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

Study 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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

sodium phenylbutyrate



Phase 1/Phase 2

Study Chair(s)/Principal Investigator(s):

Robert Ferrante, PhD MSc, Edith Nourse Rogers Memorial Veterans Hospital, Bedford ID:


Neals Affiliated?


Coordinating Center Contact Information

United States

Full Study Summary:

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).

Study Sponsor:

US Department of Veterans Affairs

Participant Duration:

20 weeks

Estimated Enrollment:


Estimated Study Start Date:

03 / 31 / 2005

Estimated Study Completion Date:

09 / 01 / 2007

Posting Last Modified Date:

01 / 11 / 2010

Date Study Added to

04 / 08 / 2005

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 ]

Minimum Age:

18 Years

Maximum Age:


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.

VA Medical Center, Iowa City

Iowa City, Iowa 52246-2208
United States

VA Medical Center, Lexington

Lexington, Kentucky 40502
United States

VA Maryland Health Care System, Baltimore

Baltimore, Maryland 21201
United States

Edith Nourse Rogers Memorial Veterans Hospital, Bedford

Bedford, Massachusetts 01730
United States

VA Medical Center, Jamaica Plain Campus

Boston, Massachusetts 02130
United States

VA Medical Center, Syracuse

Syracuse, New York 13210
United States

VA Medical Center, Durham

Durham, North Carolina 27705
United States

VA Medical Center, Cincinnati

Cincinnati, Ohio 45220
United States

VA Pittsburgh Health Care System

Pittsburgh, Pennsylvania 15240
United States

Michael E. DeBakey VA Medical Center (152)

Houston, Texas 77030
United States