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Sponsors and Collaborators: |
Department of Veterans Affairs Muscular Dystrophy Association |
Information provided by: | Department of Veterans Affairs |
ClinicalTrials.gov Identifier: | NCT00107770 |
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 |
Genetics Home Reference related topics: | amyotrophic lateral sclerosis |
MedlinePlus related topics: | Amyotrophic Lateral Sclerosis |
ChemIDplus related topics: | Sodium phenylbutyrate |
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 |
Estimated Enrollment: | 40 |
Study Start Date: | April 2005 |
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).
Ages Eligible for Study: | 18 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Veterans only are eligible to participate at VA sites.
United States, Iowa | |||||
Veteran’s Administration Hospital (Iowa City) | |||||
Iowa City, Iowa, United States, 52242-1009 | |||||
United States, Kentucky | |||||
Lexington VA Medical Center | |||||
Lexington, Kentucky, United States, 40502 | |||||
United States, Maryland | |||||
Johns Hopkins University | |||||
Baltimore, Maryland, United States, 21287 | |||||
United States, Massachusetts | |||||
Edith Nourse Rogers Memorial Veterans Hospital | |||||
Bedford, Massachusetts, United States, 01730 | |||||
Massachusetts General Hospital | |||||
Boston, Massachusetts, United States, 02129 | |||||
United States, New York | |||||
Syracuse VA Medical Center | |||||
Syracuse, New York, United States, 13210 | |||||
United States, North Carolina | |||||
Durham VA Medical Center | |||||
Durham, North Carolina, United States, 27705 | |||||
United States, Ohio | |||||
Cincinnati VAMC | |||||
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 | |||||
Houston, Texas, United States, 77030 |
Department of Veterans Affairs |
Muscular Dystrophy Association |
Principal Investigator: | Merit E. Cudkowicz, MD, MSc | Massachusetts General Hospital and Edith Nourse Rogers Memorial Veterans Hospital |
Principal Investigator: | Robert J. Ferrante, PhD | Edith Nourse Rogers Memorial Veterans Hospital |
Principal Investigator: | Robert H. Brown, Jr., MD, DPhil. | Massachusetts General Hospital |
Study ID Numbers: | 0015 |
First Received: | April 7, 2005 |
Last Updated: | June 27, 2006 |
ClinicalTrials.gov Identifier: | NCT00107770 |
Health Authority: | United States: Food and Drug Administration |
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