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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

85 participants

Study Classification

INTERVENTIONAL

Study Start Date

1992-01-31

Study Completion Date

2009-12-31

Brief Summary

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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

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Detailed Description

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Investigational Plan:

A Phase III, open label, single arm, nonrandomized, non-comparative, compassionate treatment study of cholic acid in the treatment of defects of bile acid metabolism.

The study was begun with a single study site at Cincinnati Children's Hospital Medical Center (CCHMC), but in 2005 was expanded so that compassionate treatment could be provided to additional patients who had been identified with inborn errors of bile metabolism through the center's screening/diagnostic program.

Patients who were screened were contacted and evaluated with respect to the inclusion/exclusion criteria. Signed informed consent by the patient and/or parents/legal guardian was obtained as soon as it is confirmed that the patient met inclusion/exclusion criteria and the parents/guardian would agree for the child to participate in the study.

The primary interventions for the study were:

1. Administration of study drug.
2. Collection of baseline physical exam, vital signs, blood and urine samples for laboratory tests.
3. Collection of periodic physical exam, vital signs, blood and urine samples for laboratory tests during the period of administration of the study drug.
4. Collection of any adverse event information.

Time and Events Schedule:

Baseline:

1. Confirm eligibility
2. Obtain written informed consent from patient and/or parents/legal guardian
3. Collect demographic data and disease and medication history, including family history

Baseline and Ongoing:
4. Obtain body weight
5. Record adverse events
6. Obtain blood and urine samples for laboratory tests
7. Initiate study drug therapy \& monitor study drug therapy and adjust dose as needed

Conditions

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Infantile Refsum's Disease Zellweger Syndrome Adrenoleukodystrophy Peroxisomal Disorders Cholestasis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cholic Acid

Group Type OTHER

Cholic Acids

Intervention Type DRUG

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

Interventions

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Cholic Acids

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

Intervention Type DRUG

Other Intervention Names

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Cholic Cholic Acid Cholic Acid Capsules

Eligibility Criteria

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Inclusion Criteria

* Infants \< age 3 months
* Children presenting for evaluation of cholestasis defined as a conjugated bilirubin \> 2mg/dl or increased serum bile acids
* Older subjects of any age with cholestatic liver disease if urine screens suggested that they had inborn errors of bile acid metabolism
* Confirmation of a diagnosis of an inborn error of bile acid synthesis based upon urine analysis by FAB-MS to determine whether specific abnormalities in bile acid synthesis are indicated
* The patient and/or parent/legal guardian must have signed the written informed consent document before study start.
* The patient must be willing and able to comply with all study assessments and procedures.

Eligible Sex

ALL

Accepts Healthy Volunteers

No