Ursodiol in Huntington's Disease (UDCA-HD)
Recruitment status was Active, not recruiting
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Purpose
The purpose of this study is to evaluate the safety of the drug ursodiol (ursodeoxycholic acid, UDCA) in people with Huntington's disease (HD) and to explore how the compound is processed by the body.
| Condition | Intervention | Phase |
|---|---|---|
|
Huntington Disease |
Drug: ursodiol Drug: placebo |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator) Primary Purpose: Treatment |
| Official Title: | Ursodiol in Huntington's Disease |
- Safety measures (complete blood count, chemistry profile, electrocardiogram, urinalysis) [ Time Frame: 35 days ] [ Designated as safety issue: Yes ]
- Tolerability measures (adverse event severity) [ Time Frame: 35 days ] [ Designated as safety issue: Yes ]
- Pharmacokinetic measures (Serum and CSF levels of bile acids) [ Time Frame: 28 days ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 21 |
| Study Start Date: | August 2007 |
| Estimated Study Completion Date: | June 2009 |
| Estimated Primary Completion Date: | June 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: A |
Drug: ursodiol
ursodiol 300 mg twice daily for study days 0 through 28 ursodiol 600mg twice daily on study days 0 through 28 Other Names:
|
| Experimental: B |
Drug: ursodiol
ursodiol 300 mg twice daily for study days 0 through 28 ursodiol 600mg twice daily on study days 0 through 28 Other Names:
|
| Placebo Comparator: C |
Drug: placebo
placebo 600mg twice daily for study days 0 through 28
|
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.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations| United States, Oregon | |
| Oregon Health & Science University | |
| Portland, Oregon, United States, 97239 | |
| Principal Investigator: | Penelope Hogarth, M.D. | Oregon Health and Science University |
More Information
Additional Information:
Publications:
| Responsible Party: | Penelope Hogarth, MD, Oregon Health & Science University |
| ClinicalTrials.gov Identifier: | NCT00514774 History of Changes |
| Other Study ID Numbers: | 00001927 |
| Study First Received: | August 8, 2007 |
| Last Updated: | February 4, 2009 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by Oregon Health and Science University:
|
Huntington chorea chorea hereditary tauroursodeoxycholic acid |
ursodeoxycholic acid TUDCA UDCA |
Additional relevant MeSH terms:
|
Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Dementia Chorea Dyskinesias Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn |
Cognition Disorders Delirium, Dementia, Amnestic, Cognitive Disorders Mental Disorders Ursodeoxycholic Acid Tauroursodeoxycholic acid Cholagogues and Choleretics Gastrointestinal Agents Therapeutic Uses Pharmacologic Actions Antiviral Agents Anti-Infective Agents |
ClinicalTrials.gov processed this record on May 21, 2013