Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)
Tracking Information | |||||
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First Received Date ICMJE | April 7, 2005 | ||||
Last Updated Date | January 8, 2010 | ||||
Start Date ICMJE | April 2005 | ||||
Primary Completion Date | November 2006 (final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
safety and tolerability [ Time Frame: 20 weeks ] [ Designated as safety issue: No ] | ||||
Original Primary Outcome Measures ICMJE |
The ability to complete the dosage increase and maintenance phase at 21 grams per day | ||||
Change History | Complete list of historical versions of study NCT00107770 on ClinicalTrials.gov Archive Site | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Outcome Measures ICMJE | |||||
Original Other Outcome Measures ICMJE | |||||
Descriptive Information | |||||
Brief Title ICMJE | Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis) | ||||
Official Title ICMJE | Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis | ||||
Brief 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. |
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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). |
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Study Type ICMJE | Interventional | ||||
Study Phase | Phase 1 Phase 2 |
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Study Design ICMJE | Allocation: Non-Randomized Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
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Condition ICMJE | Amyotrophic Lateral Sclerosis | ||||
Intervention ICMJE | Drug: sodium phenylbutyrate
histone deacteylase inhibitor |
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Study Arm (s) | 1
ALS patient
Intervention: Drug: sodium phenylbutyrate |
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Publications * | |||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Enrollment ICMJE | 40 | ||||
Completion Date | September 2007 | ||||
Primary Completion Date | November 2006 (final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
Veterans only are eligible to participate at VA sites. |
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Gender | Both | ||||
Ages | 18 Years and older | ||||
Accepts Healthy Volunteers | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Location Countries ICMJE | United States | ||||
Administrative Information | |||||
NCT Number ICMJE | NCT00107770 | ||||
Other Study ID Numbers ICMJE | 0015 | ||||
Has Data Monitoring Committee | No | ||||
Responsible Party | Ferrante, Robert - Principal Investigator, Department of Veterans Affairs | ||||
Study Sponsor ICMJE | Department of Veterans Affairs | ||||
Collaborators ICMJE | Muscular Dystrophy Association | ||||
Investigators ICMJE |
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Information Provided By | Department of Veterans Affairs | ||||
Verification Date | October 2008 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |