Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)

Phase 1

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: sodium phenylbutyrate

• Registration Number: NCT00107770
• Lead Sponsor: US Department of Veterans Affairs

Brief Summary

The purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this medication without major side effects.

Detailed Description

Although it is known that nerve cells die in the brains and spinal cords of patients who have ALS, the cause of the cell death is unknown. There is evidence that this cell death may be caused by changes in DNA, the body's genetic material. Drugs such as sodium phenylbutyrate (NaPB) can increase the expression of genes, block how the motor nerve cells in ALS die, and may prove to be an effective therapy for ALS. NaPB has shown an improvement in survival in mice with conditions similar to ALS.

STUDY DESIGN:

All research participants will take sodium phenylbutyrate for a total of 20 weeks. The dose of medication will be increased every 2 to 4 weeks until a maximum, easily tolerated dose is achieved (study maximum is 21 g/day).

Study Timeline

• Study Start: 2005-04
• Study First Submitted: 2005-04-07
• Study First Submitted QC: 2005-04-07
• Study First Posted: 2005-04-08
• Primary Completion: 2006-11
• Study Completion: 2007-09
• Status Verified: 2008-10
• Last Update Submitted: 2010-01-08
• Last Update Posted: 2010-01-11

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 40

Inclusion Criteria

• Diagnosed with ALS
• At least 18 years of age
• Women, who can become pregnant, must actively use effective birth control measures

Exclusion Criteria

• Must not have any other neurological (nervous system) disease

Veterans only are eligible to participate at VA sites.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
1	sodium phenylbutyrate	ALS patient

Primary Outcome Measures

Name	Time	Method
safety and tolerability	20 weeks

Secondary Outcome Measures

Name	Time	Method
The number of side effects at each dosage, including abnormalities in vital signs, physical examination, blood tests and EKGs, change in vital capacity (breathing function) and ALS functional rating scale
Relationship between blood levels and sodium phenylbutyrate dosage

Trial Locations

Locations (10)

VA Medical Center, Iowa City; 🇺🇸 Iowa City, Iowa, United States

VA Medical Center, Lexington; 🇺🇸 Lexington, Kentucky, United States

VA Maryland Health Care System, Baltimore; 🇺🇸 Baltimore, Maryland, United States

Edith Nourse Rogers Memorial Veterans Hospital, Bedford; 🇺🇸 Bedford, Massachusetts, United States

VA Medical Center, Jamaica Plain Campus; 🇺🇸 Boston, Massachusetts, United States

VA Medical Center, Syracuse; 🇺🇸 Syracuse, New York, United States

VA Medical Center, Durham; 🇺🇸 Durham, North Carolina, United States

VA Medical Center, Cincinnati; 🇺🇸 Cincinnati, Ohio, United States

VA Pittsburgh Health Care System; 🇺🇸 Pittsburgh, Pennsylvania, United States

Michael E. DeBakey VA Medical Center (152); 🇺🇸 Houston, Texas, United States