Clinical trial NCT03115086: The REPLACE Registry for Cholbam® (Cholic Acid)
Official title: A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam® (Cholic Acid)
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Bile Acid Synthesis Disorders
Keywords: Bile Acid Synthesis Disorder
Zellweger Spectrum Disorder
Peroxisomal Disorder
Cholic Acid
Cholbam
The REPLACE Registry
Lead sponsor: Mirum Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2017-04-14
Enrollment: 55
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All patients with a diagnosis for which Cholbam is indicated are eligible for inclusion in the Registry.

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

Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with worsening cholestasis
timeFrame: 10 Years
measure: Number of participants with new-onset cholestasis
timeFrame: 10 Years
measure: Number of participants with steatorrhea leading to poor growth
timeFrame: 10 Years
measure: Changes in serum levels of fat-soluble vitamins
timeFrame: 10 Years
measure: Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies
timeFrame: 10 Years
measure: Number of participants with growth failure
timeFrame: 10 Years
measure: Death
timeFrame: 10 Years
measure: Adverse effects on pregnancy, pregnancy outcomes, and infant status
timeFrame: 10 Years
measure: All Adverse Events (AEs) and Serious AEs (SAEs)
timeFrame: 10 years
Secondary outcomes:
measure: Changes in Cholbam dosing regimens and reasons for any dose modifications or treatment discontinuations
timeFrame: 10 years
measure: Changes from baseline in weight
timeFrame: 10 years
measure: Changes from baseline in length/height
timeFrame: 10 years
measure: Changes from baseline in head circumference in infants
timeFrame: 10 years
measure: Age-appropriate developmental milestones in infants
timeFrame: 10 years
measure: Changes in prothrombin time (PT)
timeFrame: 10 years
measure: Changes in international normalized ratio (INR)
timeFrame: 10 years
measure: Changes in albumin
timeFrame: 10 years
measure: Changes in bilirubin
timeFrame: 10 years
measure: Changes in direct bilirubin
timeFrame: 10 years
measure: Changes in alanine aminotransferase (ALT)
timeFrame: 10 years
measure: Changes in aspartate aminotransferase (AST)
timeFrame: 10 years
measure: Changes in gamma-glutamyl transferase (GGT)
timeFrame: 10 years
measure: Changes in alkaline phosphatase
timeFrame: 10 years
measure: Presence or absence of urinary bile acids and levels of bile acid intermediaries and urinary bile alcohol
timeFrame: 10 years
measure: All indications for which Cholbam has been prescribed
timeFrame: 10 years
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama Birmingham
state: Alabama
city: Palo Alto
country: United States
facility: Stanford School of Medicine
state: California
city: San Francisco
country: United States
facility: UC San Francisco
state: California
city: Hollywood
country: United States
facility: Kidz Pediatric Multispecialty Group
state: Florida
city: Jacksonville
country: United States
facility: Nemours duPont Pediatrics Specialty Clinic
state: Florida
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Atlanta
country: United States
facility: Children's Center For Digestive Health
state: Georgia
city: Topeka
country: United States
facility: The Community Health Clinic
state: Indiana
city: New Orleans
country: United States
facility: Ochsner Hospital for Children
state: Louisiana
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Columbia
country: United States
facility: Office of Dr. Esperanza Font-Montgomery MD
state: Missouri
city: Saint Louis
country: United States
facility: Washington University at St Louis
state: Missouri
city: New York
country: United States
facility: Icahn School Of Medicine at Mount Sinai
state: New York
city: New York
country: United States
facility: Montefiore Medical Center
state: New York
city: Rochester
country: United States
facility: Golisano Children's Hospital, URMC
state: New York
city: Chapel Hill
country: United States
facility: UNC Chapel Hill
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma
state: Oklahoma
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Monroe-Carell Jr. Children's Hospital at Vanderbilt
state: Tennessee
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
Study officials:
affiliation: Vice President, Clinical Development
name: Sagar A Vaidya, MD, PhD
role: STUDY_DIRECTOR
References: