Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy

Muscular Dystrophy, Facioscapulohumeral Clinical Trial

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00004685
• Other study ID #: 199/13285
• Secondary ID: OSURF-96H0022OSU
• Status: Completed
• Phase: N/A
• First received: February 24, 2000
• Last updated: March 24, 2015
• Start date: January 1998
• Est. completion date: September 2000

Study information

• Verified date: March 1999
• Source: FDA Office of Orphan Products Development
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Federal Government
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 90
• Est. completion date: September 2000
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 80 Years

Eligibility

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

Study Design

Allocation: Randomized, Masking: Double-Blind, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Facioscapulohumeral

Intervention

Drug:
• albuterol

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Ohio State University