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

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 1 Phase 2


Study Type:	Interventional
Study Design:	Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: 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:

Genetics Home Reference related topics: amyotrophic lateral sclerosis

MedlinePlus related topics: Amyotrophic Lateral Sclerosis

Drug Information available for: Sodium phenylbutyrate

Genetic and Rare Diseases Information Center resources: Amyotrophic Lateral Sclerosis

U.S. FDA Resources

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

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 (Adult, Senior)
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

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 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

VA Office of Research and Development

Muscular Dystrophy Association

Investigators


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
ClinicalTrials.gov Identifier:	NCT00107770 History of Changes
Other Study ID Numbers:	0015
Study First Received:	April 7, 2005
Last Updated:	January 8, 2010
Health Authority:	United States: Federal Government United States: Food and Drug Administration

Keywords provided by VA Office of Research and Development:


ALS histone deacetylase inhibition open label safety study sodium phenylbutyrate

Additional relevant MeSH terms:


Sclerosis 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

ClinicalTrials.gov processed this record on September 30, 2016