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

March 7, 2014 updated by: Cooperative International Neuromuscular Research Group
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment

15

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Children's National Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 6 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cooperative International Neuromuscular Research Group

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

January 1, 2002

Primary Completion (Actual)

December 1, 2006

Study Registration Dates

First Submitted

April 10, 2002

First Submitted That Met QC Criteria

April 10, 2002

First Posted (Estimate)

April 11, 2002

Study Record Updates

Last Update Posted (Estimate)

March 10, 2014

Last Update Submitted That Met QC Criteria

March 7, 2014

Last Verified

March 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • KUL0401

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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