Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy

NCT00004685 | Completed

• 3 other identifiers: 199/13285OSURF-96H0022OSURF-FDR001293
• Study Type: interventional
• Target: 90
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Muscular Dystrophy, Facioscapulohumeral

Keywords

genetic diseases and dysmorphic syndromes; muscular dystrophy; neurologic and psychiatric disorders; rare disease

Interventions

albuterol DRUG

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Ohio State University: lead

MeSH Terms

Conditions

Muscular Dystrophy, Facioscapulohumeral; Genetic Diseases, Inborn; Muscular Dystrophies; Neurologic Manifestations; Mental Disorders; Rare Diseases

Interventions

Albuterol

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Disease Attributes; Pathologic Processes

Intervention Hierarchy (Ancestors)

Ethanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Amines; Phenethylamines; Ethylamines

Study Officials

• John T. Kissel

  Ohio State University

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: February 24, 2000
• First Posted: February 25, 2000
• Study Start: January 1, 1998
• Study Completion: September 1, 2000
• Last Updated: March 25, 2015

Record last verified: 1999-03