Back to resultsStudy of Bile Acids in Patients With Peroxisomal Disorders
Primary Purpose
Infantile Refsum's Disease, Zellweger Syndrome, Bifunctional Enzyme Deficiency
Status
Terminated
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
chenodeoxycholic acid
cholic acid
ursodiol
Sponsored by
About this trial
This is an interventional treatment trial for Infantile Refsum's Disease focused on measuring Zellweger syndrome, adrenoleukodystrophy, bifunctional enzyme deficiency, inborn errors of metabolism, infantile Refsum's disease, peroxisomal disorders, pseudo-Zellweger syndrome, rare disease, sphingolipidoses
Eligibility Criteria
Biochemically proven peroxisomal disorder, including: Zellweger syndrome Pseudo-Zellweger syndrome Neonatal adrenoleukodystrophy Bifunctional enzyme deficiency Infantile Refsum's disease
Sites / Locations
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
NCT ID
NCT00004442
First Posted
October 18, 1999
Last Updated
March 24, 2015
Sponsor
University of Cincinnati
Collaborators
Children's Hospital Medical Center, Cincinnati
1. Study Identification
Unique Protocol Identification Number
NCT00004442
Brief Title
Study of Bile Acids in Patients With Peroxisomal Disorders
Study Type
Interventional
2. Study Status
Record Verification Date
November 2000
Overall Recruitment Status
Terminated
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
April 1999 (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
University of Cincinnati
Collaborators
Children's Hospital Medical Center, Cincinnati
4. Oversight
5. Study Description
Brief 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. 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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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, sphingolipidoses
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Enrollment
25 (Anticipated)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
chenodeoxycholic acid
Intervention Type
Drug
Intervention Name(s)
cholic acid
Intervention Type
Drug
Intervention Name(s)
ursodiol
10. Eligibility
Sex
All
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Biochemically proven peroxisomal disorder, including:
Zellweger syndrome
Pseudo-Zellweger syndrome
Neonatal adrenoleukodystrophy
Bifunctional enzyme deficiency
Infantile Refsum's disease
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kenneth Setchell
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Study Chair
12. IPD Sharing Statement
Learn more about this trial
Study of Bile Acids in Patients With Peroxisomal Disorders
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