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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00107770
Other study ID # 0015
Secondary ID
Status Completed
Phase Phase 1/Phase 2
First received April 7, 2005
Last updated January 8, 2010
Start date April 2005
Est. completion date September 2007

Study information

Verified date October 2008
Source VA Office of Research and Development
Contact n/a
Is FDA regulated No
Health authority United States: Federal GovernmentUnited States: Food and Drug Administration
Study type Interventional

Clinical Trial Summary

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.


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).


Recruitment information / eligibility

Status Completed
Enrollment 40
Est. completion date September 2007
Est. primary completion date November 2006
Accepts healthy volunteers No
Gender Both
Age group 18 Years and older
Eligibility 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.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Intervention

Drug:
sodium phenylbutyrate
histone deacteylase inhibitor

Locations

Country Name City State
United States VA Maryland Health Care System, Baltimore Baltimore Maryland
United States Edith Nourse Rogers Memorial Veterans Hospital, Bedford Bedford Massachusetts
United States VA Medical Center, Jamaica Plain Campus Boston Massachusetts
United States VA Medical Center, Cincinnati Cincinnati Ohio
United States VA Medical Center, Durham Durham North Carolina
United States Michael E. DeBakey VA Medical Center (152) Houston Texas
United States VA Medical Center, Iowa City Iowa City Iowa
United States VA Medical Center, Lexington Lexington Kentucky
United States VA Pittsburgh Health Care System Pittsburgh Pennsylvania
United States VA Medical Center, Syracuse Syracuse New York

Sponsors (2)

Lead Sponsor Collaborator
VA Office of Research and Development Muscular Dystrophy Association

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary safety and tolerability 20 weeks No
Secondary 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 No
Secondary Relationship between blood levels and sodium phenylbutyrate dosage No
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