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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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3 October 2023 |
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Main ID: |
NCT03936894 |
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Date of registration:
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29/04/2019 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy
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Scientific title:
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A Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) Assessing Safety and Biomarker Changes in Boys With Duchenne Muscular Dystrophy |
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Date of first enrolment:
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May 1, 2019 |
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Target sample size:
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3 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT03936894 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Christopher Spurney |
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Address:
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Telephone:
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Email:
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Affiliation:
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Children's National Research Institute |
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Key inclusion & exclusion criteria
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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.
Age minimum:
2 Years
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy
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Intervention(s)
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Drug: Canakinumab Injection [Ilaris]
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Primary Outcome(s)
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Laboratory adverse events
[Time Frame: 2 weeks]
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Laboratory adverse events
[Time Frame: 4 weeks]
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Clinical adverse events
[Time Frame: 2 weeks]
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Clinical adverse events
[Time Frame: 4 weeks]
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Secondary Outcome(s)
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Changes in serum biomarkers of inflammation after treatment
[Time Frame: 2 weeks]
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Changes in serum biomarkers of inflammation after treatment
[Time Frame: 4 weeks]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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