Genetic Hallmarks of Patients With Congenital Portosystemic Shunts and Portopulmonary Hypertension

NCT ID: NCT07314814

Last Updated: 2026-01-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

120 participants

Study Classification

OBSERVATIONAL

Study Start Date

2026-02-01

Study Completion Date

2030-01-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Congenital portosystemic shunt (CPSS) are rare vascular malformations causing blood from the intestines to bypass the liver and directly flow into body's general circulation. Such liver bypass can cause several health problems, one of the most severe being portopulmonary hypertension (PoPH).

The goal of this study is to identify pathogenic and potentially pathogenic genetic variants in patients who have both CPSS and PoPH. Future research will assess the contribution of these genetic variants to the development of PoPH.

The long-term goal is to use genetic information to identify patients with congenital portosystemic shunts (CPSS) or chronic liver disease who are at risk of developing PoPH to offer anticipatory management.

Children and adult patients with both CPSS and PoPH, as well as their close relatives (patient's parents and siblings) can take part in the study. Genetic variations within each family will be studied.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Portopulmonary Hypertension Pulmonary Arterial Hypertension (PAH) Congenital Portosystemic Shunt

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

FAMILY_BASED

Study Time Perspective

OTHER

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

30 families

patient + parents+ siblings

targeted gene panels analysis

Intervention Type GENETIC

The following gene panels will be analyzed : pulmonary arterial hypertension ; hereditary hemorrhagic telangiectasia ; congenital heart disease and potentially pathogenic variants in genes previously associated with PoPH in cirrhosis cohort.

whole genome analysis

Intervention Type GENETIC

Family-based identification of dominant or recessive potentially pathogenic variants.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

targeted gene panels analysis

The following gene panels will be analyzed : pulmonary arterial hypertension ; hereditary hemorrhagic telangiectasia ; congenital heart disease and potentially pathogenic variants in genes previously associated with PoPH in cirrhosis cohort.

Intervention Type GENETIC

whole genome analysis

Family-based identification of dominant or recessive potentially pathogenic variants.

Intervention Type GENETIC

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Patient is a participant to the IRCPSS with history of PoPH
* Trios composed of CPSS PoPH patients and their parents (trios are mandatory)
* Brother/sister of an enrolled patient
* Trios accept to provide biological samples (blood), sign the inform consent.
* Siblings and/or siblings' legal representatives accept to provide biological samples (blood), sign the inform consent.

Exclusion Criteria

* Trio condition is not met.
* No genuine parent-offspring trios (check for medically assisted procreation with donors, and adoption)
* For siblings, half-brothers or half-sisters are excluded, as well as adopted children, or children issued from medically assisted procreation with donors.
* Secondary portosystemic shunts
* The refusal by the patient or the patient's legal representatives to provide biological samples or agree with the proposed procedure or after voluntary withdrawal from the project.
* The refusal of one of the parents to provide biological samples or to agree with the proposed procedure or after voluntary withdrawal from the project.
Minimum Eligible Age

1 Day

Maximum Eligible Age

99 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Prof. Valérie Mc Lin

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Prof. Valérie Mc Lin

Prof. Dr. Med.

Responsibility Role SPONSOR_INVESTIGATOR

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University Hospitals Geneva / University of Geneva

Geneva, Canton of Geneva, Switzerland

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Switzerland

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Prof. Dr. med Valérie A McLIn, MD

Role: CONTACT

+41223724545

Dr. phil. nat Isabelle Schepens, PhD

Role: CONTACT

+41223724545

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2024-01698

Identifier Type: OTHER

Identifier Source: secondary_id

2024-01698

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Personalized Genomic Research
NCT01294345 COMPLETED