Progressive Familial Intrahepatic Cholestasis in Indian Children - Establishing an Indian PFIC Re… (NCT05704517) | Clinical Trial Compass
UnknownNot Applicable
Progressive Familial Intrahepatic Cholestasis in Indian Children - Establishing an Indian PFIC Registry
India200 participantsStarted 2023-01-28
Plain-language summary
The project will amalgamate data from several large Indian centers to describe the genotype, clinical spectrum, natural course, genotype-phenotype correlation, outcome, and response to medical therapy in Indian children with progressive familial intrahepatic cholestasis (PFIC). This will be the first such Indian registry of children with PFIC. There are currently limited single-center studies describing the genotype, natural course, and outcome of Indian children with PFIC.
Data will be collected retrospectively from the participating centers across the country. Only genetically confirmed cases would be included.
Who can participate
Age range
18 Years
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:
* Genetically proven homozygous or compound heterozygous mutations of ATP8B1/ ABCB11/ ABCB4/ TJP2/ NR1H4/ MYO5B/ USP53/ KIF12 AND
* Clinical and biochemical evidence of chronic cholestatic disease AND / OR
* Histological features of intrahepatic cholestasis with suggestive immunohistochemistry
Exclusion Criteria:
* Genetic analysis showing mutations unrelated to intrahepatic cholestasis according to database
* Clinical, biochemical, and histological evidence of progressive familial intrahepatic cholestasis without a genetic sequencing report
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
Native liver survival (in percentage) at the latest follow up in different types of PFIC
Timeframe: Through study completion - average of 1 year
Trial details
NCT IDNCT05704517
SponsorInstitute of Liver and Biliary Sciences, India