SEVERE HEMOPHILIA A
Clinical trials for SEVERE HEMOPHILIA A explained in plain language.
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New hope for young hemophilia patients: jivi safety trial shows promise for fewer injections
Disease control CompletedThis study tested the safety of Jivi (BAY 94-9027), a longer-lasting clotting factor, in 36 children aged 7 to 12 with severe hemophilia A. The medicine is already approved for older patients, but this trial focused on younger kids to check for allergic reactions and whether the …
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE3 • Sponsor: Bayer • Aim: Disease control
Last updated Jun 27, 2026 08:12 UTC
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New hope for kids with hemophilia: Long-Acting clotting factor shows promise
Disease control CompletedThis trial tested a long-acting clotting factor called N8-GP in 124 young children (under 6 years old) with severe hemophilia A who had never been treated before. The goal was to see if it safely prevents and treats bleeding episodes. The study found that N8-GP helped control ble…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE3 • Sponsor: Novo Nordisk A/S • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
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New hemophilia shot shows early promise in small safety trial
Disease control CompletedThis early-phase study tested a new drug called MG1113, given as a shot under the skin, in 15 adults with severe hemophilia A or B. The main goal was to check safety and how the drug moves through the body. Participants received multiple doses to see if the drug is well-tolerated…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE1 • Sponsor: GC Biopharma Corp • Aim: Disease control
Last updated Jun 27, 2026 08:00 UTC
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One-Time gene therapy shows promise for hemophilia a
Disease control CompletedThis study tested a one-time gene therapy (valoctocogene roxaparvovec) in 22 adults with severe hemophilia A. The treatment aims to help the body produce its own clotting factor VIII, reducing the need for regular infusions. Participants also received steroids to manage immune re…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE3 • Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 07:53 UTC
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One-Time gene therapy could change life for severe hemophilia patients
Disease control CompletedThis study tested a new gene therapy (SB-525) in 13 adults with severe hemophilia A, a bleeding disorder. The goal was to see if a single infusion could safely raise the body's own clotting factor levels and reduce bleeding episodes. Participants were monitored for up to 5 years …
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE2 • Sponsor: Pfizer • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC
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New clotting factor could mean fewer shots for hemophilia a
Disease control CompletedThis early study tested a new clotting factor called efanesoctocog alfa (BIVV001) in 13 adults with severe hemophilia A. The goal was to see how long it stays in the body compared to standard and extended half-life factors. Participants received single injections of each type in …
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE1 • Sponsor: Sanofi • Aim: Disease control
Last updated Jun 26, 2026 18:13 UTC
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New clotting factor shows promise in reducing bleeds for hemophilia a patients
Disease control CompletedThis phase 3 study tested a new medicine called BIVV001 (efanesoctocog alfa) in 159 people aged 12 and older with severe hemophilia A. The goal was to see if regular injections could prevent bleeding episodes, improve joint health, and enhance quality of life. Participants receiv…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE3 • Sponsor: Bioverativ, a Sanofi company • Aim: Disease control
Last updated Jun 26, 2026 18:07 UTC
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One-Time gene therapy shot aims to ease severe hemophilia a
Disease control CompletedThis early-phase trial tested a gene therapy called BAX 888 in 4 men with severe hemophilia A. The therapy uses a harmless virus to deliver a working gene for factor VIII, a blood-clotting protein they lack. Participants received a single intravenous infusion, and the study focus…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE1, PHASE2 • Sponsor: Baxalta now part of Shire • Aim: Disease control
Last updated Jun 26, 2026 16:52 UTC
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New drug could help kids with hemophilia a bleed less
Disease control CompletedThis study tested a new drug called BIVV001 in 74 children under 12 with severe hemophilia A, a condition where blood doesn't clot properly. The drug is given through a vein and aims to prevent bleeding episodes. The trial found that the drug was safe and effective at reducing bl…
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE3 • Sponsor: Bioverativ, a Sanofi company • Aim: Disease control
Last updated Jun 26, 2026 16:27 UTC
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New hemophilia drug may last longer, reducing need for frequent shots
Knowledge-focused CompletedThis study tested a new drug called efanesoctocog alfa in 24 adults with severe hemophilia A. Researchers compared how long it stays in the body versus two existing drugs. Participants received one dose of their usual drug, then later one dose of the new drug, with blood samples …
Matched conditions: SEVERE HEMOPHILIA A
Phase: PHASE1 • Sponsor: Swedish Orphan Biovitrum • Aim: Knowledge-focused
Last updated Jun 27, 2026 14:03 UTC