- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00514774
Ursodiol in Huntington's Disease (UDCA-HD)
Study Overview
Detailed Description
Huntington's disease is an inherited neurodegenerative disease that causes a movement disorder, dementia, and psychiatric and behavioral disturbance in affected individuals.
Tauroursodeoxycholic acid (TUDCA) is a bile acid synthesized in the liver by the conjugation of taurine to ursodeoxycholic acid (UDCA). It is thought to function as an anti-apoptotic agent in HD, evidenced by studies in toxic cell models and both toxic and transgenic rodent models of the disease.
Ursodiol is a commercially-available exogenous form of UDCA, the precursor of TUDCA. Although the compound has an established dosing, safety, tolerability and efficacy profile in patients with hepatobiliary disorders, gaps exist in the understanding of the pharmacokinetics / pharmacodynamics of the compound, particularly in patients with normal gastrointestinal function, and no human data exist for its therapeutic use in neurodegenerative disorders. The specific aims of this study are:
- To establish whether treatment with the drug ursodiol will result in measurable levels of its bile acid metabolites in serum and CSF at standard oral doses; and whether a dose-response can be detected using these measures.
- To establish a preliminary safety and tolerability profile of the drug in subjects with HD.
Study Type
Enrollment (Anticipated)
Phase
- Phase 1
Contacts and Locations
Study Locations
-
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Oregon
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Portland, Oregon, United States, 97239
- Oregon Health & Science University
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-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- All subjects will be age 18 or older
- All subjects will have manifest Huntington disease determined by clinical exam plus either documented prior DNA testing for the HD gene or a documented family history of the disease
Exclusion Criteria:
- Subjects taking oral contraceptives, cholestyramine, colestipol, or aluminum-based antacids will be excluded
- Subjects with known allergy or other contraindication to the study drug will be excluded
- Subjects with bleeding diathesis, or on coumadin or mandatory aspirin will be excluded
- Subjects with unstable medical or psychiatric illness will be excluded
- Subjects with clinically significant lab / EKG abnormalities at screening will be excluded
- Subjects who are currently pregnant or breastfeeding will be excluded
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Double
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: C
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placebo 600mg twice daily for study days 0 through 28
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Experimental: A
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ursodiol 300 mg twice daily for study days 0 through 28 ursodiol 600mg twice daily on study days 0 through 28
Other Names:
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Experimental: B
|
ursodiol 300 mg twice daily for study days 0 through 28 ursodiol 600mg twice daily on study days 0 through 28
Other Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Safety measures (complete blood count, chemistry profile, electrocardiogram, urinalysis)
Time Frame: 35 days
|
35 days
|
Tolerability measures (adverse event severity)
Time Frame: 35 days
|
35 days
|
Pharmacokinetic measures (Serum and CSF levels of bile acids)
Time Frame: 28 days
|
28 days
|
Collaborators and Investigators
Investigators
- Principal Investigator: Penelope Hogarth, M.D., Oregon Health and Science University
Publications and helpful links
General Publications
- Keene CD, Rodrigues CM, Eich T, Chhabra MS, Steer CJ, Low WC. Tauroursodeoxycholic acid, a bile acid, is neuroprotective in a transgenic animal model of Huntington's disease. Proc Natl Acad Sci U S A. 2002 Aug 6;99(16):10671-6. doi: 10.1073/pnas.162362299. Epub 2002 Jul 29.
- Keene CD, Rodrigues CM, Eich T, Linehan-Stieers C, Abt A, Kren BT, Steer CJ, Low WC. A bile acid protects against motor and cognitive deficits and reduces striatal degeneration in the 3-nitropropionic acid model of Huntington's disease. Exp Neurol. 2001 Oct;171(2):351-60. doi: 10.1006/exnr.2001.7755.
Helpful Links
Study record dates
Study Major Dates
Study Start
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Mental Disorders
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Neurocognitive Disorders
- Genetic Diseases, Inborn
- Basal Ganglia Diseases
- Movement Disorders
- Neurodegenerative Diseases
- Dyskinesias
- Heredodegenerative Disorders, Nervous System
- Dementia
- Cognition Disorders
- Chorea
- Huntington Disease
- Gastrointestinal Agents
- Cholagogues and Choleretics
- Ursodeoxycholic Acid
Other Study ID Numbers
- 00001927
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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