Condition or disease | Intervention/treatment | Phase |
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Duchenne Muscular Dystrophy | Drug: Canakinumab Injection [Ilaris] | Phase 1 Phase 2 |
The study is an open-label, single ascending dose study to assess safety and to evaluate short-term changes in biomarkers. The first 3 boys will receive canakinumab at a dose of 2 mg/kg, and the second 3 will receive a dose of 4 mg/kg. The study is comprised of four visits: a screening visit with baseline lab assessment, treatment day, a 10-14 day post-treatment evaluation with safety labs, and a 30 day post-treatment evaluation with safety labs. There will be a phone assessment on day 3-5 after treatment to screen for any side effects.
Clinical evaluation will screen for clinical AEs and SAEs. If dose escalation (4 mg/kg) must be terminated due to dose-limiting toxicities, the remaining subjects may be enrolled to evaluate the safety of canakinumab at the lower dose level (2 mg/kg).
One blood draw will occur after consent at least 48 hours prior to treatment day and on days 10-14 and 30 after the canakinumab injection to screen for laboratory abnormalities and collect serum for biomarker analysis. After obtaining consent, prior to treatment, 2 red top and 1 purple top 4 cc tubes will be obtained for screening labs and serum biomarker collection. Tuberculosis screening (Quantiferon-Gold) will use 4 blood tubes (grey, yellow, purple, green) with 1 cc of blood in each. At the following 2 visits with blood draws, two red top and one purple top collection tubes of 4 cc will be collected per subject. For red top blood collection tubes, one tube will be sent to the clinical lab for safety lab processing. The second tube will be processed in the research lab for serum biomarkers.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 6 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Intervention Model Description: | This is an open-label, single ascending dose pilot trial |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) Assessing Safety and Biomarker Changes in Boys With Duchenne Muscular Dystrophy |
Actual Study Start Date : | May 1, 2019 |
Estimated Primary Completion Date : | April 2022 |
Estimated Study Completion Date : | April 2022 |
Arm | Intervention/treatment |
---|---|
Experimental: Treatment
Canakinumab treatment
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Drug: Canakinumab Injection [Ilaris]
Subject receives subcutaneous injection of canakinumab 2 mg/ kg or 4 mg/kg
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Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | Male |
Gender Based Eligibility: | Yes |
Gender Eligibility Description: | Only males with genetically confirmed Duchenne muscular dystrophy are eligible |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Christopher Spurney, MD | 2024762020 | cspurney@childrensnational.org | |
Contact: Jessica Chong, PA | jchong2@childrensnational.org |
United States, District of Columbia | |
Children's National Medical Center | Recruiting |
Washington, District of Columbia, United States, 20010 | |
Contact: Christopher F Spurney, MD 202-476-2020 cspurney@childrensnational.org | |
Contact: Jessica Chong, PA jchong2@childrensnational.org |
Principal Investigator: | Christopher Spurney | Children's National Research Institute |
Tracking Information | |||||||||
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First Submitted Date ICMJE | April 29, 2019 | ||||||||
First Posted Date ICMJE | May 3, 2019 | ||||||||
Last Update Posted Date | February 24, 2021 | ||||||||
Actual Study Start Date ICMJE | May 1, 2019 | ||||||||
Estimated Primary Completion Date | April 2022 (Final data collection date for primary outcome measure) | ||||||||
Current Primary Outcome Measures ICMJE |
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Original Primary Outcome Measures ICMJE | Same as current | ||||||||
Change History | |||||||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||||||
Descriptive Information | |||||||||
Brief Title ICMJE | Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) in Duchenne Muscular Dystrophy | ||||||||
Official Title ICMJE | A Single Escalating Dose Pilot Trial of Canakinumab (ILARIS®) Assessing Safety and Biomarker Changes in Boys With Duchenne Muscular Dystrophy | ||||||||
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. | ||||||||
Detailed Description |
The study is an open-label, single ascending dose study to assess safety and to evaluate short-term changes in biomarkers. The first 3 boys will receive canakinumab at a dose of 2 mg/kg, and the second 3 will receive a dose of 4 mg/kg. The study is comprised of four visits: a screening visit with baseline lab assessment, treatment day, a 10-14 day post-treatment evaluation with safety labs, and a 30 day post-treatment evaluation with safety labs. There will be a phone assessment on day 3-5 after treatment to screen for any side effects. Clinical evaluation will screen for clinical AEs and SAEs. If dose escalation (4 mg/kg) must be terminated due to dose-limiting toxicities, the remaining subjects may be enrolled to evaluate the safety of canakinumab at the lower dose level (2 mg/kg). One blood draw will occur after consent at least 48 hours prior to treatment day and on days 10-14 and 30 after the canakinumab injection to screen for laboratory abnormalities and collect serum for biomarker analysis. After obtaining consent, prior to treatment, 2 red top and 1 purple top 4 cc tubes will be obtained for screening labs and serum biomarker collection. Tuberculosis screening (Quantiferon-Gold) will use 4 blood tubes (grey, yellow, purple, green) with 1 cc of blood in each. At the following 2 visits with blood draws, two red top and one purple top collection tubes of 4 cc will be collected per subject. For red top blood collection tubes, one tube will be sent to the clinical lab for safety lab processing. The second tube will be processed in the research lab for serum biomarkers. |
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Study Type ICMJE | Interventional | ||||||||
Study Phase ICMJE | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Intervention Model Description: This is an open-label, single ascending dose pilot trial Masking: None (Open Label)Primary Purpose: Treatment |
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Condition ICMJE | Duchenne Muscular Dystrophy | ||||||||
Intervention ICMJE | Drug: Canakinumab Injection [Ilaris]
Subject receives subcutaneous injection of canakinumab 2 mg/ kg or 4 mg/kg
|
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Study Arms ICMJE | Experimental: Treatment
Canakinumab treatment
Intervention: Drug: Canakinumab Injection [Ilaris]
|
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Publications * | Not Provided | ||||||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||||||
Recruitment Status ICMJE | Recruiting | ||||||||
Estimated Enrollment ICMJE |
6 | ||||||||
Original Estimated Enrollment ICMJE | Same as current | ||||||||
Estimated Study Completion Date ICMJE | April 2022 | ||||||||
Estimated Primary Completion Date | April 2022 (Final data collection date for primary outcome measure) | ||||||||
Eligibility Criteria ICMJE |
Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 2 Years and older (Child, Adult, Older Adult) | ||||||||
Accepts Healthy Volunteers ICMJE | No | ||||||||
Contacts ICMJE |
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Listed Location Countries ICMJE | United States | ||||||||
Removed Location Countries | |||||||||
Administrative Information | |||||||||
NCT Number ICMJE | NCT03936894 | ||||||||
Other Study ID Numbers ICMJE | 10234 | ||||||||
Has Data Monitoring Committee | Yes | ||||||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Christopher Spurney, Children's National Research Institute | ||||||||
Study Sponsor ICMJE | Children's National Research Institute | ||||||||
Collaborators ICMJE | Foundation to Eradicate Duchenne | ||||||||
Investigators ICMJE |
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PRS Account | Children's National Research Institute | ||||||||
Verification Date | February 2021 | ||||||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |