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The REPLACE Registry

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ClinicalTrials.gov Identifier: NCT03115086
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : December 4, 2017
Information provided by (Responsible Party):

Study Description
Brief Summary:
This is a prospective, observational, non-interventional patient registry designed to document long term safety and clinical outcomes for 10 years in patients treated with Cholbam/Kolbam, including those who have been using Cholbam/Kolbam and those who start Cholbam/Kolbam treatment at enrollment.

Condition or disease
Bile Acid Synthesis Disorders

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 Design

Study Type : Observational [Patient Registry]
Estimated Enrollment : 110 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 10 Years
Official Title: A Prospective, Observational, Non-Interventional, Post-Marketing, Patient Registry to Collect Data on Routine Clinical Care in Patients Treated With Cholbam®/Kolbam® (Cholic Acid)
Actual Study Start Date : July 10, 2017
Estimated Primary Completion Date : July 2030
Estimated Study Completion Date : August 2030

Groups and Cohorts

Outcome Measures

Primary Outcome Measures :
  1. Number of Participants with New-Onset or Worsening Cholestasis [ Time Frame: 10 Years ]
    Safety and effectiveness will be measured by new-onset or worsening cholestasis. Worsening cholestasis will be identified by measuring direct total bilirubin concentration, and will be defined as a 25% increase from previous measurement if the total bilirubin is >1mg/dL. 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.

  2. Number of Participants with Steatorrhea Leading to Poor Growth [ Time Frame: 10 Years ]
    Safety and effectiveness will be measured by steatorrhea leading to poor growth in children, which will be defined as a decrease in percentiles from the original percentile at enrollment to the registry study.

  3. Number of Participants changes in Serum Levels of Fat Soluble Vitamins [ Time Frame: 10 Years ]
    Safety and effectiveness will be measured changes in serum levels of fat soluble vitamins measured by deficiencies 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.

  4. Number of Participants with Growth Failure [ Time Frame: 10 Years ]
    Safety and effectiveness will be measured by growth failure will be defined as a decrease in percentiles from the original percentile at entry point into the registry study.

  5. Number of Participants with Treated Related Adverse Events [ Time Frame: 10 years ]
    Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
All patients with a diagnosis for which Cholbam/Kolbam is indicated are eligible for inclusion in the Registry.

Inclusion Criteria:

  1. Male and female patients, of any age.
  2. The patient has a diagnosis for which Cholbam/Kolbam is indicated.
  3. The patient is treated with Cholbam/Kolbam at enrollment.

Exclusion Criteria:

1. Patients who will not be able to comply with the requirements of the protocol will be excluded.

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03115086

Contact: Retrophin Medical Information 1-877-659-5518 medinfo@retrophin.com

United States, Alabama
University of Alabama Birmingham Recruiting
Birmingham, Alabama, United States, 35233
Contact: Janaina Nogueira-Souza    205-638-9918    JNogueira@peds.uab.edu   
Contact: Meg Mosteller-Barnum    1.205.638.5843    lbarnum@peds.uab.edu   
United States, California
Stanford School of Medicine Recruiting
Palo Alto, California, United States, 94305
Contact: Gregory Enns    650-498-5798    genns@stanford.edu   
Contact: Thu Quan    650-736-8166    tquan@stanford.edu   
UC San Francisco Not yet recruiting
San Francisco, California, United States, 94158
Contact: Phillip Rosenthal    415-476-7114    prosenth@ucsf.edu   
Contact: Natasha Feier    415-476-1539    natasha.feier@ucsf.edu   
United States, Florida
Miami Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: Erick Hernandez, MD    305-243-4029    Erick.Hernandez@mch.com   
Contact: Rebeca Deleon    786-624-3829    Rebeca.deleon@mch.com   
United States, Georgia
Children's Center For Digestive Health Recruiting
Atlanta, Georgia, United States, 30342
Contact: Jeffery Lewis    404-257-0799    jlewis@ccdhc.org   
Contact: Angela Stallworth    404-503-2291    astallworth@gicareforkids.com   
United States, New York
Icahn School Of Medicine at Mount Sinai Recruiting
New York, New York, United States, 10029
Contact: Ronen Arnon    212-659-8060    ronen.arnon@mountsinai.org   
Contact: Beatrice Secheli    212-659-8046    Beatrice.Secheli@MountSinai.org   
United States, North Carolina
UNC Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Steve Lichtman    919-966-2419    Lichtman@med.unc.edu   
Contact: Nancy Garbarino    919-843-8734    Nancy84@med.unc.edu   
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: James E Heubi, MD    513-636-8046    james.heubi@cchmc.org   
Contact: Donna Buckley    (513) 636-8549    Donna.Buckley@cchmc.org   
United States, Tennessee
Monroe-Carell Jr. Children's Hospital at Vanderbilt Not yet recruiting
Nashville, Tennessee, United States, 37232
Contact: Lynette Gillis, MD    615-322-7449    Lynette.Gillis@vanderbilt.edu   
Contact: Jessica Lugo    (615) 343-4929    jessica.m.lugo@vanderbilt.edu   
Sponsors and Collaborators
Retrophin, Inc.
More Information

Responsible Party: Retrophin, Inc.
ClinicalTrials.gov Identifier: NCT03115086     History of Changes
Other Study ID Numbers: 031CHO15001
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: December 4, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Retrophin, Inc.:
Bile Acid Synthesis Disorder
Zellweger Spectrum Disorder
Peroxisomal Disorder
Cholic Acid
The Replace Registry

Additional relevant MeSH terms:
Cholic Acids
Gastrointestinal Agents