A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)

Muscular Dystrophy, Duchenne Clinical Trial

Official title:

A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00018109
• Other study ID #: NCRR-M01RR00036-5083
• Status: Completed
• Phase: Phase 3
• First received: July 3, 2001
• Last updated: June 23, 2005

Study information

• Verified date: December 2003
• Source: National Center for Research Resources (NCRR)
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial Summary

To establish a collaborative group of clinical trial centers, with standardized equipment and protocols, able to conduct both drug and gene therapy trials in DMD. To evaluate the therapeutic effect of glutamine and creatine monohydrate on muscle strength in children with DMD. To validate the use of QMT (quantitative muscle strength testing) and gait analysis in children with DMD as reliable tools to quantify muscle strength, monitor disease progression and assess therapeutic response.

Description:

Duchenne muscular dystrophy (DMD) is the most common lethal inherited disorder worldwide. Despite the exponential increase in our understanding of the disorder since the discovery and characterization of the causative gene and its product dystrophin in 1987, current therapeutic management remains largely supportive. Awaiting a final genetic cure to be available in the future, further investments in developing better drug therapies for DMD remain important. Not only because the uniform use of prednisone (the only drug proven to be beneficial) is hampered by potential adverse effects, but also because it may very well be the case that ultimately a combination of both gene and drug therapy will be needed to cure Duchenne children. Here, we test two compounds that have shown promise in a 45-drug screen in the mdx mouse model of Duchenne dystrophy.

The effect of glutamine (0.6/kg/day) and creatine monohydrate (5g/day) on muscle strength will be evaluated in a multi-center randomized double-blind placebo-controlled 3-arm study. Ambulant children aged 5-10 years with an established DMD diagnosis will be studied. Patients will undergo 2 screening evaluations within 2 weeks. Patients will be randomized into treatment groups on the second screening visits, followed by a 6-month treatment period. During the treatment period, patients will be evaluated at monthly intervals. The primary endpoints are percentage change in average muscle strength score and QMT performance for specific muscle groups. Secondary endpoints include timed function tests, functional grades for arms and legs, and pulmonary function tests.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 0
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 10 Years

Eligibility

Ambulant children age 5-10 years with an established diagnosis of Duchenne Muscular Dystrophy

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• glutamine

• creatine monohydrate

Locations

Country	Name	City	State
United States	Washington University School of Medicine	St. Louis	Missouri

Sponsors (2)

Lead Sponsor	Collaborator
National Center for Research Resources (NCRR)	Children's Research Institute

Country where clinical trial is conducted

United States,