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The Replace Registry

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified April 2017 by Retrophin, Inc.
Information provided by (Responsible Party):
Retrophin, Inc. Identifier:
First received: January 10, 2017
Last updated: April 10, 2017
Last verified: April 2017
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.

Bile Acid Synthesis Disorders

Study Type: Observational [Patient Registry]
Study Design: 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)

Resource links provided by NLM:

Further study details as provided by Retrophin, Inc.:

Primary Outcome Measures:
  • 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

Estimated Enrollment: 110
Anticipated Study Start Date: May 2017
Estimated Study Completion Date: August 2030
Estimated Primary Completion Date: May 2030 (Final data collection date for primary outcome measure)
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.

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
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT03115086

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

United States, Florida
Miami Children's Hospital Not yet recruiting
Miami, Florida, United States, 33155
Contact: Erick Hernandez, MD    305-243-4029   
United States, Ohio
Cincinnati Children's Hospital Medical Center Not yet recruiting
Cincinnati, Ohio, United States, 45229
Contact: James E Heubi, MD    513-636-8046   
Contact: Donna Buckley    (513) 636-8549   
Principal Investigator: James E Heubi, MD         
United States, Tennessee
Vanderbilt Medical Medical Center Not yet recruiting
Nashville, Tennessee, United States, 37232
Contact: Lynette Gillis, MD    615-322-7449   
Sponsors and Collaborators
Retrophin, Inc.
  More Information

Responsible Party: Retrophin, Inc. Identifier: NCT03115086     History of Changes
Other Study ID Numbers: 031CHO15001
Study First Received: January 10, 2017
Last Updated: April 10, 2017
Individual Participant Data  
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 processed this record on April 24, 2017