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The REPLACE Registry for Cholbam®/Kolbam® (Cholic Acid)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03115086
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : July 1, 2020
Information provided by (Responsible Party):
Retrophin, Inc.

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.

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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 2038
Estimated Study Completion Date : July 2039

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
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.

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 identifier (NCT number): NCT03115086

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Contact: Retrophin Medical Information 1-877-659-5518

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Sponsors and Collaborators
Retrophin, Inc.
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Principal Investigator: Feriandas Greblikas, MD Senior Medical Director
Additional Information:

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Responsible Party: Retrophin, Inc. Identifier: NCT03115086    
Other Study ID Numbers: 031CHO15001
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: July 1, 2020
Last Verified: June 2020
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