Progressive Familial Intrahepatic CholestasisNCT05704517
Progressive Familial Intrahepatic Cholestasis in Indian Children - Establishing an Indian PFIC Registry
An observational study of Progressive Familial Intrahepatic Cholestasis, run by Institute of Liver and Biliary Sciences, India at 1 site in India.
Trial 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). Read more about this study
- Who is it for?
- Ages Up to 18 years
- Conditions the study is for
- Progressive Familial Intrahepatic Cholestasis
- How many people
- 200 participants
- Where
- 1 siteIndia
- Study dates
- 28 Jan 2023to 31 Dec 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: All genetically confirmed cases of progressive familial intrahepatic cholestasis (PFIC)
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
India1 site, 1 recruiting
- Recruiting
Institute of Liver and Biliary Sciences
New Delhi
Bikrant Lal, DM
About this study
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.
Read the detailed description
There is a lack of robust literature from India on PFIC. The study would be the first to extensively describe the genotype of Indian children with PFIC and their natural course. Being a multicentric study, the results generated would therefore be applicable to the whole of the country. Understanding the prevalent genotypes in the Indian population and their related phenotype would help both the individual management decisions of these patients and further policy-making for their diagnosis and treatment. With the advent of genetic diagnosis through sequencing techniques and these tests becoming more affordable, every Indian center is now diagnosing a fair number of these cases which used to go undiagnosed previously. This has changed the landscape of cholestatic liver disease in children where PFICs are now the most prevalent pediatric cholestatic disorder. European studies have demonstrated 2 common mutations where patients respond very well to surgical biliary diversion and have good native liver survival. These mutations have rarely been reported in India. Results from this study could thus guide appropriate decision-making based on outcome and help choose the modality of treatment for the individual patient - medical, surgical biliary diversion, or liver transplantation.
- Medical subject headings
- Cholestasis, progressive familial intrahepatic 1
What is being measured
The primary outcome is the main question the study is designed to answer.
Native liver survival (in percentage) at the latest follow up in different types of PFIC
The proportion of patients of each subtype of PFIC who have survived with their own with their native liver till the time of last follow up
Measured: Through study completion - average of 1 year
Secondary outcomes (6)
Describe the spectrum of genetic mutations in Indian children with familial intrahepatic cholestasis
This objective would be assessed by studying the frequency of each of these subtypes of PFIC and describing the various genotypes under each subtype.
Measured: Through study completion - average of 1 year
Genotype-phenotype correlation in Indian children with various types of progressive familial intrahepatic cholestasis
Well defined clinical parameters (jaundice, pruritus, presentation, presence of cirrhosis) and clinical end points (death, liver transplantation, native liver survival) would be used to describe the genotype-phenotype correlation. We will try to club various mutations into categories based on the type of mutations to better assess the genotype-phenotype correlation.
Measured: Through study completion - average of 1 year
Analyse the natural course after surgical biliary diversion in Indian children with various types of progressive familial intrahepatic cholestasis
We will analyze the response to surgical biliary diversion in various types of PFIC with emphasis on identifying the genotypes likely to gain maximum benefit through this procedure. Biochemical parameters to assess/ predict the response would be evaluated by regression analysis.
Measured: Through study completion - average of 1 year
Complications after liver transplantation in children with various types of progressive familial intrahepatic cholestasis till the time of last follow up
The proportion of children going for liver transplantation at various time points would be assessed. Genetic, clinical or biochemical parameters predicting need for imminent/ future liver transplantation would be determined by regression analysis.
Measured: Through study completion - average of 1 year
Proportion of patients surviving with native liver in uncommon variants of progressive familial intrahepatic cholestasis
Describe the prevalence, natural course and outcome of uncommon variants of progressive familial intrahepatic cholestasis in Indian children
Measured: Through study completion - average of 1 year
Develop and maintain a registry of Indian patients with progressive familial intrahepatic cholestasis (Indian PFIC registry)
A registry (Indian PFIC registry) would be maintained with use of appropriate data managing software.
Measured: Through study completion - average of 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2023
- Study start28 Jan 2023
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Progressive Familial Intrahepatic CholestasisStudies of the same condition, open and under way.
- More trials run by Institute of Liver and Biliary Sciences, IndiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Institute of Liver and Biliary Sciences, India
- Sponsor type
- University, hospital or other
- Collaborators
- Apollo Hospital, New Delhi, India, Sahyadri Superspeciality Hospital Pvt Ltd, Pune, Glenegales Global Health City, Chennai, KEM Hospital Research Centre, Jaslok Hospital and Research Centre, Institute of Child Health, Chennai
- Responsible party
- Sponsor
- Investigators
- Bikrant B Lal, MD, DM, Principal investigator, Institute of Liver and Biliary Sciences, New Delhi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05704517
- Sponsor's protocol ID
- India_PFIC
- Status last verified
- Mar 2024
- First submitted
- 21 Dec 2022
- Last updated
- 21 Mar 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual patient data would not be shared with other researchers.
Publications
Agarwal S, Lal BB, Rawat D, Rastogi A, Bharathy KG, Alam S. Progressive Familial Intrahepatic Cholestasis (PFIC) in Indian Children: Clinical Spectrum and Outcome. J Clin Exp Hepatol. 2016 Sep;6(3):203-208. doi: 10.1016/j.jceh.2016.05.003. Epub 2016 May 24.
PubMed 27746616Sharma A, Poddar U, Agnihotry S, Phadke SR, Yachha SK, Aggarwal R. Spectrum of genomic variations in Indian patients with progressive familial intrahepatic cholestasis. BMC Gastroenterol. 2018 Jul 4;18(1):107. doi: 10.1186/s12876-018-0835-6.
PubMed 29973134Mitra S, Das A, Thapa B, Kumar Vasishta R. Phenotype-Genotype Correlation of North Indian Progressive Familial Intrahepatic Cholestasis type2 Children Shows p.Val444Ala and p.Asn591Ser Variants and Retained BSEP Expression. Fetal Pediatr Pathol. 2020 Apr;39(2):107-123. doi: 10.1080/15513815.2019.1641860. Epub 2019 Jul 23.
PubMed 31335238van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipinski P, Czubkowski P, Rock N, Shagrani M, Broering D, Algoufi T, Mazhar N, Nicastro E, Kelly DA, Nebbia G, Arnell H, Bjorn Fischler, Hulscher JBF, Serranti D, Arikan C, Polat E, Debray D, Lacaille F, Goncalves C, Hierro L, Munoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsofi A, Calvo PL, Grabhorn E, Sturm E, van der Woerd WJ, Kamath BM, Wang JS, Li L, Durmaz O, Onal Z, Bunt TMG, Hansen BE, Verkade HJ; NAtural course and Prognosis of PFIC and Effect of biliary Diversion (NAPPED) consortium. Genotype correlates with the natural history of severe bile salt export pump deficiency. J Hepatol. 2020 Jul;73(1):84-93. doi: 10.1016/j.jhep.2020.02.007. Epub 2020 Feb 20.
PubMed 32087350van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Shneider BL, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipinski P, Czubkowski P, Rock N, Shagrani M, Broering D, Algoufi T, Mazhar N, Nicastro E, Kelly D, Nebbia G, Arnell H, Fischler B, Hulscher JBF, Serranti D, Arikan C, Debray D, Lacaille F, Goncalves C, Hierro L, Munoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsofi A, Luigi Calvo P, Krebs-Schmitt D, Hartleif S, van der Woerd WL, Wang JS, Li LT, Durmaz O, Kerkar N, Horby Jorgensen M, Fischer R, Jimenez-Rivera C, Alam S, Cananzi M, Laverdure N, Targa Ferreira C, Ordonez F, Wang H, Sency V, Mo Kim K, Chen HL, Carvalho E, Fabre A, Quintero Bernabeu J, Alonso EM, Sokol RJ, Suchy FJ, Loomes KM, McKiernan PJ, Rosenthal P, Turmelle Y, Rao GS, Horslen S, Kamath BM, Rogalidou M, Karnsakul WW, Hansen B, Verkade HJ; Natural Course and Prognosis of PFIC and Effect of Biliary Diversion Consortium. Impact of Genotype, Serum Bile Acids, and Surgical Biliary Diversion on Native Liver Survival in FIC1 Deficiency. Hepatology. 2021 Aug;74(2):892-906. doi: 10.1002/hep.31787. Epub 2021 Jul 13.
PubMed 33666275Alam S, Lal BB. Recent updates on progressive familial intrahepatic cholestasis types 1, 2 and 3: Outcome and therapeutic strategies. World J Hepatol. 2022 Jan 27;14(1):98-118. doi: 10.4254/wjh.v14.i1.98.
PubMed 35126842Alam S, Lal BB, Kumar K, Sibal A, Shah V, Ramakrishna SH, Shah I, Nalwalla Z, Snehavardhan P, Bavdekar A, Wadhwa N, Maria A, Gupta S, Nagral A, Dheivamani N, Deshmukh A, Verma L, Rai A, Ravindranath A, Verma SK, Shah A, Ashritha A, Suchismita A, Waikar Y, Tripathi PR, Upadhyay P, Matthai J, Malhotra S, Gautam V, Kumar V, Durga D, Bhat NK, Peethambaran M, Srikanth KP, Sivaramakrishnan VM, Sood V, Khanna R. Impact of genotype on childhood bile salt export pump deficiency in Indian Progressive Familial Intrahepatic Cholestasis registry. J Pediatr Gastroenterol Nutr. 2026 Oct 26. doi: 10.1002/jpn3.70610. Online ahead of print.
PubMed 42929328Kaur P, Lal BB, Janakiraman D, Dheivamani N, Pandey S, Bavdekar A, Shah A, Verma SK, Shah V, Maria A, Wadhwa N, Singh SK, Sood V, Khanna R, Alam S. Portal hypertension in doublecortin domain-containing protein 2 (DCDC2)-related neonatal sclerosing cholangitis. J Pediatr Gastroenterol Nutr. 2025 Jan;80(1):189-196. doi: 10.1002/jpn3.12414. Epub 2024 Nov 18.
PubMed 39552453
Study at a glance
From the registry record, last updated by the sponsor on 21 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Progressive Familial Intrahepatic Cholestasis in Indian Children - Establishing an Indian PFIC Registry
- ClinicalTrials.gov ID
- NCT05704517
- Acronym / sponsor ID
- India_PFIC
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Institute of Liver and Biliary Sciences, IndiaUniversity, hospital or other
- Collaborators
- Apollo Hospital, New Delhi, India, Sahyadri Superspeciality Hospital Pvt Ltd, Pune, Glenegales Global Health City, Chennai, KEM Hospital Research Centre, Jaslok Hospital and Research Centre, Institute of Child Health, Chennai
- Study start
- 28 Jan 2023 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 30 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
There is a lack of robust literature from India on PFIC. The study would be the first to extensively describe the genotype of Indian children with PFIC and their natural course. Being a multicentric study, the results generated would therefore be applicable to the whole of the country. Understanding the prevalent genotypes in the Indian population and their related phenotype would help both the individual management decisions of these patients and further policy-making for their diagnosis and treatment. With the advent of genetic diagnosis through sequencing techniques and these tests becoming more affordable, every Indian center is now diagnosing a fair number of these cases which used to go undiagnosed previously. This has changed the landscape of cholestatic liver disease in children where PFICs are now the most prevalent pediatric cholestatic disorder. European studies have demonstrated 2 common mutations where patients respond very well to surgical biliary diversion and have good native liver survival. These mutations have rarely been reported in India. Results from this study could thus guide appropriate decision-making based on outcome and help choose the modality of treatment for the individual patient - medical, surgical biliary diversion, or liver transplantation.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- All genetically confirmed cases of progressive familial intrahepatic cholestasis (PFIC)
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Native liver survival (in percentage) at the latest follow up in different types of PFIC
Time frame: Through study completion - average of 1 year
The proportion of patients of each subtype of PFIC who have survived with their own with their native liver till the time of last follow up
6 secondary outcomes
Describe the spectrum of genetic mutations in Indian children with familial intrahepatic cholestasis
Time frame: Through study completion - average of 1 year
Genotype-phenotype correlation in Indian children with various types of progressive familial intrahepatic cholestasis
Time frame: Through study completion - average of 1 year
Analyse the natural course after surgical biliary diversion in Indian children with various types of progressive familial intrahepatic cholestasis
Time frame: Through study completion - average of 1 year
Complications after liver transplantation in children with various types of progressive familial intrahepatic cholestasis till the time of last follow up
Time frame: Through study completion - average of 1 year
Proportion of patients surviving with native liver in uncommon variants of progressive familial intrahepatic cholestasis
Time frame: Through study completion - average of 1 year
Develop and maintain a registry of Indian patients with progressive familial intrahepatic cholestasis (Indian PFIC registry)
Time frame: Through study completion - average of 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2024.
- Background: PubMed 27746616Agarwal S, Lal BB, Rawat D, Rastogi A, Bharathy KG, Alam S. Progressive Familial Intrahepatic Cholestasis (PFIC) in Indian Children: Clinical Spectrum and Outcome. J Clin Exp Hepatol. 2016 Sep;6(3):203-208. doi: 10.1016/j.jceh.2016.05.003. Epub 2016 May 24.
- Background: PubMed 29973134Sharma A, Poddar U, Agnihotry S, Phadke SR, Yachha SK, Aggarwal R. Spectrum of genomic variations in Indian patients with progressive familial intrahepatic cholestasis. BMC Gastroenterol. 2018 Jul 4;18(1):107. doi: 10.1186/s12876-018-0835-6.
- Background: PubMed 31335238Mitra S, Das A, Thapa B, Kumar Vasishta R. Phenotype-Genotype Correlation of North Indian Progressive Familial Intrahepatic Cholestasis type2 Children Shows p.Val444Ala and p.Asn591Ser Variants and Retained BSEP Expression. Fetal Pediatr Pathol. 2020 Apr;39(2):107-123. doi: 10.1080/15513815.2019.1641860. Epub 2019 Jul 23.
- Background: PubMed 32087350van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipinski P, Czubkowski P, Rock N, Shagrani M, Broering D, Algoufi T, Mazhar N, Nicastro E, Kelly DA, Nebbia G, Arnell H, Bjorn Fischler, Hulscher JBF, Serranti D, Arikan C, Polat E, Debray D, Lacaille F, Goncalves C, Hierro L, Munoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsofi A, Calvo PL, Grabhorn E, Sturm E, van der Woerd WJ, Kamath BM, Wang JS, Li L, Durmaz O, Onal Z, Bunt TMG, Hansen BE, Verkade HJ; NAtural course and Prognosis of PFIC and Effect of biliary Diversion (NAPPED) consortium. Genotype correlates with the natural history of severe bile salt export pump deficiency. J Hepatol. 2020 Jul;73(1):84-93. doi: 10.1016/j.jhep.2020.02.007. Epub 2020 Feb 20.
- Background: PubMed 33666275van Wessel DBE, Thompson RJ, Gonzales E, Jankowska I, Shneider BL, Sokal E, Grammatikopoulos T, Kadaristiana A, Jacquemin E, Spraul A, Lipinski P, Czubkowski P, Rock N, Shagrani M, Broering D, Algoufi T, Mazhar N, Nicastro E, Kelly D, Nebbia G, Arnell H, Fischler B, Hulscher JBF, Serranti D, Arikan C, Debray D, Lacaille F, Goncalves C, Hierro L, Munoz Bartolo G, Mozer-Glassberg Y, Azaz A, Brecelj J, Dezsofi A, Luigi Calvo P, Krebs-Schmitt D, Hartleif S, van der Woerd WL, Wang JS, Li LT, Durmaz O, Kerkar N, Horby Jorgensen M, Fischer R, Jimenez-Rivera C, Alam S, Cananzi M, Laverdure N, Targa Ferreira C, Ordonez F, Wang H, Sency V, Mo Kim K, Chen HL, Carvalho E, Fabre A, Quintero Bernabeu J, Alonso EM, Sokol RJ, Suchy FJ, Loomes KM, McKiernan PJ, Rosenthal P, Turmelle Y, Rao GS, Horslen S, Kamath BM, Rogalidou M, Karnsakul WW, Hansen B, Verkade HJ; Natural Course and Prognosis of PFIC and Effect of Biliary Diversion Consortium. Impact of Genotype, Serum Bile Acids, and Surgical Biliary Diversion on Native Liver Survival in FIC1 Deficiency. Hepatology. 2021 Aug;74(2):892-906. doi: 10.1002/hep.31787. Epub 2021 Jul 13.
- Background: PubMed 35126842Alam S, Lal BB. Recent updates on progressive familial intrahepatic cholestasis types 1, 2 and 3: Outcome and therapeutic strategies. World J Hepatol. 2022 Jan 27;14(1):98-118. doi: 10.4254/wjh.v14.i1.98.
- Derived from this study: PubMed 42929328Alam S, Lal BB, Kumar K, Sibal A, Shah V, Ramakrishna SH, Shah I, Nalwalla Z, Snehavardhan P, Bavdekar A, Wadhwa N, Maria A, Gupta S, Nagral A, Dheivamani N, Deshmukh A, Verma L, Rai A, Ravindranath A, Verma SK, Shah A, Ashritha A, Suchismita A, Waikar Y, Tripathi PR, Upadhyay P, Matthai J, Malhotra S, Gautam V, Kumar V, Durga D, Bhat NK, Peethambaran M, Srikanth KP, Sivaramakrishnan VM, Sood V, Khanna R. Impact of genotype on childhood bile salt export pump deficiency in Indian Progressive Familial Intrahepatic Cholestasis registry. J Pediatr Gastroenterol Nutr. 2026 Oct 26. doi: 10.1002/jpn3.70610. Online ahead of print.
- Derived from this study: PubMed 39552453Kaur P, Lal BB, Janakiraman D, Dheivamani N, Pandey S, Bavdekar A, Shah A, Verma SK, Shah V, Maria A, Wadhwa N, Singh SK, Sood V, Khanna R, Alam S. Portal hypertension in doublecortin domain-containing protein 2 (DCDC2)-related neonatal sclerosing cholangitis. J Pediatr Gastroenterol Nutr. 2025 Jan;80(1):189-196. doi: 10.1002/jpn3.12414. Epub 2024 Nov 18.
- Individual participant dataSharing planned: No. Individual patient data would not be shared with other researchers.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Bikrant B Lal, MD, DM principal investigator
Institute of Liver and Biliary Sciences, New Delhi
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Progressive Familial Intrahepatic Cholestasis, run by Institute of Liver and Biliary Sciences, India at 1 site in India.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 21 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in India. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Bikrant B Lal, MD, DM (+919540951063, bikrant18may@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Native liver survival (in percentage) at the latest follow up in different types of PFIC (time frame: Through study completion - average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05704517, which also lists 8 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Mar 2024.