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Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

NCT00007020 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 85

Last updated 2023-10-03

Study results available
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Summary

OBJECTIVES:

I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism

II. To assess the safety and tolerability of cholic acid

Conditions

  • Infantile Refsum's Disease
  • Zellweger Syndrome
  • Adrenoleukodystrophy
  • Peroxisomal Disorders
  • Cholestasis

Interventions

DRUG

Cholic Acids

10-15 mg/kg body weight/day taken orally.

Sponsors & Collaborators

  • Children's Hospital Medical Center, Cincinnati

    collaborator OTHER

  • Mirum Pharmaceuticals, Inc.

    lead INDUSTRY

Principal Investigators

  • James Heubi, MD · Children's Hospital Medical Center, Cincinnati

  • Kenneth Setchell, PhD · Children's Hospital Medical Center, Cincinnati

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
1992-01-31
Primary Completion
2009-12-31
Completion
2009-12-31

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00007020 on ClinicalTrials.gov