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

NCT ID: NCT00107770

Last Updated: 2010-01-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-04-30

Study Completion Date

2007-09-30

Brief Summary

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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

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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).

Conditions

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Amyotrophic Lateral Sclerosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

ALS patient

Group Type OTHER

sodium phenylbutyrate

Intervention Type DRUG

histone deacteylase inhibitor

Interventions

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sodium phenylbutyrate

histone deacteylase inhibitor

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Muscular Dystrophy Association

OTHER

Sponsor Role collaborator

US Department of Veterans Affairs

FED

Sponsor Role lead

Responsible Party

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Department of Veterans Affairs

Principal Investigators

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Robert Ferrante, PhD MSc

Role: PRINCIPAL_INVESTIGATOR

Edith Nourse Rogers Memorial Veterans Hospital, Bedford

Locations

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VA Medical Center, Iowa City

Iowa City, Iowa, United States

Site Status

VA Medical Center, Lexington

Lexington, Kentucky, United States

Site Status

VA Maryland Health Care System, Baltimore

Baltimore, Maryland, United States

Site Status

Edith Nourse Rogers Memorial Veterans Hospital, Bedford

Bedford, Massachusetts, United States

Site Status

VA Medical Center, Jamaica Plain Campus

Boston, Massachusetts, United States

Site Status

VA Medical Center, Syracuse

Syracuse, New York, United States

Site Status

VA Medical Center, Durham

Durham, North Carolina, United States

Site Status

VA Medical Center, Cincinnati

Cincinnati, Ohio, United States

Site Status

VA Pittsburgh Health Care System

Pittsburgh, Pennsylvania, United States

Site Status

Michael E. DeBakey VA Medical Center (152)

Houston, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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0015

Identifier Type: -

Identifier Source: org_study_id

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