant (includes less than 5x normal for AST
and ALT), in the opinion of the Investigator. TB serology is negative.
- Subject and parent/guardian are willing and able to comply with, drug administration
plan, and follow up visits.
Exclusion Criteria:
- Subject is <2 years of age
- Subject has current or history of major renal or hepatic impairment, diabetes mellitus
or immunosuppression;
- Subject has current or history of chronic systemic fungal or viral infections;
- Subject has had an acute illness within 4 weeks prior to the first dose of study
medication;
- Subject received live vaccination within the previous month
- Subject has evidence of symptomatic cardiomyopathy [Note: Asymptomatic cardiac
abnormality on investigation would not be exclusionary];
- Subject is currently being treated or has received previous treatment with oral
glucocorticoids or other immunosuppressive agents [Notes: Past transient use of oral
glucocorticoids or other oral immunosuppressive agents for indication other than DMD
for no longer than 3 months cumulative, with last use at least 3 months prior to first
dose of study medication, will be considered for eligibility on a case-by-case basis.
Inhaled and/or topical glucocorticoids prescribed for an indication other than DMD are
permitted but must be administered at stable dose for at least 3 months prior to study
drug administration];
- Subject has previous or ongoing medical condition, medical history, physical findings
or laboratory abnormalities that could affect safety, make it unlikely that treatment
and follow-up will be correctly completed or impair the assessment of study results,
in the opinion of the Investigator;
- Subject is currently taking any other investigational drug or has taken any other
investigational drug within 3 months prior to the start of study treatment; Note: Any
parameter/test may be repeated at the Investigator's discretion during Screening to
determine reproducibility. In addition, subjects may be rescreened if ineligible due
to a transient condition which would prevent the subject from participating, such as
an upper respiratory tract infection or injury.