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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.
 
ClinicalTrials.gov Identifier: NCT03115086
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : July 1, 2020
Sponsor:
Information provided by (Responsible Party):
Retrophin, Inc.

Tracking Information
First Submitted Date January 10, 2017
First Posted Date April 14, 2017
Last Update Posted Date July 1, 2020
Actual Study Start Date July 10, 2017
Estimated Primary Completion Date July 2038   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: April 10, 2017)
  • 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.
  • 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.
  • 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.
  • 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.
  • 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
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title The REPLACE Registry for Cholbam®/Kolbam® (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®/Kolbam® (Cholic Acid)
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.
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 Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 10 Years
Biospecimen Not Provided
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.
Condition Bile Acid Synthesis Disorders
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: April 10, 2017)
110
Original Estimated Enrollment Same as current
Estimated Study Completion Date July 2039
Estimated Primary Completion Date July 2038   (Final data collection date for primary outcome measure)
Eligibility Criteria

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.

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Retrophin Medical Information 1-877-659-5518 medinfo@retrophin.com
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03115086
Other Study ID Numbers 031CHO15001
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Retrophin, Inc.
Study Sponsor Retrophin, Inc.
Collaborators Not Provided
Investigators
Principal Investigator: Feriandas Greblikas, MD Senior Medical Director
PRS Account Retrophin, Inc.
Verification Date June 2020