Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)
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Purpose
| Condition | Intervention | Phase |
|---|---|---|
| Amyotrophic Lateral Sclerosis | Drug: sodium phenylbutyrate | Phase 1 Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
| Official Title: | Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis |
- safety and tolerability [ Time Frame: 20 weeks ]
- 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
- Relationship between blood levels and sodium phenylbutyrate dosage
| 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
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, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00107770
| 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 | |
| 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 |
| First Submitted: | April 7, 2005 |
| First Posted: | April 8, 2005 |
| Last Update Posted: | January 11, 2010 |
| Last Verified: | October 2008 |
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 |