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

This study has been completed.

First Received: February 24, 2000 Last Updated: June 23, 2005 History of Changes

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)
ClinicalTrials.gov Identifier:	NCT00004646

Purpose

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.

Condition	Intervention	Phase
Duchenne Muscular Dystrophy	Drug: prednisone	Phase III

Study Type:	Interventional
Study Design:	Treatment, Randomized, Double-Blind

Resource links provided by NLM:

Genetics Home Reference related topics: Duchenne and Becker muscular dystrophy L1 syndrome

MedlinePlus related topics: Muscular Dystrophy

Drug Information available for: Prednisone

U.S. FDA Resources

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment:	20
Study Start Date:	April 1995

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.

Eligibility

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

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004646

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

More Information

No publications provided

Study ID Numbers:	199/11695, URMC-2251
Study First Received:	February 24, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004646 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by Office of Rare Diseases (ORD):

Duchenne muscular dystrophy
genetic diseases and dysmorphic syndromes
muscular dystrophy
neurologic and psychiatric disorders
rare disease

Additional relevant MeSH terms:

Anti-Inflammatory Agents
Prednisone
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Physiological Effects of Drugs
Nervous System Diseases
Hormones, Hormone Substitutes, and Hormone Antagonists
Glucocorticoids
Hormones
Pharmacologic Actions

Muscular Dystrophies
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn
Therapeutic Uses
Muscular Dystrophy, Duchenne
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on November 27, 2009