SEVERE HEMOPHILIA A
Clinical trials for SEVERE HEMOPHILIA A explained in plain language.
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New hope for babies with hemophilia: once-every-2-weeks shot cuts bleeding risk
Disease control OngoingThis study tests a medicine called emicizumab in 55 babies from birth to 12 months old with severe hemophilia A. The medicine is given as a shot under the skin every 2 weeks for a year, then less often for up to 7 more years. The goal is to see if it safely reduces the number of …
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE3 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated May 17, 2026 04:33 UTC
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New drug shows promise for hemophilia a joint health
Disease control OngoingThis study looks at how a medicine called emicizumab affects joint health, physical activity, and overall well-being in people aged 13 to 69 with moderate or severe hemophilia A. Participants receive emicizumab as a preventive treatment. The goal is to see if it reduces joint pro…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE4 • Sponsor: Hoffmann-La Roche • Aim: Disease control
Last updated May 17, 2026 04:14 UTC
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One-Dose gene therapy could change life for hemophilia patients
Disease control OngoingThis study tests a single-dose gene therapy for Chinese adults with severe hemophilia A, a bleeding disorder. The treatment uses a harmless virus to deliver a gene that helps the liver produce a missing clotting protein. Seven participants are being monitored for safety and how w…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE1, PHASE2 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated May 11, 2026 20:46 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 families will take pa…
Matched conditions: SEVERE HEMOPHILIA A
Phase: NA • Sponsor: Children's National Research Institute • Aim: Knowledge-focused
Last updated May 17, 2026 04:13 UTC