HEMOPHILIA B
Clinical trials for HEMOPHILIA B explained in plain language.
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One-Shot gene therapy aims to free hemophilia patients from lifelong injections
⭐️ CURE ⭐️ OngoingThis early-stage trial is testing a single injection of gene therapy for adults with hemophilia B. The goal is to see if the treatment is safe and if it can help the body produce its own missing clotting factor, potentially reducing or eliminating the need for regular injections.…
Matched conditions: HEMOPHILIA B
Phase: PHASE1, PHASE2 • Sponsor: Baxalta now part of Shire • Aim: ⭐️ CURE ⭐️
Last updated Mar 27, 2026 12:39 UTC
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One-Shot gene therapy aims to free patients from lifelong bleeding disorder treatments
Disease control OngoingThis study is testing a one-time gene therapy for adult men with severe hemophilia B, a genetic disorder that prevents blood from clotting properly. The therapy aims to give the body long-lasting instructions to make its own clotting factor, potentially reducing or eliminating th…
Matched conditions: HEMOPHILIA B
Phase: PHASE3 • Sponsor: Pfizer • Aim: Disease control
Last updated Apr 01, 2026 14:43 UTC
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One-Time gene injection aims to free hemophilia patients from constant treatments
Disease control OngoingThis study is following people with Hemophilia B who received an experimental gene therapy to see how safe and effective it remains over the long term. It also includes a new group testing a higher dose of the same therapy. The goal is to see if a single treatment can help the bo…
Matched conditions: HEMOPHILIA B
Phase: PHASE2 • Sponsor: Pfizer • Aim: Disease control
Last updated Mar 30, 2026 14:35 UTC
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New Long-Lasting treatment tested for hemophilia b in china
Disease control OngoingThis study is testing a long-acting clotting factor medicine (rIX-FP) for Chinese males with hemophilia B. The goal is to see how well it prevents bleeding episodes when used regularly, and how it works to treat bleeds that happen anyway. Researchers will measure how long the med…
Matched conditions: HEMOPHILIA B
Phase: PHASE3 • Sponsor: CSL Behring • Aim: Disease control
Last updated Mar 30, 2026 14:33 UTC
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One-Shot gene therapy tested to fix bleeding disorder
Disease control OngoingThis early-stage study is testing the safety of a one-time gene therapy for adults with severe hemophilia B. Researchers are using a modified, harmless virus to deliver a working copy of the Factor IX gene directly to the liver, aiming to help the body produce its own clotting pr…
Matched conditions: HEMOPHILIA B
Phase: PHASE1 • Sponsor: St. Jude Children's Research Hospital • Aim: Disease control
Last updated Mar 30, 2026 14:32 UTC
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Scientists watch and wait: gathering clues for future hemophilia b treatments
Knowledge-focused OngoingThis study is gathering information, not testing a new treatment. It follows 11 males with Hemophilia B who are already receiving standard preventive therapy (FIX replacement) to control bleeding. Researchers will track their bleeding events, therapy use, quality of life, and joi…
Matched conditions: HEMOPHILIA B
Sponsor: Regeneron Pharmaceuticals • Aim: Knowledge-focused
Last updated Apr 01, 2026 14:42 UTC