Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)

This study has been completed.

First Received on December 5, 2001. Last Updated on January 31, 2006 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00027391

Purpose

This is a study to determine whether albuterol or oxandrolone, alone or in combination, are able to increase strength and muscle mass in patients with FSHD. It also will determine if albuterol given in "pulsed" fashion will have more effect than when given continuously.

Condition	Intervention
Muscular Dystrophies	Drug: Albuterol Drug: Oxandrolone

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Efficacy Study Masking: Double-Blind Primary Purpose: Treatment
Official Title:	Clinical Trials of Albuterol and Oxandrolone in FSH Dystrophy

Resource links provided by NLM:

MedlinePlus related topics: Muscular Dystrophy

Drug Information available for: Oxandrolone Albuterol Levalbuterol hydrochloride Albuterol sulfate Levalbuterol tartrate

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	160
Study Start Date:	September 2001
Estimated Study Completion Date:	August 2004

Detailed Description:

Patients will be randomized to 1 of 4 groups: placebo, pulsed albuterol, oxandrolone, or both pulsed albuterol and oxandrolone. Treatment will continue for 52 weeks unless unacceptable side effects occur. Patients will undergo testing of muscle function. All patients will return for follow-up assessments at Weeks 4, 12, 26, and 52.

Eligibility

Ages Eligible for Study:	18 Years to 80 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion criteria:

Presence of 4q35 "small fragment" of less than 40 kb by standard DNA testing
Weakness of the facial muscles, including frontalis, orbicularis oculi, or orbicularis oris
Weakness of scapular stabilizers or foot dorsiflexors
Ambulatory
Weakness grade 2 or worse in the arm using upper extremity grading scale

Exclusion criteria:

Prior use of oral beta-2 agonists for a period of at least 1 year or within the past 3 months
Concurrent use of other sympathomimetic agents, antidepressants, or beta-2 receptor blockers
Pregnancy
Known hypersensitivity to anabolic steroids
Any medical or psychological condition that would interfere with the study
Requirement for a wheelchair

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00027391

Locations

United States, Ohio
Ohio State University Medical Center
Columbus, Ohio, United States, 43210

Sponsors and Collaborators

FDA Office of Orphan Products Development

Investigators

Principal Investigator:

John T. Kissel, M.D.

Ohio State University

More Information

No publications provided

ClinicalTrials.gov Identifier:	NCT00027391 History of Changes
Other Study ID Numbers:	FD-R-2029-01, FD-R-002029-01
Study First Received:	December 5, 2001
Last Updated:	January 31, 2006
Health Authority:	United States: Food and Drug Administration

Keywords provided by FDA Office of Orphan Products Development:

Muscle Weakness, Muscular Dystrophy

Additional relevant MeSH terms:

Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Albuterol
Oxandrolone
Tocolytic Agents
Reproductive Control Agents
Physiological Effects of Drugs
Pharmacologic Actions
Therapeutic Uses
Adrenergic beta-2 Receptor Agonists

Adrenergic beta-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Anti-Asthmatic Agents
Respiratory System Agents
Anabolic Agents
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Androgens

ClinicalTrials.gov processed this record on February 12, 2012