SEVERE HEMOPHILIA A
Clinical trials for SEVERE HEMOPHILIA A explained in plain language.
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One-Time gene shot could free hemophilia patients from frequent infusions
Disease control OngoingThis study tests a one-time gene therapy called GS1191-0445 in 7 Chinese men with severe hemophilia A. The treatment uses a harmless virus to deliver a working gene to the liver, so the body can produce its own clotting factor VIII. The goal is to see if it is safe and reduces th…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE1, PHASE2 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated May 26, 2026 09:38 UTC
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New shot helps hemophilia patients move better and bleed less
Disease control OngoingThis study looks at how a medicine called emicizumab affects the joints, physical activity, and overall health of people aged 13 to 69 with moderate or severe hemophilia A. Participants receive emicizumab as a shot to prevent bleeding episodes. The goal is to see if it improves j…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE4 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated May 26, 2026 09:28 UTC
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New hope for tiniest hemophilia patients: once-every-2-weeks shot cuts bleeds
Disease control OngoingThis study tests a medicine called emicizumab in babies from birth to 12 months old who have severe hemophilia A. The goal is to see if the medicine can prevent bleeding episodes. Babies receive a shot every 2 weeks for a year, then continue treatment for up to 7 more years. This…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE3 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated May 26, 2026 09:27 UTC
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New study aims to support families of children with rare diseases
Knowledge-focused ENROLLING_BY_INVITATIONThis study looks at the unmet palliative care needs of children with rare diseases and their family caregivers. Researchers will test a program called FACE-Rare to see if it helps families prepare for medical decisions and improves quality of life. About 480 children and their ca…
Matched conditions: SEVERE HEMOPHILIA A
Phase: NA • Sponsor: Children's National Research Institute • Aim: Knowledge-focused
Last updated May 22, 2026 13:51 UTC