Prospective Analysis of the Treatment of Progressive Familial Intrahepatic Cholestasis (TreatFIC)
Recruiting now
Conditions studied: Progressive Familial Intrahepatic Cholestasis
In brief
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.
Key facts
- Study ID
- NCT06778174
- Run by
- University Medical Center Groningen
- People needed
- 200
- Starts
- 2023-02-09
- Expected to finish
- 2033-12-01
- Last updated by the study team
- 2025-01-24
Who can join
Age: any. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- Cases with suspected PFIC type disease, but without genetic testing data available.
Where it is running
- University Medical Center Groningen — Groningen, Netherlands (enrolling)
Full record on ClinicalTrials.gov
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