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| Sponsor: | FDA Office of Orphan Products Development |
|---|---|
| Collaborator: |
Ohio State University |
| Information provided by: | FDA Office of Orphan Products Development |
| ClinicalTrials.gov Identifier: | NCT00004685 |
Purpose
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: | Treatment, Randomized, Double-Blind, Placebo Control |
| Estimated Enrollment: | 90 |
| Study Start Date: | January 1998 |
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.
Eligibility| Ages Eligible for Study: | 18 Years to 80 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
More Information
| Study ID Numbers: | 199/13285, OSURF-96H0022, OSURF-FDR001293 |
| Study First Received: | February 24, 2000 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00004685 History of Changes |
| Health Authority: | United States: Federal Government |
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genetic diseases and dysmorphic syndromes muscular dystrophy neurologic and psychiatric disorders rare disease |
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Respiratory System Agents Neurotransmitter Agents Molecular Mechanisms of Pharmacological Action Adrenergic Agents Adrenergic beta-Agonists Nervous System Diseases Albuterol Physiological Effects of Drugs Anti-Asthmatic Agents Reproductive Control Agents Adrenergic Agonists Pharmacologic Actions |
Muscular Dystrophy, Facioscapulohumeral Muscular Dystrophies Muscular Diseases Tocolytic Agents Muscular Disorders, Atrophic Musculoskeletal Diseases Neuromuscular Diseases Genetic Diseases, Inborn Autonomic Agents Therapeutic Uses Peripheral Nervous System Agents Bronchodilator Agents |