Primary biliary cholangitis 1
MONDO:0007193Also known as: PBC1, Pbc, biliary cirrhosis, primary, 1
6 clinical trials for this condition and its sub-types, 3 tagged with Primary biliary cholangitis 1 itself.
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Experimental infusion aims to fix bone marrow failure in rare telomere disease
Disease control OngoingThis early-stage trial tests a single infusion of EXG34217 in 12 people aged 12 and older who have bone marrow failure due to telomere biology disorders. The main goal is to check safety and tolerability, while also looking at changes in telomere length and blood cell counts. Bec…
Phase 1 • Sponsor: Elixirgen Therapeutics, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:02 UTC
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New pill aims to stop the relentless itch of a rare liver disease
Symptom relief OngoingThis study tests an experimental drug called volixibat for people with primary biliary cholangitis (PBC) who suffer from severe itching. The trial will involve 260 adults and compare volixibat against a placebo to see if it safely reduces itch severity and improves quality of lif…
Phase 2 • Sponsor: Mirum Pharmaceuticals, Inc. • Aim: Symptom relief
Last updated Aug 27, 2026 00:00 UTC
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Spanish databases used to map a rare liver disease
Knowledge-focused OngoingPrimary biliary cholangitis is a rare liver disease that slowly damages the bile ducts and can lead to cirrhosis or a liver transplant. Researchers are studying anonymised health records from about 3,000 adults in four Spanish databases to estimate how many people have the diseas…
Sponsor: Fundacio Privada Mon Clinic Barcelona • Aim: Knowledge-focused
Last updated Sep 17, 2026 00:00 UTC