Study of Albuterol and Oxandrolone in Patients With Facioscapulohumeral Dystrophy (FSHD)

NCT00027391 | Completed

• 2 other identifiers: FD-R-2029-01FD-R-002029-01
• Study Type: interventional
• Target: 160
• Locations: 1 country
• Sites: 1

Brief Summary

This is a study to determine whether albuterol or oxandrolone, alone or in combination, are able to increase strength and muscle mass in patients with FSHD. It also will determine if albuterol given in "pulsed" fashion will have more effect than when given continuously.

Conditions

Muscular Dystrophies

Keywords

Muscle Weakness, Muscular Dystrophy

Interventions

Albuterol DRUG

Oxandrolone DRUG

Eligibility Criteria

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

You may qualify if:

• Presence of 4q35 "small fragment" of less than 40 kb by standard DNA testing
• Weakness of the facial muscles, including frontalis, orbicularis oculi, or orbicularis oris
• Weakness of scapular stabilizers or foot dorsiflexors
• Ambulatory
• Weakness grade 2 or worse in the arm using upper extremity grading scale

You may not qualify if:

• Prior use of oral beta-2 agonists for a period of at least 1 year or within the past 3 months
• Concurrent use of other sympathomimetic agents, antidepressants, or beta-2 receptor blockers
• Pregnancy
• Known hypersensitivity to anabolic steroids
• Any medical or psychological condition that would interfere with the study
• Requirement for a wheelchair

Sponsors & Collaborators

• FDA Office of Orphan Products Development: lead

Study Sites (1)

Ohio State University Medical Center

Columbus, Ohio, 43210, United States

Location

MeSH Terms

Conditions

Muscular Dystrophies; Muscle Weakness

Interventions

Albuterol; Oxandrolone

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Neuromuscular Manifestations; Neurologic Manifestations; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Ethanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Amines; Phenethylamines; Ethylamines; Androstanols; Androstanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• John T. Kissel, M.D.

  Ohio State University

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: December 5, 2001
• First Posted: December 7, 2001
• Study Start: September 1, 2001
• Study Completion: August 1, 2004
• Last Updated: March 25, 2015

Record last verified: 2001-11

Locations

US (1)