The REPLACE Registry for Cholbam® (Cholic Acid) (NCT03115086) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
The REPLACE Registry for Cholbam® (Cholic Acid)
United States55 participantsStarted 2017-07-10
Plain-language summary
This is a prospective, observational, non-interventional patient registry study designed to document product safety and effectiveness outcomes for 10 years in patients treated with Cholbam, including those who have been using Cholbam for at least 30 days (existing users) and those who are first-time initiators of Cholbam.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male and female patients, of any age.
. The patient and/or the patient's parent/legal guardian is willing and able to provide signed informed consent, and the patient, if less than 18 years of age, is willing to provide assent as appropriate and in accordance with local regulatory, IRB, and EC requirements.
. The patient has a diagnosis for which Cholbam is indicated.
. The patient is or will be treated with Cholbam at the time of signing the informed consent form (ICF) (enrollment).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of participants with worsening cholestasis
Timeframe: 10 Years
2
Number of participants with new-onset cholestasis
Timeframe: 10 Years
3
Number of participants with steatorrhea leading to poor growth
Timeframe: 10 Years
4
Changes in serum levels of fat-soluble vitamins
Timeframe: 10 Years
5
Neuropathic signs or symptoms related to fat-soluble vitamin deficiencies
Timeframe: 10 Years
6
Number of participants with growth failure
Timeframe: 10 Years
7
Death
Timeframe: 10 Years
8
Adverse effects on pregnancy, pregnancy outcomes, and infant status