Study of Bile Acids in Patients With Peroxisomal Disorders

Brief Title

Study of Bile Acids in Patients With Peroxisomal Disorders


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
    


Study Type

Interventional




Condition

Infantile Refsum's Disease

Intervention

chenodeoxycholic acid


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

25


Completion Date

April 1999


Eligibility Criteria

        Biochemically proven peroxisomal disorder, including:

          -  Zellweger syndrome

          -  Pseudo-Zellweger syndrome

          -  Neonatal adrenoleukodystrophy

          -  Bifunctional enzyme deficiency

          -  Infantile Refsum's disease
      

Gender

All

Ages

N/A - 5 Years

Accepts Healthy Volunteers

No

Contacts

Kenneth Setchell, , 



Administrative Informations


NCT ID

NCT00004442

Organization ID

199/13442

Secondary IDs

CHMC-C-FDR000995


Study Sponsor

University of Cincinnati

Collaborators

 Children's Hospital Medical Center, Cincinnati

Study Sponsor

Kenneth Setchell, Study Chair, Children's Hospital Medical Center, Cincinnati


Verification Date

November 2000