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

January 8, 2010 updated by: US Department of Veterans Affairs

Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

40

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Iowa
      • Iowa City, Iowa, United States, 52246-2208
        • VA Medical Center, Iowa City
    • Kentucky
      • Lexington, Kentucky, United States, 40502
        • VA Medical Center, Lexington
    • Maryland
      • Baltimore, Maryland, United States, 21201
        • VA Maryland Health Care System, Baltimore
    • Massachusetts
      • Bedford, Massachusetts, United States, 01730
        • Edith Nourse Rogers Memorial Veterans Hospital, Bedford
      • Boston, Massachusetts, United States, 02130
        • VA Medical Center, Jamaica Plain Campus
    • New York
      • Syracuse, New York, United States, 13210
        • VA Medical Center, Syracuse
    • North Carolina
      • Durham, North Carolina, United States, 27705
        • VA Medical Center, Durham
    • Ohio
      • Cincinnati, Ohio, United States, 45220
        • VA Medical Center, Cincinnati
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15240
        • VA Pittsburgh Health Care System
    • Texas
      • Houston, Texas, United States, 77030
        • Michael E. DeBakey VA Medical Center (152)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: 1
ALS patient
Drug: sodium phenylbutyrate
histone deacteylase inhibitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
safety and tolerability
Time Frame: 20 weeks
20 weeks

Secondary Outcome Measures

Outcome Measure
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

US Department of Veterans Affairs

Collaborators

Muscular Dystrophy Association

Investigators

  • Principal Investigator: Robert Ferrante, PhD MSc, Edith Nourse Rogers Memorial Veterans Hospital, Bedford

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2005

Primary Completion (Actual)

November 1, 2006

Study Completion (Actual)

September 1, 2007

Study Registration Dates

First Submitted

April 7, 2005

First Submitted That Met QC Criteria

April 7, 2005

First Posted (Estimate)

April 8, 2005

Study Record Updates

Last Update Posted (Estimate)

January 11, 2010

Last Update Submitted That Met QC Criteria

January 8, 2010

Last Verified

October 1, 2008

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 0015

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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