KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive 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. Read our disclaimer for details. Identifier: NCT00033813
Recruitment Status : Completed
First Posted : April 11, 2002
Last Update Posted : March 10, 2014
Information provided by (Responsible Party):
Cooperative International Neuromuscular Research Group

Brief Summary:
This study will help to determine the safety and efficacy of the mast cell stabilizer Oxatomide as a treatment for Duchenne muscular dystrophy (DMD). Boys with DMD who are enrolled in this study will should not have taken steroids to treat DMD for at least twelve months, and should not have taken any nutritional supplements for at least three months. Subjects will complete a two screening visits within a one-week period, and if enrolled will then have their strength tested monthly for three months before beginning therapy with Oxatomide. Once Oxatomide therapy is started, participants will have their strength tested monthly for six months. Following the six month treatment period, participants will be given the option to remain on Oxatomide until the study is completed.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: Oxatomide (tinset) Phase 2

Study Type : Interventional  (Clinical Trial)
Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : January 2002
Actual Primary Completion Date : December 2006

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 10 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Subject Inclusion Criteria

  1. 5 to 10 years of age
  2. ambulatory
  3. diagnosis of DMD confirmed by at least one of the following:

    • Positive x-linked family history of DMD in older male relatives (onset by 5 years, wheelchair bound by 12 years), or;
    • Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin deficiency, and clinical picture consistent with typical DMD, or;
    • Gene deletion test positive (missing one or more exons) in the central rod domain (exons 25-60) of dystrophin, where reading frame can be predicted as 'out of frame', and clinical picture consistent with typical DMD.
  4. glucocorticosteroid-naive (i.e. has not been treated with prednisone or deflazacort within the past year)
  5. Evidence of muscle weakness by MRC score or clinical functional evaluation
  6. QMT biceps score variability no greater than 10% between screening visits

Subject Exclusion Criteria

  1. Failure to achieve one or more of the inclusion criteria listed above
  2. Inability to suitably cooperate with strength assessments
  3. Symptomatic DMD carrier
  4. Use of oxatomide (or other anti-histamine drugs) within the last 6 months for DMD or any other disease
  5. Use of creatine monohydrate or glutamine within the last 6 months
  6. Use of carnitine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months
  7. History of symptomatic cardiomyopathy
  8. History of impairment of hepatic function
  9. History of significant concomitant illness or significant impairment of renal function.

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 identifier (NCT number): NCT00033813

United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010
Sponsors and Collaborators
Cooperative International Neuromuscular Research Group

Responsible Party: Cooperative International Neuromuscular Research Group Identifier: NCT00033813     History of Changes
Other Study ID Numbers: KUL0401
First Posted: April 11, 2002    Key Record Dates
Last Update Posted: March 10, 2014
Last Verified: March 2014

Keywords provided by Cooperative International Neuromuscular Research Group:
controlled clinical trial

Additional relevant 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
Histamine H1 Antagonists
Histamine Antagonists
Histamine Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs
Anti-Allergic Agents
Anti-Asthmatic Agents
Respiratory System Agents