Observational analysis of Progressive Familial Intrahepatic Cholestasis treatment
Prospective Analysis of the Treatment of Progressive Familial Intrahepatic Cholestasis (TreatFIC)
This study is looking at how different treatments for Progressive Familial Intrahepatic Cholestasis work and what happens to patients over time to help improve care for those with this rare liver disease.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 200 (estimated) |
| Sex | All |
| Sponsor | University Medical Center Groningen Academic / other |
| Locations | 1 site (Groningen) |
| Trial ID | NCT06778174 on ClinicalTrials.gov |
What this trial studies
This observational study aims to follow the natural course and prognosis of various types of Progressive Familial Intrahepatic Cholestasis (PFIC) to enhance understanding of these rare diseases. It will evaluate the efficacy of different treatments, including symptomatic therapies and surgical interventions, while also assessing the safety and complications associated with these treatments. By collecting biochemical, clinical, and surgical data, the study seeks to provide insights into disease progression and treatment outcomes for PFIC patients.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with genetically confirmed cases of PFIC.
Not a fit: Patients with suspected PFIC but lacking genetic testing data may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved management strategies and treatment options for patients with PFIC.
How similar studies have performed: While there have been studies on PFIC, this specific observational approach to understanding the natural course and treatment efficacy is relatively novel.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Groningen
- University Medical Center Groningen — Groningen, Netherlands (Recruiting)
Study contacts
- Study coordinator: Henkjan J Verkade, MD, PhD, Professor
- Email: h.j.verkade@umcg.nl; pfic@bkk.umcg.nl
- Phone: 31-50-3614147
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.