Huntington DiseasePhase 1UDCA-HDNCT00514774
Ursodiol in Huntington's Disease
A Phase 1 study testing ursodiol in people with Huntington Disease, run by Oregon Health and Science University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Huntington Disease
- What is being tested?
- ursodiol
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 21 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2007to Jun 2009
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Oregon Health & Science University
Portland, Oregon, 97239
About this study
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.
Read the 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.
- Conditions
- Huntington Disease
- Being tested
- ursodiol, placebo
- Keywords
- Huntington chorea, chorea, hereditary, tauroursodeoxycholic acid, ursodeoxycholic acid, TUDCA, UDCA
- Medical subject headings
- Huntington Disease, Chorea
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ursodiolDrug
Also called Ursodeoxycholic acid, UDCA
ursodiol 300 mg twice daily for study days 0 through 28
ursodiol 600mg twice daily on study days 0 through 28
placeboDrug
placebo 600mg twice daily for study days 0 through 28
| Study group | Role | What they receive |
|---|---|---|
| A | Experimental | Not described Receives: Drug: ursodiol |
| B | Experimental | Not described Receives: Drug: ursodiol |
| C | Placebo comparator | Not described Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety measures (complete blood count, chemistry profile, electrocardiogram, urinalysis)
Measured: 35 days
Tolerability measures (adverse event severity)
Measured: 35 days
Pharmacokinetic measures (Serum and CSF levels of bile acids)
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2007 (estimated)
- Study startAug 2007
- Primary completionJun 2009 (estimated)
- Study completionJun 2009 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- More trials run by Oregon Health and Science UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Collaborators
- Huntington Study Group, Huntington Society of Canada
- Investigators
- Penelope Hogarth, M.D., Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00514774
- Sponsor's protocol ID
- 00001927
- Acronym
- UDCA-HD
- Status last verified
- Feb 2009
- First submitted
- 8 Aug 2007
- Last updated
- 5 Feb 2009
- Results posted
- No
- Data monitoring committee
- Yes
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.
PubMed 12149470Keene 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.
PubMed 11573988
Study at a glance
From the registry record, last updated by the sponsor on 5 Feb 2009. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Ursodiol in Huntington's Disease
- ClinicalTrials.gov ID
- NCT00514774
- Acronym / sponsor ID
- UDCA-HD / 00001927
- Conditions
- Huntington Disease
- Keywords
- Huntington chorea, chorea, hereditary, tauroursodeoxycholic acid, ursodeoxycholic acid, TUDCA, UDCA
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Collaborators
- Huntington Study Group, Huntington Society of Canada
- Study start
- Aug 2007
- Primary completion
- Jun 2009 (estimated)
- Study completion
- Jun 2009 (estimated)
- First posted
- 10 Aug 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| A | Experimental | Drug: ursodiol |
| B | Experimental | Drug: ursodiol |
| C | Placebo comparator | Drug: placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ursodiolAlso: Ursodeoxycholic acid, UDCAursodiol 300 mg twice daily for study days 0 through 28 ursodiol 600mg twice daily on study days 0 through 28 | Drug | A, B |
| placeboplacebo 600mg twice daily for study days 0 through 28 | Drug | C |
Primary outcome
Safety measures (complete blood count, chemistry profile, electrocardiogram, urinalysis)
Time frame: 35 days
Tolerability measures (adverse event severity)
Time frame: 35 days
Pharmacokinetic measures (Serum and CSF levels of bile acids)
Time frame: 28 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Feb 2009.
- Background: PubMed 12149470Keene 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.
- Background: PubMed 11573988Keene 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.
- Official website for the Huntington Study GroupLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Penelope Hogarth, M.D. principal investigator
Oregon Health and Science University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ursodiol in people with Huntington Disease, run by Oregon Health and Science University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Feb 2009. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety measures (complete blood count, chemistry profile, electrocardiogram, urinalysis) (time frame: 35 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00514774, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Feb 2009.