Clinical trial NCT06041906: International Registry of Congenital Portosystemic Shunt (IRCPSS)
Official title: International Registry of Congenital Portosystemic Shunts (IRCPSS) - A Multi-centre, Retrospective and Prospective Registry of Neonates, Children and Adults With Congenital Portosystemic Shunts
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Congenital Portosystemic Shunt
CPSS (Congenital Portosystemic Shunt)
Keywords: shunt
portosystemic
portasystemic
porto-systemic
porta-systemic
registry
liver
rare disease
Abernethy malformation
vascular malformation
Hypertension, Pulmonary
Hepatopulmonary Syndrome
Liver Neoplasms
hyperinsulinemic
hypoglycemia
hyperinsulinism
Insulin resistance
hyperandrogenism
Hepatic Encephalopathy
Hyperammonemia
Lead sponsor: University Hospital, Geneva
Sponsor class: OTHER
Collaborators: name: European Society of Pediatric Gastroenterology, Hepatology and Nutrition
name: European Association for the Study of the Liver (EASL)
name: Fondation Andrea Ferrari
First posted: 2023-09-18
Enrollment: 500
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: neonates; children; adults

Brief summary: Congenital Portosystemic Shunt (CPSS) is a rare condition important by the multiplicity and severity of associated complications.

CPSS is venous anomaly in which blood coming from the intestines only partially passes through the liver.

This leads to the accumulation of potentially toxic factors that cause systemic effects.

Complications vary among the individuals, and currently, it is challenging to predict which individuals will develop severe complications.

The IRCPSS registry is established with the aim of centralizing detailed clinical follow-up and biological information from participants around the world who suffer from Congenital Portosystemic Shunt (CPSS). A multidisciplinary consortium of experts is collaborating to enhance our understanding of the prevalence, natural history, individual risks, and physiopathology of the disease through the IRCPSS registry.


Eligibility criteria: Inclusion Criteria:

Individual with CPSS

Exclusion Criteria:

Secondary shunt without evidence of congenital shunt

Primary outcomes:
measure: Numbers of patients with spontaneous shunt closure
timeFrame: 3; 6; 12; 24 months
measure: Number of patients developing one or more complications
timeFrame: 5; 10; 20 years
measure: Number of patients undergoing preemptive closure
timeFrame: 6; 12; 24 months
Secondary outcomes:
Other outcomes:
Locations:
city: New Haven
country: United States
facility: Yale University School of Medicine
state: Connecticut
city: Chicago
country: United States
facility: Lurie Children's Hospital
state: Illinois
city: Parkville
country: Australia
facility: Royal Children's Hospital Melbourne
state: Victoria
city: Brussels
country: Belgium
facility: Universitary Clinics Saint-Luc, UCL
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Clichy
country: France
facility: Beaujon University Hospital
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre-Hôpitaux Universitaires Paris-Sud- Assistance Publique Hôpitaux de Paris.
city: Paris
country: France
facility: Hôpital Necker
city: Hannover
country: Germany
facility: Hannover Medical School
city: Munich
country: Germany
facility: Haunersche Kinderklinik - LMU Munich
city: Tuebingen
country: Germany
facility: University of Tuebingen
city: Budapest
country: Hungary
facility: First Department of Pediatrics, Semmelweis University
city: Kochi
country: India
facility: Amrita institute of Medical Sciences
state: Kerala
city: New Delhi
country: India
facility: Department of Pediatric Hepatology Institute of Liver and Biliary Sciences
city: Petach Tikvah
country: Israel
facility: Schneider Children's Medical Center of Israel
city: Bergamo
country: Italy
facility: Papa Giovanni XXIII Hospital
city: Naples
country: Italy
facility: Children's Hospital Santobono
city: Palermo
country: Italy
facility: Istituto di Ricovero e Cura a Carattere Scientifico (ISMETT)
city: Tokyo
country: Japan
facility: National Center for Child Health and Development
city: Amsterdam
country: Netherlands
facility: Academic Medical Centre
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
city: Barcelona
country: Spain
facility: Hospital Clínic de Barcelona
city: Geneva
country: Switzerland
facility: Hôpitaux Universitaires de Geneve (HUG)
state: GE
city: Yenimahalle
country: Turkey
facility: Gazi University, Faculty of Medicine
state: Ankara
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital
city: Edinburgh
country: United Kingdom
facility: Royal Hospital for Children and Young People
Study officials:
affiliation: University Hospital, Geneva
name: Valérie A Mc Lin, Prof. Dr. med.
role: STUDY_DIRECTOR
References:
citation: Hanquinet S, Morice C, Courvoisier DS, Cousin V, Anooshiravani M, Merlini L, McLin VA. Globus pallidus MR signal abnormalities in children with chronic liver disease and/or porto-systemic shunting. Eur Radiol. 2017 Oct;27(10):4064-4071. doi: 10.1007/s00330-017-4808-x. Epub 2017 Apr 6.
citation: Cudalbu C, McLin VA, Lei H, Duarte JM, Rougemont AL, Oldani G, Terraz S, Toso C, Gruetter R. The C57BL/6J mouse exhibits sporadic congenital portosystemic shunts. PLoS One. 2013 Jul 23;8(7):e69782. doi: 10.1371/journal.pone.0069782. Print 2013.
citation: Joye R, Lador F, Aggoun Y, Farhat N, Wacker J, Wildhaber BE, Vallee JP, Hachulla AL, McLin VA, Beghetti M. Outcome of paediatric portopulmonary hypertension in the modern management era: A case report of 6 patients. J Hepatol. 2021 Mar;74(3):742-747. doi: 10.1016/j.jhep.2020.11.039. Epub 2020 Dec 1.
citation: McLin V, Beghetti M, D'Antiga L, Franchi-Abella S; International Registry of Congenital Porto-Systemic Shunts (IRCPSS). Current Quandaries in the Management of Congenital Portosystemic Shunts. Liver Transpl. 2021 Aug;27(8):1212-1213. doi: 10.1002/lt.26055. Epub 2021 Jul 20. No abstract available.
citation: Korff S, Mostaguir K, Beghetti M, D'Antiga L, Debray D, Franchi-Abella S, Gonzales E, Guerin F, Hachulla AL, Lambert V, Makrythanasis P, Roduit N, Savale L, Senat MV, Spaltenstein J, van Steenbeek F, Wildhaber BE, Zwahlen M, McLin VA. International registry of congenital porto-systemic shunts: a multi-centre, retrospective and prospective registry of neonates, children and adults with congenital porto-systemic shunts. Orphanet J Rare Dis. 2022 Jul 19;17(1):284. doi: 10.1186/s13023-022-02412-8.
citation: Lambert V, Ladarre D, Fortas F, Durand P, Herve P, Gonzales E, Guerin F, Savale L, McLin VA, Ackermann O, Franchi-Abella S; International Registry of Congenital Portosystemic Shunts (IRCPSS). Cardiovascular disorders in patients with congenital portosystemic shunts: 23 years of experience in a tertiary referral centre. Arch Cardiovasc Dis. 2021 Mar;114(3):221-231. doi: 10.1016/j.acvd.2020.10.003. Epub 2020 Dec 3.
citation: McLin VA, D'Antiga L. The current pediatric perspective on type B and C hepatic encephalopathy. Anal Biochem. 2022 Apr 15;643:114576. doi: 10.1016/j.ab.2022.114576. Epub 2022 Jan 29.
citation: McLin VA, Franchi Abella S, Debray D, Guerin F, Beghetti M, Savale L, Wildhaber BE, Gonzales E; Members of the International Registry of Congenital Porto-Systemic Shunts. Congenital Portosystemic Shunts: Current Diagnosis and Management. J Pediatr Gastroenterol Nutr. 2019 May;68(5):615-622. doi: 10.1097/MPG.0000000000002263.
citation: Bahadori A, Kuhlmann B, Debray D, Franchi-Abella S, Wacker J, Beghetti M, Wildhaber BE, McLin VA, On Behalf Of The Ircpss. Presentation of Congenital Portosystemic Shunts in Children. Children (Basel). 2022 Feb 11;9(2):243. doi: 10.3390/children9020243.
citation: Guerin F, Franchi Abella S, McLin V, Ackermann O, Girard M, Cervoni JP, Savale L, Hernandez-Gea V, Valla D, Hillaire S, Dutheil D, Bureau C, Gonzales E, Plessier A. Congenital portosystemic shunts: Vascular liver diseases: Position papers from the francophone network for vascular liver diseases, the French Association for the Study of the Liver (AFEF), and ERN-rare liver. Clin Res Hepatol Gastroenterol. 2020 Sep;44(4):452-459. doi: 10.1016/j.clinre.2020.03.004. Epub 2020 Apr 9. No abstract available.
citation: Rock NM, Beghetti M, Tissot C, Willi JP, Bouhabib M, McLin VA, Maggio ABR. Reliable Detection of Intrapulmonary Shunts Using Contrast-Enhanced Echocardiography in Children With Portal Hypertension or Portosystemic Shunt. J Pediatr Gastroenterol Nutr. 2021 Jul 1;73(1):73-79. doi: 10.1097/MPG.0000000000003079.