Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy

Study Overview
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Brief Summary
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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.

Detailed Description
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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

Conditions
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Muscular Dystrophy, Facioscapulohumeral

Intervention / Treatment
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* Drug: albuterol


Participation Criteria
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Eligibility Criteria
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PROTOCOL ENTRY CRITERIA: --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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 80 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Masking: Double


Arms and Interventions

| Intervention/Treatment |
| --- |
|Drug: albuterol|nan|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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genetic diseases and dysmorphic syndromes, muscular dystrophy, neurologic and psychiatric disorders, rare disease