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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00033813
• Date of registration: 10/04/2002
• Prospective Registration: No
• Primary sponsor: Cooperative International Neuromuscular Research Group
• Public title: KUL0401: An Open-label Pilot Study of Oxatomide in Steroid-Naive Duchenne Muscular Dystrophy
• Date of first enrolment: January 2002
• Target sample size: 15
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00033813
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion 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.



Age minimum: 5 Years
Age maximum: 10 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophy, Duchenne

Intervention(s)

Drug: Oxatomide (tinset)

Secondary ID(s)

KUL0401

Source(s) of Monetary Support

Please refer to primary and secondary sponsors