KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier:
NCT00033813
First received: April 10, 2002
Last updated: March 7, 2014
Last verified: March 2014
  Purpose

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 Intervention Phase
Muscular Dystrophy, Duchenne
Drug: Oxatomide (tinset)
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Cooperative International Neuromuscular Research Group:

Estimated Enrollment: 15
Study Start Date: January 2002
Primary Completion Date: December 2006 (Final data collection date for primary outcome measure)
  Eligibility

Ages Eligible for Study:   5 Years to 10 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00033813

Locations
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
  More Information

No publications provided

Responsible Party: Cooperative International Neuromuscular Research Group
ClinicalTrials.gov Identifier: NCT00033813     History of Changes
Other Study ID Numbers: KUL0401
Study First Received: April 10, 2002
Last Updated: March 7, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by Cooperative International Neuromuscular Research Group:
Duchenne
muscular
dystrophy
controlled clinical trial
CINRG

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases
Oxatomide
Anti-Allergic Agents
Anti-Asthmatic Agents
Histamine Agents
Histamine Antagonists
Histamine H1 Antagonists
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Pharmacologic Actions
Physiological Effects of Drugs
Respiratory System Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on October 23, 2014