HEMOPHILIA B
Clinical trials for HEMOPHILIA B explained in plain language.
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Monthly shot could free kids with hemophilia from frequent infusions
Disease control OngoingThis study tests fitusiran, a monthly injection, in boys aged 1 to under 12 with severe hemophilia A or B who have developed inhibitors (antibodies that block standard treatment). The goal is to find the right dose to safely lower a protein called antithrombin, which helps preven…
Matched conditions: HEMOPHILIA B
Phase: PHASE2, PHASE3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 14:01 UTC
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One-Time gene therapy could slash bleeding in hemophilia b
Disease control OngoingThis study tests a one-time gene therapy for adult men with moderate to severe hemophilia B, a bleeding disorder. The therapy delivers a working gene to help the body produce a clotting protein called Factor IX. Researchers will track how many bleeding episodes participants have …
Matched conditions: HEMOPHILIA B
Phase: PHASE3 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 13:01 UTC
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One-Shot gene therapy could free hemophilia b patients from frequent injections
Disease control OngoingThis clinical trial is testing a one-time gene therapy called BBM-H901 for people with hemophilia B, a bleeding disorder caused by low levels of clotting factor IX. The therapy uses a harmless virus to deliver a working copy of the factor IX gene to the liver, so the body can pro…
Matched conditions: HEMOPHILIA B
Phase: PHASE2, PHASE3 • Sponsor: Shanghai Xinzhi BioMed Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 12:01 UTC
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Single gene shot could tame hemophilia b bleeding
Disease control OngoingThis study tests a single intravenous dose of a gene therapy called AskBio009 in up to 30 adults with severe hemophilia B. The therapy delivers a working copy of the factor IX gene to help the body produce its own clotting factor. The main goal is to check safety, but researchers…
Matched conditions: HEMOPHILIA B
Phase: PHASE1, PHASE2 • Sponsor: Baxalta now part of Shire • Aim: Disease control
Last updated Jun 27, 2026 11:03 UTC
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New drug shows promise in reducing bleeding for hemophilia patients
Disease control OngoingThis study looks at the long-term safety and effectiveness of a drug called fitusiran in people with hemophilia A or B, including those with inhibitors that make standard treatments less effective. The main goal is to see if fitusiran is safe over time and if it reduces bleeding …
Matched conditions: HEMOPHILIA B
Phase: PHASE3 • Sponsor: Genzyme, a Sanofi Company • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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Newborn screening study aims to catch rare diseases at birth
Diagnosis OngoingThis study offers voluntary screening for newborns in North Carolina to detect a wide range of rare health conditions early. Using a small blood sample already collected at birth, the program tests for dozens of disorders, including spinal muscular atrophy, cystic fibrosis, and m…
Matched conditions: HEMOPHILIA B
Sponsor: RTI International • Aim: Diagnosis
Last updated Jul 03, 2026 00:00 UTC
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No new drug: study tracks bleeding in hemophilia b patients on usual care
Knowledge-focused OngoingThis study follows 11 males with Hemophilia B who already receive regular factor IX therapy to prevent bleeding. Researchers will collect information on bleeding events, treatment use, and quality of life over at least 6 months. No experimental treatment is given; the goal is to …
Matched conditions: HEMOPHILIA B
Sponsor: Regeneron Pharmaceuticals • Aim: Knowledge-focused
Last updated Aug 09, 2026 00:00 UTC
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Massive survey aims to map haemophilia in Asia-Pacific
Knowledge-focused ENROLLING_BY_INVITATIONThis study collects information from doctors and patient groups across Asia and the Pacific to better understand how haemophilia affects people in the region. No treatments or medications are given; instead, researchers will analyze data on how many people have the condition, how…
Matched conditions: HEMOPHILIA B
Sponsor: Novo Nordisk A/S • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:55 UTC