Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03936894
Recruitment Status : Recruiting
First Posted : May 3, 2019
Last Update Posted : May 8, 2019
Sponsor:
Collaborator:
Foundation to Eradicate Duchenne
Information provided by (Responsible Party):
Christopher Spurney, Children's Research Institute

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.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Canakinumab Injection [Ilaris] Phase 1 Phase 2

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.


Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
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
Estimated Study Start Date : May 2019
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : April 2021


Arm Intervention/treatment
Experimental: Treatment
Canakinumab treatment
Drug: Canakinumab Injection [Ilaris]
Subject receives subcutaneous injection of canakinumab 2 mg/ kg or 4 mg/kg




Primary Outcome Measures :
  1. Clinical adverse events [ Time Frame: 2 weeks ]
    Monitor for changes in health status related to medication use

  2. Laboratory adverse events [ Time Frame: 2 weeks ]
    Monitor for changes in laboratory results related to medication use

  3. Clinical adverse events [ Time Frame: 4 weeks ]
    Monitor for changes in health status related to medication use

  4. Laboratory adverse events [ Time Frame: 4 weeks ]
    Monitor for changes in laboratory results related to medication use


Secondary Outcome Measures :
  1. Changes in serum biomarkers of inflammation after treatment [ Time Frame: 2 weeks ]
    Monitor serum biomarker changes associated with anti-inflammatory properties including CD23, Protein C, CCL22, lymphotoxin a1/b1, CD49a, Ly9 and MMP-9, 12 and compare to baseline levels to demonstrate increase or decrease in biomarker levels

  2. Changes in serum biomarkers of inflammation after treatment [ Time Frame: 4 weeks ]
    Monitor serum biomarker changes associated with anti-inflammatory properties including CD23, Protein C, CCL22, lymphotoxin a1/b1, CD49a, Ly9 and MMP-9, 12 and compare to baseline levels to demonstrate increase or decrease in biomarker levels



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   2 Years to 5 Years   (Child)
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
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 and <6 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 or >6 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03936894


Contacts
Layout table for location contacts
Contact: Christopher Spurney, MD 2024762020 cspurney@childrensnational.org
Contact: Jessica Chong, PA jchong2@childrensnational.org

Locations
Layout table for location information
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   
Sponsors and Collaborators
Children's Research Institute
Foundation to Eradicate Duchenne
Investigators
Layout table for investigator information
Principal Investigator: Christopher Spurney Children's Research Institute

Layout table for additonal information
Responsible Party: Christopher Spurney, Associate Professor, Children's Research Institute
ClinicalTrials.gov Identifier: NCT03936894     History of Changes
Other Study ID Numbers: 10234
First Posted: May 3, 2019    Key Record Dates
Last Update Posted: May 8, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Keywords provided by Christopher Spurney, Children's Research Institute:
Canakinumab
Biomarker

Additional relevant MeSH terms:
Layout table for MeSH terms
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs