HEMOPHILIA B
Clinical trials for HEMOPHILIA B explained in plain language.
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Gene therapy trial offers hope for hemophilia b patients
Disease control CompletedThis early-stage study tested a new gene therapy for adults with severe hemophilia B, a bleeding disorder caused by a faulty gene. The treatment uses a harmless virus to deliver a working copy of the factor IX gene into the body. The main goal was to check safety in 14 participan…
Matched conditions: HEMOPHILIA B
Phase: PHASE1 • Sponsor: St. Jude Children's Research Hospital • Aim: Disease control
Last updated May 17, 2026 02:16 UTC
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Real-World study reveals how alprolix controls bleeding in hemophilia b
Disease control CompletedThis study followed 151 people with hemophilia B for 24 months to see how well Alprolix works in real-world settings. Researchers tracked bleeding episodes, how often the medicine was used, and the amount needed. The goal was to understand the drug's effectiveness and usage patte…
Matched conditions: HEMOPHILIA B
Sponsor: Swedish Orphan Biovitrum • Aim: Disease control
Last updated May 13, 2026 16:00 UTC
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One-Time gene shot could free hemophilia b patients from frequent infusions
Disease control CompletedThis phase 3 trial tested a one-time gene therapy (AMT-061) in 67 adults with severe or moderately severe hemophilia B. The treatment uses a harmless virus to deliver a working gene for factor IX, a clotting protein. The goal was to reduce bleeding episodes and the need for regul…
Matched conditions: HEMOPHILIA B
Phase: PHASE3 • Sponsor: CSL Behring • Aim: Disease control
Last updated May 13, 2026 15:58 UTC
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Hemophilia Patients' bleeding patterns tracked ahead of gene therapy trials
Knowledge-focused CompletedThis study gathered information on how often adults with moderate to severe hemophilia A or B experience bleeding while on their usual clotting factor treatment. The goal was to create a baseline to compare against future gene therapy results. About 212 participants took part, an…
Matched conditions: HEMOPHILIA B
Phase: PHASE3 • Sponsor: Pfizer • Aim: Knowledge-focused
Last updated May 17, 2026 02:04 UTC
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Global survey reveals what hemophilia b patients really know about gene therapy
Knowledge-focused CompletedThis study interviewed 150 people with hemophilia B, their caregivers, and healthcare workers from around the world to find out what they know and think about gene therapy. The goal is to create better educational materials and improve the consent process, especially in countries…
Matched conditions: HEMOPHILIA B
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated May 17, 2026 02:03 UTC