Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy

• Sponsor: National Center for Research Resources (NCRR) (NIH)
• Overall Status: Completed
• CT.gov ID: NCT00004646
• Collaborator: National Institute of Neurological Disorders and Stroke (NINDS) (NIH), University of Rochester (Other)
• Enrollment: 20

Study Details

Study Description

Brief Summary

OBJECTIVES: I. Characterize the effect of prednisone on muscle protein metabolism in patients with Duchenne muscular dystrophy.

1. Determine whether prednisone changes levels of insulin-like growth factor 1, growth hormone, and insulin.

2. Characterize the effect of prednisone on muscle morphometry and muscle localization of utrophin.

3. Compare the prednisone response in patients with Duchenne muscular dystrophy to that seen in normal individuals and in patients with facioscapulohumeral dystrophy.

Condition or Disease	Intervention/Treatment	Phase
Duchenne Muscular Dystrophy	Drug: prednisone	Phase 3

Detailed Description

PROTOCOL OUTLINE: This is a randomized, double-blind study. Patients are randomly assigned to prednisone or placebo. Therapy is administered daily for 12 weeks; prednisone is then tapered.

Study Design

• Study Type: Interventional
• Allocation: Randomized
• Masking: Double
• Primary Purpose: Treatment
• Study Start Date: Apr 1, 1995

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

• Ages Eligible for Study: 5 Years to 15 Years
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

PROTOCOL ENTRY CRITERIA:

• Ambulatory males with Duchenne muscular dystrophy

• No medical/psychiatric contraindication to protocol therapy

• No requirement for regular use of prescription medication

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• National Center for Research Resources (NCRR)
• National Institute of Neurological Disorders and Stroke (NINDS)
• University of Rochester

Investigators

• Study Chair: Robert Griggs, University of Rochester

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00004646
• Other Study ID Numbers: 199/11695; URMC-2251
• First Posted: Feb 25, 2000
• Last Update Posted: Jun 24, 2005
• Last Verified: Dec 1, 2001

• Keywords provided by , ,: Duchenne muscular dystrophy; genetic diseases and dysmorphic syndromes; muscular dystrophy; neurologic and psychiatric disorders; rare disease
• Additional relevant MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Prednisone

Study Results

No Results Posted as of Jun 24, 2005