Ignite Creation Date:
2024-05-05 @ 11:22 AM
Last Modification Date:
2024-10-26 @ 9:06 AM
Study NCT ID:
NCT00018109
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
2001-07-03
Brief Title:
A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy DMD
Sponsor:
National Center for Research Resources NCRR
Organization:
National Center for Research Resources NCRR
Study Overview
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
Status:
COMPLETED
Status Verified Date:
2003-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
Detailed 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 06kgday and creatine monohydrate 5gday 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
The effect of glutamine 06kgday and creatine monohydrate 5gday 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
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?: