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NCT01529060 · ClinicalTrials.gov registry record · Phase 2
Phenylbutyrate Therapy for Maple Syrup Urine Disease
A Phase 2 study, sponsored by Brendan Lee.
- Completed
- Registry status
- Phase 2
- Development phase
- 20
- Enrollment target
NCT01529060 is a Phase 2 study that has completed, run by Brendan Lee. The registered enrollment target is 20 participants.
The verdict
NCT01529060, a Phase 2 study, has completed, sponsored by Brendan Lee.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 20 participants
- Enrollment target
Study Summary
The investigators have learned in past research that the drug phenylbutyrate can decrease the amounts of branched chain amino acids and their byproducts in the bloodstreams of healthy volunteer patients and also patients with certain disorders of protein breakdown including maple syrup urine disease. Through this study, the investigators will try to find out how well phenylbutyrate (NaPBA), also known by name brand "Buphenyl-TM", decreases BCAA and branched chain keto chain acids in the blood of patients with MSUD. The investigators hope is that through this research the investigators will be better able to treat these patients. Subjects with MSUD will take phenylbutyrate (NaPBA) in powder form for a two-week treatment period and powder placebo, a substance with no effect on the body, for a two-week treatment period. They will be given the same amount of powder and undergo the same laboratory testing during both of the two-week treatment periods. The results will be compared once the study is over.
Interventions
- DRUG Placebo powder
- DRUG Phenylbutyrate
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 20 participants |
| Start Date | 2013-02 |
| Est. Completion | 2017-12 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01529060
The ClinicalTrials.gov registry entry for NCT01529060 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 20 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Brendan Lee, which has 2 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 2 interventions - of which Placebo powder is the first listed.
NCT01529060 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01529060 about?
NCT01529060 is a clinical study titled "Phenylbutyrate Therapy for Maple Syrup Urine Disease". The investigators have learned in past research that the drug phenylbutyrate can decrease the amounts of branched chain amino acids and their byproducts in the bloodstreams of healthy volunteer patients and also patients with certain disorders of protein breakdown including maple syrup urine disease...
What is the current status of trial NCT01529060?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 20 participants. The study started on 2013-02. Estimated completion is 2017-12.
What interventions are being tested in trial NCT01529060?
The interventions under investigation include: Placebo powder (DRUG), Phenylbutyrate (DRUG).
Who is sponsoring clinical trial NCT01529060?
This trial is sponsored by Brendan Lee, which has 2 total clinical trials registered on ClinicalTrials.gov.
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