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

NCT03936894 | Unknown Phase 1 DMC FDA Drug

• 1 other identifier: 10234
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Keywords

Canakinumab; Biomarker

Outcome Measures

Primary Outcomes (4)

• Clinical adverse events

  Monitor for changes in health status related to medication use

  2 weeks

• Laboratory adverse events

  Monitor for changes in laboratory results related to medication use

  2 weeks

• Clinical adverse events

  Monitor for changes in health status related to medication use

  4 weeks

• Laboratory adverse events

  Monitor for changes in laboratory results related to medication use

  4 weeks

Secondary Outcomes (2)

• Changes in serum biomarkers of inflammation after treatment

  2 weeks

• Changes in serum biomarkers of inflammation after treatment

  4 weeks

Study Arms (1)

Treatment

EXPERIMENTAL

Canakinumab treatment

Drug: Canakinumab Injection [Ilaris]

Interventions

Canakinumab Injection [Ilaris] DRUG

Subject receives subcutaneous injection of canakinumab 2 mg/ kg or 4 mg/kg

Treatment

Eligibility Criteria

• Age: 2 Years+
• Gender Eligibility Details: Only males with genetically confirmed Duchenne muscular dystrophy are eligible
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 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.

Sponsors & Collaborators

• Children's National Research Institute: lead
• Foundation to Eradicate Duchenne: collaborator

Study Sites (1)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

canakinumab

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Christopher Spurney

  Children's National Research Institute

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: This is an open-label, single ascending dose pilot trial

Study Record Dates

• First Submitted: April 29, 2019
• First Posted: May 3, 2019
• Study Start: May 1, 2019
• Primary Completion: April 1, 2024
• Study Completion: April 1, 2024
• Last Updated: September 21, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)