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

National Center for Research Resources (NCRR)

Status and phase: Completed; Phase 3

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: prednisone

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT00004646

199/11695

URMC-2251

Details and patient eligibility

About

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.

Full 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.

Sex: Male

Ages: 5 to 15 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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