Ursodiol in Huntington's Disease (UDCA-HD)

The recruitment status of this study is unknown because the information has not been verified recently.
Verified February 2009 by Oregon Health and Science University.
Recruitment status was  Active, not recruiting
Sponsor:
Collaborators:
Huntington Study Group
Huntington Society of Canada
Information provided by:
Oregon Health and Science University
ClinicalTrials.gov Identifier:
NCT00514774
First received: August 8, 2007
Last updated: February 4, 2009
Last verified: February 2009
  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

Resource links provided by NLM:


Further study details as provided by Oregon Health and Science University:

Primary Outcome Measures:
  • 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:
  • 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

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.
  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00514774

Locations
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
Investigators
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:
Chorea
Huntington Disease
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Dementia
Dyskinesias
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn
Cognition Disorders
Delirium, Dementia, Amnestic, Cognitive Disorders
Mental Disorders
Ursodeoxycholic Acid
Cholagogues and Choleretics
Gastrointestinal Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 30, 2014