The REPLACE Registry for Cholbam® (Cholic Acid)

September 29, 2023 updated by: Mirum Pharmaceuticals, Inc.

A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid)

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
55

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • University of Alabama Birmingham
    • California
      • Palo Alto, California, United States, 94305
        • Stanford School of Medicine
      • San Francisco, California, United States, 94158
        • UC San Francisco
    • Florida
      • Hollywood, Florida, United States, 33021
        • Kidz Pediatric Multispecialty Group
      • Jacksonville, Florida, United States, 32207
        • Nemours duPont Pediatrics Specialty Clinic
      • Miami, Florida, United States, 33155
        • Nicklaus Children's Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Children's Healthcare of Atlanta
      • Atlanta, Georgia, United States, 30342
        • Children's Center For Digestive Health
    • Indiana
      • Topeka, Indiana, United States, 46571
        • The Community Health Clinic
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Ochsner Hospital for Children
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • University of Minnesota
      • Rochester, Minnesota, United States, 55902
        • Mayo Clinic
    • Missouri
      • Columbia, Missouri, United States, 65201
        • Office of Dr. Esperanza Font-Montgomery MD
      • Saint Louis, Missouri, United States, 63110
        • Washington University at St Louis
    • New York
      • New York, New York, United States, 10029
        • ICAHN School of Medicine at Mount Sinai
      • New York, New York, United States, 10032
        • Montefiore Medical Center
      • Rochester, New York, United States, 14642
        • Golisano Children's Hospital, URMC
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • UNC Chapel Hill
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • University of Oklahoma
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Monroe-Carell Jr. Children's Hospital at Vanderbilt
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Primary Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

All patients with a diagnosis for which Cholbam is indicated are eligible for inclusion in the Registry.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Existing User
Patients who have been using Cholbam for at least 30 days
Drug: Cholbam
Cholbam prescribed according to the approved label.
New User
First-time initiators of Cholbam
Drug: Cholbam
Cholbam prescribed according to the approved label.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Number of participants with worsening cholestasis
Time Frame: 10 Years
Worsening cholestasis will be identified by measuring direct total bilirubin concentration in blood and will be defined as a 25% increase from previous measurement if the total bilirubin is >1mg/dL.
10 Years
Number of participants with new-onset cholestasis
Time Frame: 10 Years
Patients with new-onset cholestasis will be identified by detecting abnormal direct bilirubin concentration >1mg/dL or direct bilirubin greater than 20% of the total bilirubin if total bilirubin is >5mg/dL.
10 Years
Number of participants with steatorrhea leading to poor growth
Time Frame: 10 Years
Steatorrhea leading to poor growth in children, which will be defined as a decrease in growth percentiles from the original percentile at enrollment to the registry study.
10 Years
Changes in serum levels of fat-soluble vitamins
Time Frame: 10 Years
Fat-soluble vitamin (A, D, E, K) deficiencies will be identified by comparing results from serum assays (Vitamin A: Retinol, Vitamin D: 250HD2 + 250HD3, Vitamin K: Serum Vitamin K, Vitamin E: Serum Vitamin E) for each vitamin with standard ranges.
10 Years
Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies
Time Frame: 10 Years
Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies (peripheral neuropathy, cerebellar ataxia) will be assessed by the Investigator.
10 Years
Number of participants with growth failure
Time Frame: 10 Years
Growth failure that the physician judges to be attributable to malabsorption.
10 Years
Death
Time Frame: 10 Years
AEs and SAEs leading to death will be recorded. The relatedness of death to Cholbam or disease progression will be recorded.
10 Years
Adverse effects on pregnancy, pregnancy outcomes, and infant status
Time Frame: 10 Years
Adverse effects on pregnancy, pregnancy outcomes, and infant status will be recorded.
10 Years
All Adverse Events (AEs) and Serious AEs (SAEs)
Time Frame: 10 years
All other AEs and SAEs will be collected.
10 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Changes in Cholbam dosing regimens and reasons for any dose modifications or treatment discontinuations
Time Frame: 10 years
10 years
Changes from baseline in weight
Time Frame: 10 years
For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.
10 years
Changes from baseline in length/height
Time Frame: 10 years
For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Some patients will be adult or will have achieved final height. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.
10 years
Changes from baseline in head circumference in infants
Time Frame: 10 years
For patients aged 18 years of or less, these data will be converted into Z-scores based on the WHO growth standards for infants aged 0-2 years and the Centers for Disease Control and Prevention growth standards for children 2-20 years. Growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.
10 years
Age-appropriate developmental milestones in infants
Time Frame: 10 years
Measured by WHO Motor Development Milestones
10 years
Changes in prothrombin time (PT)
Time Frame: 10 years
10 years
Changes in international normalized ratio (INR)
Time Frame: 10 years
10 years
Changes in albumin
Time Frame: 10 years
10 years
Changes in bilirubin
Time Frame: 10 years
10 years
Changes in direct bilirubin
Time Frame: 10 years
10 years
Changes in alanine aminotransferase (ALT)
Time Frame: 10 years
10 years
Changes in aspartate aminotransferase (AST)
Time Frame: 10 years
10 years
Changes in gamma-glutamyl transferase (GGT)
Time Frame: 10 years
10 years
Changes in alkaline phosphatase
Time Frame: 10 years
10 years
Presence or absence of urinary bile acids and levels of bile acid intermediaries and urinary bile alcohol
Time Frame: 10 years
10 years
All indications for which Cholbam has been prescribed
Time Frame: 10 years
Description of all indications for which Cholbam has been prescribed
10 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Mirum Pharmaceuticals, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Sagar A Vaidya, MD, PhD, Vice President, Clinical Development

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 10, 2017

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 1, 2038

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 1, 2039

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 10, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 10, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 14, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 2, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 29, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2023

More Information

Terms related to this study

Keywords

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 031CHO15001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

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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