Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy

This study has been completed.
Information provided by:
FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:
First received: February 24, 2000
Last updated: March 24, 2015
Last verified: March 1999

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 Intervention
Muscular Dystrophy, Facioscapulohumeral
Drug: albuterol

Study Type: Interventional
Study Design: Allocation: Randomized
Masking: Double-Blind
Primary Purpose: Treatment

Resource links provided by NLM:

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

Estimated Enrollment: 90
Study Start Date: January 1998
Estimated Study Completion Date: September 2000
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


Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
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

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Please refer to this study by its ClinicalTrials.gov identifier: NCT00004685

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

ClinicalTrials.gov Identifier: NCT00004685     History of Changes
Other Study ID Numbers: 199/13285  OSURF-96H0022  OSURF-FDR001293 
Study First Received: February 24, 2000
Last Updated: March 24, 2015
Health Authority: United States: Federal Government

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:
Muscular Dystrophy, Facioscapulohumeral
Muscular Dystrophies
Genetic Diseases, Inborn
Muscular Diseases
Muscular Disorders, Atrophic
Musculoskeletal Diseases
Nervous System Diseases
Neuromuscular Diseases
Adrenergic Agents
Adrenergic Agonists
Adrenergic beta-2 Receptor Agonists
Adrenergic beta-Agonists
Anti-Asthmatic Agents
Autonomic Agents
Bronchodilator Agents
Molecular Mechanisms of Pharmacological Action
Neurotransmitter Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Reproductive Control Agents
Respiratory System Agents
Tocolytic Agents

ClinicalTrials.gov processed this record on May 26, 2016