PFIC Clinical Trials and Research Studies
On this page you will find information regarding ongoing external research projects. The PFIC Network does not evaluate or endorse the listed studies. This page is for your information only.
An integrated medical and research center, founded with a focus on progressive familial intrahepatic cholestasis (PFIC) and other rare or genetic liver diseases, with the goal to address unmet needs in the rare liver disease community.
CURL offers improved therapies for PFIC and other rare liver disease and the expertise to find innovative solutions for complications of the treatment and disease process. CURL offers consultation and second opinions on clinical plans of care for both adults and pediatric patients. It is dedicated to helping each patient achieve their unique care goals.
The LOGIC study is a natural history study that includes patients with PFIC and three other rare liver diseases.
A longitudinal study of genetic causes of intrahepatic cholestasis. Children and adults ages 6 months through 25 years diagnosed with Alagille Syndrome, Alpha-1 Antitrypsin deficiency, Progressive Familial Intrahepatic Cholestasis, or bile acid synthesis defects, both before and after liver transplantation. See the Clinical Research Study here.
The NAPPED study or NAtural Course and Prognosis of PFIC and Effect of Biliary Diversion.
An observational study to define the natural course of the disease.
The study is aimed to define the natural course of the disease as well as define the change in the natural course of disease in response to biliary diversion surgery and or liver transplantation. To learn more, go to the clinicaltrials.gov webpage here.
PFIC Clinical Trials
Phase 3 Clinical Trial
A Phase 3 study called MARCH (MARalixibat in CHolestasis) has been initiated to evaluate the safety and efficacy of Maralixibat as a potential novel treatment for children with PFIC.
Maralixibat is a novel, minimally-absorbed investigational medicine. It is being evaluated in rare cholestatic liver diseases for pediatric populations, including PFIC. Maralixibat works by blocking an important bile acid transport protein on the surface of the small bowel, leading to lower levels of systemic bile acids. To learn more, go to the clinicaltrials.gov webpage here.
The FDA has now approved odevixibat. It is available in the United States, UK, and Germany.
Odevixibat is a highly potent and selective inhibitor of the ileal bile acid transporter (IBAT), sometimes also referred to as the apical sodium dependent bile acid transporter (ASBT) that has minimal systemic exposure and acts locally in the gut. To learn more, go to the clinicaltrials.gov webpage here.
Phase 2 Clinical Trial
The RISE study is a clinical research study
for infants with Progressive Familial Intrahepatic Cholestasis (PFIC) or Alagille Syndrome (ALGS)
This study is designed to assess whether the investigational drug maralixibat, is safe and well tolerated in children <12 months of age with Alagille Syndrome [ALGS] or Progressive Familial Intrahepatic Cholestasis [PFIC]. To learn more, go to the clinicaltrials.gov webpage here.