Hemophilia
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CLINICAL_TRIALS trial_result · 2026-08-14Clinical Trial Results: Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B [Phase 3]
Phase 3 trial results for PF-06741086 in adults and adolescents with severe Hemophilia A or moderately to severely severe Hemophilia B; stud…
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CLINICAL_TRIALS trial_result · 2026-07-31Clinical Trial Results: Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery [Phase 3]
Phase 3 trial evaluating the safety and efficacy of FVIIa to prevent excessive bleeding in hemophilia patients with inhibitors undergoing ma…
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CLINICAL_TRIALS trial_result · 2026-06-12Clinical Trial Update: A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A [Phase 3]
Phase 3 clinical trial update for recombinant human coagulation factor VIII in patients with severe Hemophilia A.
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CLINICAL_TRIALS trial_result · 2026-04-27Clinical Trial Update: A Study to Evaluate Overall Health, Physical Activity, and Joint Outcomes in Participants With Severe or Moderate Hemophilia A Without Factor VIII Inhibitors on Emicizumab Prophylaxis [Phase 4]
Phase 4 clinical trial evaluating overall health, physical activity, and joint outcomes in people with severe or moderate Hemophilia A on em…
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CLINICAL_TRIALS trial_result · 2026-04-24Clinical Trial Update: Post-marketing Surveillance (Use Result Surveillance) With Refixia®
Completion of a post-marketing surveillance study of Refixia (recombinant factor IX-Fc fusion protein) in hemophilia B, summarizing observed…
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CLINICAL_TRIALS trial_result · 2026-04-23Clinical Trial Update: Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products [Phase 3]
Phase 3 trial evaluating recombinant single-chain Factor VIII (rVIII-SingleChain) in Chinese participants with congenital Hemophilia A who w…
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CLINICAL_TRIALS trial_result · 2026-04-14Clinical Trial Update: Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors [Phase 3]
Pfizer is conducting an open-label extension of marstacimab in Hemophilia A or Hemophilia B patients, including those with or without inhibi…
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CLINICAL_TRIALS trial_result · 2026-04-08Clinical Trial Update: Liver Biopsy Following Gene Therapy For Hemophilia
A clinical trial update indicates a liver biopsy is planned in a gene therapy study for Hemophilia A and Hemophilia B, with the trial curren…
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CLINICAL_TRIALS trial_result · 2026-04-02Clinical Trial Update: Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B [Phase 1]
Phase 1 dose-escalation study of a self-complementary AAV vector delivering factor IX for gene transfer in Hemophilia B has completed.
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CLINICAL_TRIALS trial_result · 2026-04-01Clinical Trial Update: A Study Looking at How Different Doses of Study Medicine (Inno8) Works in the Body of Healthy Men [Phase 1]
A Phase 1 study evaluating pharmacokinetics and safety of different doses of Inno8 in healthy male volunteers. The trial has completed.
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CLINICAL_TRIALS trial_result · 2026-04-01Clinical Trial Update: Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug [Phase 2, Phase 3]
This document notes an ongoing Phase 2/Phase 3 clinical trial evaluating the safety and efficacy of a gene therapy for Hemophilia B, sponsor…
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CLINICAL_TRIALS trial_result · 2026-03-30Clinical Trial Update: A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B [Phase 3]
Pfizer's Marstacimab is studied in a Phase 3 trial in pediatric patients with Hemophilia A or Hemophilia B and is currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-03-24Clinical Trial Update: Blood Flow Restriction in Improving Muscle Strength of Patients With Hemophilic Ankle Arthropathy [NA]
A completed clinical trial evaluated blood flow restriction as a method to improve muscle strength in patients with hemophilia-related ankle…
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CLINICAL_TRIALS trial_result · 2026-03-20Clinical Trial Update: Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B [Phase 1, Phase 2]
This open-label study investigates a single ascending dose of an adeno-associated virus serotype 8-based factor IX gene therapy in adults wi…
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CLINICAL_TRIALS trial_result · 2026-03-19Clinical Trial Update: Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A [Phase 3]
BioMarin's Phase 3 trial evaluating efficacy and safety outcomes of valoctocogene roxaparvovec in Japanese patients with severe Hemophilia A…
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CLINICAL_TRIALS trial_result · 2026-03-19Clinical Trial Update: A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
A 48-month trial is evaluating the long-term effectiveness of Elocta in maintaining joint health for individuals with Hemophilia A.
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CLINICAL_TRIALS trial_result · 2026-03-10Clinical Trial Update: A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency) [Phase 1]
Phase 1 clinical trial evaluating SR604 for safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy participants and in pati…
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CLINICAL_TRIALS trial_result · 2026-03-05Clinical Trial Update: 3D Ultrasound in Hemophilic Ankles [NA]
A clinical trial from The Hospital for Sick Children evaluating 3D ultrasound in hemophilic ankles is not yet recruiting.
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CLINICAL_TRIALS trial_result · 2026-03-04Clinical Trial Update: Dosage and PD Study of Eftrenonacog-alfa
Completed clinical trial update evaluating dosage and pharmacodynamics of Alprolix (eftrenonacog-alfa) for hemophilia B.
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CLINICAL_TRIALS trial_result · 2026-03-02Clinical Trial Update: Individualized Exercise Combined With Manual Therapy in Severe Hemophilia Patients With Polyarthropathy [NA]
The clinical trial evaluating individualized exercise combined with manual therapy for severe hemophilia with polyarthropathy has completed.
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CLINICAL_TRIALS trial_result · 2026-02-27Clinical Trial Update: Low-Cost Platelet-Rich Plasma for Hemarthropathy [NA]
A completed clinical trial from the University of Utah evaluating low-cost platelet-rich plasma for hemarthrosis in hemophilia patients; res…
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CLINICAL_TRIALS trial_result · 2026-02-27Clinical Trial Update: Real-World Joint Outcomes After Selective Transition to Low-Dose Emicizumab in Pediatric Hemophilia A [Phase 2, Phase 3]
An update on a pediatric Hemophilia A trial examining real-world joint outcomes after a selective transition to low-dose emicizumab. The stu…
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CLINICAL_TRIALS trial_result · 2026-02-20Clinical Trial Update: Central Sensitisation in Patients With Haemophilia and Degenerative Arthropathy
Update on a clinical trial investigating central sensitisation in patients with hemophilia; the study has completed.
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CLINICAL_TRIALS trial_result · 2026-02-20Clinical Trial Update: Incidence of Ultrasonographically Detected Articular Damage in Initially Healthy Joints of Patients With Hemophilia A Receiving Prophylactic Treatment With Emicizumab
A clinical trial update examining the incidence of ultrasound-detected articular damage in initially healthy joints of patients with Hemophi…
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CLINICAL_TRIALS trial_result · 2026-02-18Clinical Trial Update: A Long-term Study of ADYNOVI/ADYNOVATE in Participants With Haemophilia A
A long-term extension trial of ADYNOVI and ADYNOVATE in participants with Hemophilia A is currently active but not recruiting, sponsored by …