Infantile Refsum's DiseaseNCT00004442
Study of Bile Acids in Patients With Peroxisomal Disorders
A study testing chenodeoxycholic acid, cholic acid and ursodiol in people with Infantile Refsum's Disease and Zellweger Syndrome, run by University of Cincinnati.
Trial summary
OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid, chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders involving impaired primary bile acid synthesis. II. Read more about this study
- Who is it for?
- Ages Up to 5 years
- Conditions the study is for
- Infantile Refsum's Disease, Zellweger Syndrome, Bifunctional Enzyme Deficiency
- What is being tested?
- chenodeoxycholic acid, cholic acid and ursodiol
- How many people
- 25 participants
- Where
- No sites listed yet
- Study dates
- Start not givento Apr 1999
- Phase
- Interventional
- 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 Up to 5 years
- All sexes
- No healthy volunteers
- Child
Biochemically proven peroxisomal disorder, including:
- Zellweger syndrome
- Pseudo-Zellweger syndrome
- Neonatal adrenoleukodystrophy
- Bifunctional enzyme deficiency
- Infantile Refsum's disease
About this study
OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid, chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders involving impaired primary bile acid synthesis.
II. Determine whether suppression of synthesis of atypical bile acids and enrichment of bile acid pool with this regimen is effective in treating this patient population and improving quality of life.
Read the detailed description
PROTOCOL OUTLINE: Patients receive oral cholic acid and oral chenodeoxycholic acid on day 1. On day 4, patients receive oral cholic and ursodeoxycholic acids. Patients are assessed at 3 and 6 months for liver function response, neurologic status, and nutritional status.
Patients receive treatment until disease progression or unacceptable toxic effects are observed.
Completion date provided represents the completion date of the grant per OOPD records
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Bifunctional Enzyme Deficiency, Adrenoleukodystrophy
- Being tested
- chenodeoxycholic acid, cholic acid, ursodiol
- Keywords
- Zellweger syndrome, adrenoleukodystrophy, bifunctional enzyme deficiency, inborn errors of metabolism, infantile Refsum's disease, peroxisomal disorders, pseudo-Zellweger syndrome, rare disease, sphingolipidoses
- Medical subject headings
- Refsum Disease, Infantile, Zellweger Syndrome, Pseudo-Zellweger syndrome, Adrenoleukodystrophy, Metabolism, Inborn Errors, Peroxisomal Disorders, Rare Diseases, Sphingolipidoses
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
chenodeoxycholic acidDrug
cholic acidDrug
ursodiolDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Oct 1999 (estimated)
- Study completionApr 1999
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile Refsum's DiseaseStudies of the same condition, open and under way.
- More trials run by University of CincinnatiThe 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 N/A
- Primary purpose
- Treatment
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Hospital Medical Center, Cincinnati
- Investigators
- Kenneth Setchell, Study chair, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00004442
- Sponsor's protocol ID
- 199/13442
- Other IDs
- CHMC-C-FDR000995
- Status last verified
- Nov 2000
- First submitted
- 18 Oct 1999
- Last updated
- 25 Mar 2015
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Bile Acids in Patients With Peroxisomal Disorders
- ClinicalTrials.gov ID
- NCT00004442
- Acronym / sponsor ID
- 199/13442
- Conditions
- Infantile Refsum's Disease, Zellweger Syndrome, Bifunctional Enzyme Deficiency, Adrenoleukodystrophy
- Keywords
- Zellweger syndrome, adrenoleukodystrophy, bifunctional enzyme deficiency, inborn errors of metabolism, infantile Refsum's disease, peroxisomal disorders, pseudo-Zellweger syndrome, rare disease
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- University of CincinnatiUniversity, hospital or other
- Collaborators
- Children's Hospital Medical Center, Cincinnati
- Study completion
- Apr 1999
- First posted
- 19 Oct 1999 (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.
OBJECTIVES: I. Determine the effectiveness of oral bile acid therapy with cholic acid, chenodeoxycholic acid, and ursodeoxycholic acid in patients with peroxisomal disorders involving impaired primary bile acid synthesis.
II. Determine whether suppression of synthesis of atypical bile acids and enrichment of bile acid pool with this regimen is effective in treating this patient population and improving quality of life.
Detailed description
PROTOCOL OUTLINE: Patients receive oral cholic acid and oral chenodeoxycholic acid on day 1. On day 4, patients receive oral cholic and ursodeoxycholic acids. Patients are assessed at 3 and 6 months for liver function response, neurologic status, and nutritional status.
Patients receive treatment until disease progression or unacceptable toxic effects are observed.
Completion date provided represents the completion date of the grant per OOPD records
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- Up to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
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 |
|---|---|---|
| chenodeoxycholic acid | Drug | – |
| cholic acid | Drug | – |
| ursodiol | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Mar 2015.
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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.
Kenneth Setchell study chair
Children's Hospital Medical Center, Cincinnati
Questions about this trial
What is this clinical trial studying?
A study testing chenodeoxycholic acid, cholic acid and ursodiol in people with Infantile Refsum's Disease and Zellweger Syndrome, run by University of Cincinnati.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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: stopped early, as of the sponsor's last update on 25 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early. 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.
Where can I read the original study information?
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 25 Mar 2015.