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Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00004685
Recruitment Status : Completed
First Posted : February 25, 2000
Last Update Posted : March 25, 2015
Information provided by:
FDA Office of Orphan Products Development

Brief Summary:

OBJECTIVES: I. Determine whether albuterol increases strength in patients with facioscapulohumeral dystrophy as measured by quantitative voluntary isometric contraction testing.

II. Determine whether albuterol increases muscle mass in this patient population as determined by 24 hour urinary creatinine excretion and dual energy x-ray absorptiometry (DEXA).

III. Examine the long term safety of albuterol in this patient population.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Facioscapulohumeral Drug: albuterol Not Applicable

Detailed Description:

PROTOCOL OUTLINE: This is a randomized, double blind, placebo controlled study. Patients are randomized into one of three treatment groups. The first group receives placebo. The second group receives low dose albuterol orally every 12 hours. The third group receives high dose albuterol orally every 12 hours. Treatment continues for 52 weeks unless unacceptable side effects occur.

All patients return for follow up assessments at weeks 4, 12, 24, and 52.

Completion date provided represents the completion date of the grant per OOPD records

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Study Type : Interventional  (Clinical Trial)
Enrollment : 90 participants
Allocation: Randomized
Masking: Double
Primary Purpose: Treatment
Study Start Date : January 1998
Study Completion Date : September 2000

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


--Disease Characteristics-- Facioscapulohumeral dystrophy Weakness of the facial muscles, including frontalis, orbicularis oculi, or orbicularis oris Weakness of scapular stabilizers or foot dorsiflexors Weakness of grade 2 or worse in the arm using the upper extremity grading scale No other neuromuscular diseases that may mimic the clinical presentation of facioscapulohumeral dystrophy: Ptosis or ophthalmoparesis (other than congenital strabismus) Elbow contractures Strictly unilateral weakness Dermatomyositis-like skin rash Symmetric distal sensory loss Muscle biopsy findings of mitochondrial myopathy, chronic denervation, dermatomyositis, inclusion body myositis, or congenital myopathy Electromyographic (EMG) findings of myotonia, fasciculations, or neurogenic motor unit potentials --Prior/Concurrent Therapy-- Endocrine therapy: No prior long term use of oral corticosteroids for more than 1 year At least 3 months since prior use of corticosteroids No concurrent use of immunosuppressive agents Surgery: No concurrent surgeries Other: No concurrent use of sympathomimetic agents, antidepressants, or beta receptor blockers --Patient Characteristics-- Age: 18 to 80 Performance status: Ambulatory Cardiovascular: No cardiovascular disease, including hypertension and coronary artery disease Other: Not pregnant or nursing No concurrent uncontrolled medical or psychological condition

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00004685

Sponsors and Collaborators
Ohio State University
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Study Chair: John T. Kissel Ohio State University

Layout table for additonal information Identifier: NCT00004685     History of Changes
Other Study ID Numbers: 199/13285
First Posted: February 25, 2000    Key Record Dates
Last Update Posted: March 25, 2015
Last Verified: March 1999
Keywords provided by FDA Office of Orphan Products Development:
genetic diseases and dysmorphic syndromes
muscular dystrophy
neurologic and psychiatric disorders
rare disease
Additional relevant MeSH terms:
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Muscular Dystrophy, Facioscapulohumeral
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Tocolytic Agents
Reproductive Control Agents
Adrenergic beta-2 Receptor Agonists
Adrenergic beta-Agonists
Adrenergic Agonists
Adrenergic Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action