The REPLACE Registry for Cholbam® (Cholic Acid)

NCT ID: NCT03115086

Last Updated: 2023-10-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

55 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-07-10

Study Completion Date

2039-07-31

Brief Summary

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This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using Cholbam for at least 30 days (existing users) and those who are first-time initiators of Cholbam.

Detailed Description

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No experimental intervention is involved. Patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.

Conditions

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Bile Acid Synthesis Disorders

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Existing User

Patients who have been using Cholbam for at least 30 days

Cholbam

Intervention Type DRUG

Cholbam prescribed according to the approved label.

New User

First-time initiators of Cholbam

Cholbam

Intervention Type DRUG

Cholbam prescribed according to the approved label.

Interventions

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Cholbam

Cholbam prescribed according to the approved label.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

1. Male and female patients, of any age.
2. The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent, and the patient, if less than 18 years of age, is willing to provide assent as appropriate and in accordance with local regulatory, IRB, and EC requirements.
3. The patient has a diagnosis for which Cholbam is indicated.
4. The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).

Exclusion Criteria

1\. Patients who, by judgement of the Investigator, will not be able to comply with the requirements of the protocol will be excluded
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Mirum Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sagar A Vaidya, MD, PhD

Role: STUDY_DIRECTOR

Vice President, Clinical Development

Locations

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University of Alabama Birmingham

Birmingham, Alabama, United States

Site Status

Stanford School of Medicine

Palo Alto, California, United States

Site Status

UC San Francisco

San Francisco, California, United States

Site Status

Kidz Pediatric Multispecialty Group

Hollywood, Florida, United States

Site Status

Nemours duPont Pediatrics Specialty Clinic

Jacksonville, Florida, United States

Site Status

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Children's Center For Digestive Health

Atlanta, Georgia, United States

Site Status

The Community Health Clinic

Topeka, Indiana, United States

Site Status

Ochsner Hospital for Children

New Orleans, Louisiana, United States

Site Status

University of Minnesota

Minneapolis, Minnesota, United States

Site Status

Mayo Clinic

Rochester, Minnesota, United States

Site Status

Office of Dr. Esperanza Font-Montgomery MD

Columbia, Missouri, United States

Site Status

Washington University at St Louis

St Louis, Missouri, United States

Site Status

Icahn School Of Medicine at Mount Sinai

New York, New York, United States

Site Status

Montefiore Medical Center

New York, New York, United States

Site Status

Golisano Children's Hospital, URMC

Rochester, New York, United States

Site Status

UNC Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

University of Oklahoma

Oklahoma City, Oklahoma, United States

Site Status

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

Monroe-Carell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, United States

Site Status

Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

Countries

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United States

Other Identifiers

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031CHO15001

Identifier Type: -

Identifier Source: org_study_id

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