ClinicalTrials.gov
ClinicalTrials.gov Menu

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.
ClinicalTrials.gov Identifier: NCT03115086
Recruitment Status : Recruiting
First Posted : April 14, 2017
Last Update Posted : June 4, 2018
Sponsor:
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.

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.


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.
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.


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


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

Locations
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 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, Missouri
Washington University at St Louis Not yet recruiting
Saint Louis, Missouri, United States, 63110
Contact: Yumirle Turmelle    314-454-4030    Turmelle_Y@kids.wustl.edu   
Contact: Stacy Postma    314-747-5931    postmas@wustl.edu   
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   
Montefiore Medical Center Recruiting
New York, New York, United States, 10032
Contact: Nadia Ovchinsky    718-741-2332    novchins@montefiore.org   
Contact: Trang Nguyen       trannguy@montefiore.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: Andre Hawkins       andre.hawkins@cchmc.org   
United States, Tennessee
Monroe-Carell Jr. Children's Hospital at Vanderbilt 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.

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: June 4, 2018
Last Verified: June 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
Cholbam
Kolbam
The Replace Registry

Additional relevant MeSH terms:
Cholic Acids
Gastrointestinal Agents