Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis) - Full Text View

Sponsor:	Department of Veterans Affairs
Collaborator:	Muscular Dystrophy Association
Information provided by:	Department of Veterans Affairs
ClinicalTrials.gov Identifier:	NCT00107770

Purpose

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 I Phase II

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety Study
Official Title:	Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis

Resource links provided by NLM:

Genetics Home Reference related topics: amyotrophic lateral sclerosis

MedlinePlus related topics: Amyotrophic Lateral Sclerosis

Drug Information available for: Sodium phenylbutyrate

U.S. FDA Resources

Further study details as provided by Department of Veterans Affairs:

Primary Outcome Measures:

safety and tolerability [ Time Frame: 20 weeks ] [ Designated as safety issue: No ]

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 [ Designated as safety issue: No ]
Relationship between blood levels and sodium phenylbutyrate dosage [ Designated as safety issue: No ]

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
1 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.

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).

Eligibility

Ages Eligible for Study:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00107770

Locations

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

Department of Veterans Affairs

Muscular Dystrophy Association

Investigators

Principal Investigator:

Robert Ferrante, PhD MSc

Edith Nourse Rogers Memorial Veterans Hospital, Bedford

More Information

No publications provided

Responsible Party:	Department of Veterans Affairs ( Ferrante, Robert - Principal Investigator )
Study ID Numbers:	0015
Study First Received:	April 7, 2005
Last Updated:	October 10, 2008
ClinicalTrials.gov Identifier:	NCT00107770 History of Changes
Health Authority:	United States: Federal Government; United States: Food and Drug Administration

Keywords provided by Department of Veterans Affairs:

ALS
histone deacetylase inhibition
open label
safety study
sodium phenylbutyrate

Additional relevant MeSH terms:

Spinal Cord Diseases
4-phenylbutyric acid
Antineoplastic Agents
Nervous System Diseases
Central Nervous System Diseases
Sclerosis
Neurodegenerative Diseases

Pharmacologic Actions
Pathologic Processes
Neuromuscular Diseases
Amyotrophic Lateral Sclerosis
Therapeutic Uses
Motor Neuron Disease

ClinicalTrials.gov processed this record on November 27, 2009