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Ursodiol in Huntington's Disease (UDCA-HD)

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ClinicalTrials.gov Identifier: NCT00514774
Recruitment Status : Unknown
Verified February 2009 by Oregon Health and Science University.
Recruitment status was:  Active, not recruiting
First Posted : August 10, 2007
Last Update Posted : February 5, 2009
Information provided by:

Study Description
Brief Summary:
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 or disease Intervention/treatment Phase
Huntington Disease Drug: ursodiol Drug: placebo Phase 1

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:

  1. 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.
  2. To establish a preliminary safety and tolerability profile of the drug in subjects with HD.

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 21 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Ursodiol in Huntington's Disease
Study Start Date : August 2007
Estimated Primary Completion Date : June 2009
Estimated Study Completion Date : June 2009

Resource links provided by the National Library of Medicine

Drug Information available for: Ursodiol
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
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:
  • Ursodeoxycholic acid
  • UDCA
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:
  • Ursodeoxycholic acid
  • UDCA
Placebo Comparator: C Drug: placebo
placebo 600mg twice daily for study days 0 through 28

Outcome Measures

Primary Outcome Measures :
  1. Safety measures (complete blood count, chemistry profile, electrocardiogram, urinalysis) [ Time Frame: 35 days ]
  2. Tolerability measures (adverse event severity) [ Time Frame: 35 days ]
  3. Pharmacokinetic measures (Serum and CSF levels of bile acids) [ Time Frame: 28 days ]

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00514774

United States, Oregon
Oregon Health & Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
Oregon Health and Science University
Huntington Study Group
Huntington Society of Canada
Principal Investigator: Penelope Hogarth, M.D. Oregon Health and Science University
More Information

Additional Information:
Responsible Party: Penelope Hogarth, MD, Oregon Health & Science University
ClinicalTrials.gov Identifier: NCT00514774     History of Changes
Other Study ID Numbers: 00001927
First Posted: August 10, 2007    Key Record Dates
Last Update Posted: February 5, 2009
Last Verified: February 2009

Keywords provided by Oregon Health and Science University:
Huntington chorea
tauroursodeoxycholic acid
ursodeoxycholic acid

Additional relevant MeSH terms:
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Neurocognitive Disorders
Mental Disorders
Ursodeoxycholic Acid
Cholagogues and Choleretics
Gastrointestinal Agents