Trials Sponsors Conditions Drugs Pricing

Conditions

Bile Acid Synthesis Disorders

Keywords

Bile Acid Synthesis DisorderZellweger Spectrum DisorderPeroxisomal DisorderCholic AcidCholbamThe REPLACE Registry

Outcome Measures

Primary Outcomes (9)

Number of participants with worsening cholestasis
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
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
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

Secondary Outcomes (16)

Changes in Cholbam dosing regimens and reasons for any dose modifications or treatment discontinuations
10 years
Changes from baseline in weight
10 years
Changes from baseline in length/height
10 years
Changes from baseline in head circumference in infants
10 years
Age-appropriate developmental milestones in infants
10 years
+11 more secondary outcomes

Study Arms (2)

Existing User

Patients who have been using Cholbam for at least 30 days

Drug: Cholbam

New User

First-time initiators of Cholbam

Drug: Cholbam

Interventions

CholbamDrug

Cholbam prescribed according to the approved label.

Existing UserNew User

Eligibility Criteria

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-Probability Sample

Study Population

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

You may qualify if:

Male and female patients, of any age.

Sponsors & Collaborators

Mirum Pharmaceuticals, Inc.
Industrylead

Study Sites (24)

University of Alabama Birmingham

Birmingham, Alabama, 35233, United States

Stanford School of Medicine

Palo Alto, California, 94305, United States

UC San Francisco

San Francisco, California, 94158, United States

Kidz Pediatric Multispecialty Group

Hollywood, Florida, 33021, United States

Nemours duPont Pediatrics Specialty Clinic

Jacksonville, Florida, 32207, United States

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, United States

MeSH Terms

Conditions

Zellweger SyndromePeroxisomal Disorders

Interventions

Cholic Acid

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy Complications

Study Officials

Sagar A Vaidya, MD, PhD
Vice President, Clinical Development
STUDY DIRECTOR

Study Design

Study Type: observational
Observational Model: COHORT
Time Perspective: PROSPECTIVE
Target Duration: 10 Years
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

January 10, 2017

First Posted

April 14, 2017

Study Start

July 10, 2017

Actual (reported)

Primary Completion

July 2038

Estimated

Planned date reported by the registry; this milestone is not confirmed as achieved.

The REPLACE Registry for Cholbam® (Cholic Acid)

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (9)

Secondary Outcomes (16)

Study Arms (2)

Existing User

New User

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (24)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Intervention Hierarchy (Ancestors)

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (9)

Secondary Outcomes (16)

Study Arms (2)

Existing User

New User

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (24)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Intervention Hierarchy (Ancestors)

Data Sharing

Source record

Locations