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

This study has been completed.
Muscular Dystrophy Association
Information provided by:
VA Office of Research and Development Identifier:
First received: April 7, 2005
Last updated: January 8, 2010
Last verified: October 2008
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.

Condition Intervention Phase
Amyotrophic Lateral Sclerosis Drug: sodium phenylbutyrate Phase 1 Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis

Resource links provided by NLM:

Further study details as provided by VA Office of Research and Development:

Primary Outcome Measures:
  • safety and tolerability [ Time Frame: 20 weeks ]

Secondary Outcome Measures:
  • 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

Enrollment: 40
Study Start Date: April 2005
Study Completion Date: September 2007
Primary Completion Date: November 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
ALS patient
Drug: sodium phenylbutyrate
histone deacteylase inhibitor

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.


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).


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00107770

United States, Iowa
VA Medical Center, Iowa City
Iowa City, Iowa, United States, 52246-2208
United States, Kentucky
VA Medical Center, Lexington
Lexington, Kentucky, United States, 40502
United States, Maryland
VA Maryland Health Care System, Baltimore
Baltimore, Maryland, United States, 21201
United States, Massachusetts
Edith Nourse Rogers Memorial Veterans Hospital, Bedford
Bedford, Massachusetts, United States, 01730
VA Medical Center, Jamaica Plain Campus
Boston, Massachusetts, United States, 02130
United States, New York
VA Medical Center, Syracuse
Syracuse, New York, United States, 13210
United States, North Carolina
VA Medical Center, Durham
Durham, North Carolina, United States, 27705
United States, Ohio
VA Medical Center, Cincinnati
Cincinnati, Ohio, United States, 45220
United States, Pennsylvania
VA Pittsburgh Health Care System
Pittsburgh, Pennsylvania, United States, 15240
United States, Texas
Michael E. DeBakey VA Medical Center (152)
Houston, Texas, United States, 77030
Sponsors and Collaborators
VA Office of Research and Development
Muscular Dystrophy Association
Principal Investigator: Robert Ferrante, PhD MSc Edith Nourse Rogers Memorial Veterans Hospital, Bedford
  More Information

Responsible Party: Ferrante, Robert - Principal Investigator, Department of Veterans Affairs Identifier: NCT00107770     History of Changes
Other Study ID Numbers: 0015
Study First Received: April 7, 2005
Last Updated: January 8, 2010

Keywords provided by VA Office of Research and Development:
histone deacetylase inhibition
open label
safety study
sodium phenylbutyrate

Additional relevant MeSH terms:
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
4-phenylbutyric acid
Antineoplastic Agents processed this record on September 25, 2017