Ignite Creation Date:
2024-05-05 @ 12:01 PM
Last Modification Date:
2024-10-26 @ 9:18 AM
Study NCT ID:
NCT00212316
Status:
COMPLETED
Last Update Posted:
2012-08-15
First Post:
2005-09-19
Brief Title:
Safety and Tolerability Study of Phenylbutyrate in Huntingtons Disease PHEND-HD
Sponsor:
University of Rochester
Organization:
University of Rochester
Study Overview
Official Title:
Phenylbutyrate Development for Huntingtons Disease PHEND-HD A Multi-Center Double-Blind Placebo-Controlled Study With Open-Label Follow-Up to Determine the Safety and Tolerability of Phenylbutyrate in Subjects With Huntingtons Disease
Status:
COMPLETED
Status Verified Date:
2007-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to evaluate the safety tolerability and clinical impact of 15-grams daily of sodium phenylbutyrate phenylbutyrate in Huntingtons disease and to lay the groundwork for possible subsequent trials designed to specifically address its ability to slow or halt the progression of the disease
Detailed Description:
Huntingtons disease HD is an autosomal dominant disorder resulting in selective loss of neurons in the striatum-an area of the brain that controls movement balance and walking-and other areas of the brain The disease is characterized by progressive motor and cognitive decline There is no cure or even plausible treatment to offset the fatal course of the disease Therefore any treatment that ameliorates the disease would be of enormous importance
The purpose of this double-blind placebo-controlled study-with open-label follow-up-is to determine the safety and tolerability of 15-grams daily of oral phenylbutyrate in people with HD The study will enroll 60 individuals Eligible participants will be initially randomized to receive either phenylbutyrate or the matching placebo for 4 weeks
After the placebo-controlled phase all participants will enter the open-label phase to receive phenylbutyrate for 12 weeks Participants will be followed for one month off phenylbutyrate
This combination of a short-term double-blind placebo-controlled phase followed by a longer open-label phase will favor the primary goals of detecting toxicity and intolerability while facilitating recruitment and maximizing number of subjects on study drug
The purpose of this double-blind placebo-controlled study-with open-label follow-up-is to determine the safety and tolerability of 15-grams daily of oral phenylbutyrate in people with HD The study will enroll 60 individuals Eligible participants will be initially randomized to receive either phenylbutyrate or the matching placebo for 4 weeks
After the placebo-controlled phase all participants will enter the open-label phase to receive phenylbutyrate for 12 weeks Participants will be followed for one month off phenylbutyrate
This combination of a short-term double-blind placebo-controlled phase followed by a longer open-label phase will favor the primary goals of detecting toxicity and intolerability while facilitating recruitment and maximizing number of subjects on study drug
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None