NCT06778174 Prospective Analysis of the Treatment of Progressive Familial Intrahepatic Cholestasis (TreatFIC)
| NCT ID | NCT06778174 |
| Status | Recruiting |
| Phase | — |
| Sponsor | University Medical Center Groningen |
| Condition | Progressive Familial Intrahepatic Cholestasis |
| Study Type | OBSERVATIONAL |
| Enrollment | 200 participants |
| Start Date | 2023-02-09 |
| Primary Completion | 2028-02 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 200 participants in total. It began in 2023-02-09 with a primary completion date of 2028-02.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The project has the following general aims: 1. Natural course and prognosis: To prospectively follow the natural course and prognosis of the different types of PFIC, to broaden the understanding of the different very rare diseases and to allow predictions about the course of disease in different types of PFIC. 2. Efficacy: To define the course of disease in FIC patients and identify associations with different treatments (symptomatic treatments, interruption of the enterohepatic circulation by surgical or medical means and other therapies such as corrector/potentiator or exon skipping therapy. The course of disease will be characterized by biochemical, clinical and surgical parameters, including liver transplantation. 3. Safety: To define the complications associated with the different treatments (symptomatic treatments, interruption of the enterohepatic circulation by surgical or medical means and other therapies such as corrector/potentiator or exon skipping therapy, liver transplantation). Follow up will be as long as possible. 4. (Surrogate) biomarker response: Biochemical parameters will be longitudinally collected and associated with changes in treatments / course of disease. 5. Genotype-phenotype relationships: If patient numbers permit, to establish genotype-phenotype relationships for (non)responsiveness towards different treatments in patients with genetic mutations causing the different forms of FIC disease.
Eligibility Criteria
Inclusion Criteria: \- Genetically confirmed cases of a PFIC type disease: FIC1 deficiency, BSEP deficiency, MDR3 deficiency, TJP2 deficiency, FXR deficiency, SLC51A deficiency, USP53 deficiency, KIF12 deficiency, ZFYE19 deficiency, MYO5B deficiency, SEMA7A deficiency, VPS33B deficiency, PSKH1 deficiency. Exclusion Criteria: \- Cases with suspected PFIC type disease, but without genetic testing data available.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT06778174 clinical trial?
This trial is open to participants of all sexes, studying Progressive Familial Intrahepatic Cholestasis. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT06778174 currently recruiting?
Yes, NCT06778174 is actively recruiting participants. Contact the research team at h.j.verkade@umcg.nl; pfic@bkk.umcg.nl for enrollment information.
Where is the NCT06778174 trial being conducted?
This trial is being conducted at Groningen, Netherlands.
Who is sponsoring the NCT06778174 clinical trial?
NCT06778174 is sponsored by University Medical Center Groningen. The trial plans to enroll 200 participants.