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
Disease:
Amyotrophic Lateral Sclerosis
Study Type:
Interventional
Type of Intervention:
Drug
Intervention Name:
sodium phenylbutyrate
Placebo:
Phase:
Phase 1/Phase 2
Study Chair(s)/Principal Investigator(s):
Robert Ferrante, PhD MSc, Edith Nourse Rogers Memorial Veterans Hospital, Bedford
Clinicaltrials.gov ID:
Neals Affiliated?
No
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.
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 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 Sponsor:
US Department of Veterans Affairs
Participant Duration:
20 weeks
Estimated Enrollment:
40
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 neals.org:
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 ]
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:
N/A
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