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

NCT00033813 | Completed Phase 2

• 1 other identifier: KUL0401
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Muscular Dystrophy, Duchenne

Keywords

Duchenne; muscular; dystrophy; controlled clinical trial; CINRG

Interventions

Oxatomide (tinset) DRUG

Eligibility Criteria

• Age: 5 Years - 10 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• to 10 years of age
• ambulatory
• 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.
• glucocorticosteroid-naive (i.e. has not been treated with prednisone or deflazacort within the past year)
• Evidence of muscle weakness by MRC score or clinical functional evaluation
• QMT biceps score variability no greater than 10% between screening visits

You may not qualify if:

• Inability to suitably cooperate with strength assessments
• Symptomatic DMD carrier
• Use of oxatomide (or other anti-histamine drugs) within the last 6 months for DMD or any other disease
• Use of creatine monohydrate or glutamine within the last 6 months
• Use of carnitine, Coenzyme Q10, other amino acids or any herbal medications within the last 3 months
• History of symptomatic cardiomyopathy
• History of impairment of hepatic function
• History of significant concomitant illness or significant impairment of renal function.

Sponsors & Collaborators

• Cooperative International Neuromuscular Research Group: lead

Study Sites (1)

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

Related Links

• The Cooperative International Neuromuscular Research Group

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

oxatomide

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 10, 2002
• First Posted: April 11, 2002
• Study Start: January 1, 2002
• Primary Completion: December 1, 2006
• Last Updated: March 10, 2014

Record last verified: 2014-03

Locations

US (1)