A Phase 1/2 interventional study of sodium phenylbutyrate in Amyotrophic Lateral Sclerosis, sponsored by US Department of Veterans Affairs. Completed at 10 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2010-01-11.
Sponsored by US Department of Veterans Affairs · Phase 1/2, Interventional, and Treatment
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.
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).
717 studies on the registry are indexed under Motor Neuron Disease; 137 are open to participants now.
This study's enrollment of 40 is above the median of 35 across 461 interventional studies indexed under Motor Neuron Disease.
Browse Motor Neuron Disease studies →US Department of Veterans Affairs is the lead sponsor of 658 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Veterans only are eligible to participate at VA sites.
ALS patient
Drug: sodium phenylbutyrate
histone deacteylase inhibitor
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
This study is completed, as verified in Oct 2008. You cannot join it, but the record below documents what was studied.
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US Department of Veterans Affairs