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

• ClinicalTrials.gov Identifier: NCT00004646
• Recruitment Status: Completed
• First Posted: February 25, 2000
• Last Update Posted: June 24, 2005
• Sponsor: National Center for Research Resources (NCRR)
• Collaborators: National Institute of Neurological Disorders and Stroke (NINDS); University of Rochester
• Information provided by: Office of Rare Diseases (ORD)

Tracking Information

• First Submitted Date: February 24, 2000
• First Posted Date: February 25, 2000
• Last Update Posted Date: June 24, 2005
• Study Start Date: April 1995
• Primary Completion Date: Not Provided
• Current Primary Outcome Measures: Not Provided
• Original Primary Outcome Measures: Not Provided
• Change History: No Changes Posted
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Phase III Randomized, Double-Blind Study of Prednisone for Duchenne Muscular Dystrophy
• Official Title: Not Provided

Brief Summary

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

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

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

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

• 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 Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Masking: Double; Primary Purpose: Treatment
• Condition: Duchenne Muscular Dystrophy
• Intervention: Drug: prednisone
• Study Arms: Not Provided
• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Enrollment (submitted: June 23, 2005): 20
• Original Enrollment: Same as current
• Study Completion Date: Not Provided
• Primary Completion Date: Not Provided

Eligibility Criteria

PROTOCOL ENTRY CRITERIA:

• Ambulatory males with Duchenne muscular dystrophy
• No medical/psychiatric contraindication to protocol therapy
• No requirement for regular use of prescription medication

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 5 Years to 15 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Not Provided

Administrative Information

• NCT Number: NCT00004646
• Other Study ID Numbers: 199/11695; URMC-2251
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Not Provided
• Original Responsible Party: Same as current
• Current Study Sponsor: National Center for Research Resources (NCRR)
• Original Study Sponsor: Same as current

Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS)
• University of Rochester

Investigators

• Study Chair: Robert Griggs; University of Rochester

• PRS Account: Office of Rare Diseases (ORD)
• Verification Date: December 2001