- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00004442
Study of Bile Acids in Patients With Peroxisomal Disorders
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.
Study Overview
Status
Conditions
Intervention / Treatment
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 Type
Enrollment (Anticipated)
Phase
- Not Applicable
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Biochemically proven peroxisomal disorder, including:
- Zellweger syndrome
- Pseudo-Zellweger syndrome
- Neonatal adrenoleukodystrophy
- Bifunctional enzyme deficiency
- Infantile Refsum's disease
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Study Chair: Kenneth Setchell, Children's Hospital Medical Center, Cincinnati
Study record dates
Study Major Dates
Study Completion
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Demyelinating Diseases
- Kidney Diseases
- Urologic Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Endocrine System Diseases
- Disease
- Congenital Abnormalities
- Liver Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Neuromuscular Diseases
- Neurodegenerative Diseases
- Peripheral Nervous System Diseases
- Metabolism, Inborn Errors
- Mental Retardation, X-Linked
- Intellectual Disability
- Heredodegenerative Disorders, Nervous System
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Nervous System Malformations
- Abnormalities, Multiple
- Leukoencephalopathies
- Adrenal Gland Diseases
- Polyneuropathies
- Hereditary Central Nervous System Demyelinating Diseases
- Adrenal Insufficiency
- Hereditary Sensory and Motor Neuropathy
- Syndrome
- Peroxisomal Disorders
- Adrenoleukodystrophy
- Refsum Disease
- Zellweger Syndrome
- Refsum Disease, Infantile
- Gastrointestinal Agents
- Cathartics
- Cholagogues and Choleretics
- Chenodeoxycholic Acid
- Cholic Acids
- Ursodeoxycholic Acid
Other Study ID Numbers
- 199/13442
- CHMC-C-FDR000995
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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