COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu
Trial record 1 of 1 for:    NCT03115086
Previous Study | Return to List | Next Study

The REPLACE Registry

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 : October 11, 2018
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.

Layout table for study information
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

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.

Layout table for eligibility information
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

Layout table for location contacts
Contact: Retrophin Medical Information 1-877-659-5518

Layout table for location information
United States, Alabama
University of Alabama Birmingham Recruiting
Birmingham, Alabama, United States, 35233
Contact: Janaina Nogueira-Souza    205-638-9918   
Contact: Meg Mosteller-Barnum    1.205.638.5843   
United States, California
Stanford School of Medicine Recruiting
Palo Alto, California, United States, 94305
Contact: Gregory Enns    650-498-5798   
Contact: Thu Quan    650-736-8166   
UC San Francisco Recruiting
San Francisco, California, United States, 94158
Contact: Phillip Rosenthal    415-476-7114   
Contact: Natasha Feier    415-476-1539   
United States, Florida
Kidz Pediatric Multispecialty Group Not yet recruiting
Hollywood, Florida, United States, 33021
Contact: Aymin Delgado    954-967-9400   
Contact: Barbie Gonzalez    954-967-9400   
Miami Children's Hospital Active, not recruiting
Miami, Florida, United States, 33155
United States, Georgia
Children's Center For Digestive Health Recruiting
Atlanta, Georgia, United States, 30342
Contact: Jeffery Lewis    404-257-0799   
Contact: Angela Stallworth    404-503-2291   
United States, Indiana
The Community Health Clinic Recruiting
Topeka, Indiana, United States, 46571
Contact: Zineb Ammous    260-593-1008   
Contact: Sharon Chupp   
United States, Minnesota
University of Minnesota Not yet recruiting
Minneapolis, Minnesota, United States, 55454
Contact: Boris Sudel    612-709-4438   
Contact: Shannon Swim    612-709-4438   
Mayo Clinic Not yet recruiting
Rochester, Minnesota, United States, 55902
Contact: Katryn Furuya    507-266-1078   
Contact: Katrina Pierce    507-266-1078   
United States, Missouri
Washington University at St Louis Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Yumirle Turmelle    314-454-4030   
Contact: Stacy Postma    314-747-5931   
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   
Contact: Beatrice Secheli    212-659-8046   
Montefiore Medical Center Recruiting
New York, New York, United States, 10032
Contact: Nadia Ovchinsky    718-741-2332   
Contact: Trang Nguyen   
Golisano Children's Hospital, URMC Not yet recruiting
Rochester, New York, United States, 14642
Contact: Nanda Kerkar    585-275-2647   
Contact: Monica Javidnia    585-275-2647   
United States, North Carolina
UNC Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Steve Lichtman    919-966-2419   
Contact: Nancy Garbarino    919-843-8734   
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: James E Heubi, MD    513-636-8046   
Contact: Andre Hawkins   
United States, Tennessee
Monroe-Carell Jr. Children's Hospital at Vanderbilt Recruiting
Nashville, Tennessee, United States, 37232
Contact: Lynette Gillis, MD    615-322-7449   
Contact: Jessica Lugo    (615) 343-4929   
Sponsors and Collaborators
Retrophin, Inc.
Additional Information:

Layout table for additonal information
Responsible Party: Retrophin, Inc. Identifier: NCT03115086    
Other Study ID Numbers: 031CHO15001
First Posted: April 14, 2017    Key Record Dates
Last Update Posted: October 11, 2018
Last Verified: October 2018
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