Hemophilia
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-04-17Clinical Trial Update: Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle" [Phase 1, Phase 2]
An early-phase clinical trial update evaluating BAY 2599023 (DTX 201), a gene therapy that delivers a functional factor VIII gene using an a…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Phase 3, Open-label, Single-dose Study of CSL222 in Adolescent Male Subjects (≥ 12 to < 18 Years of Age) With Severe or Moderately Severe Hemophilia B [Phase 3]
Phase 3 open-label, single-dose study evaluating CSL222 in adolescent males (ages 12 to under 18) with severe or moderately severe Hemophili…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
This Bayer-sponsored observational study aims to evaluate how well damoctocog alfa pegol works in previously treated children with Hemophili…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: A Study to Learn More About the Safety of Damoctocog-alfa-pegol When Used in Routine Medical Care in Korean Participants With Hemophilia A
Bayer is conducting a safety study of damoctocog alfa pegol in Korean participants with Hemophilia A, evaluating safety in routine medical c…
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CLINICAL_TRIALS research · 2026-04-15Clinical Trial Update: A Study to Investigate the Safety and Effectiveness of a Coagulation Factor IX Gene Insertion Therapy (REGV131-LNP1265) in Pediatric, Adolescent and Adult Participants With Hemophilia B [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating the safety and effectiveness of REGV131-LNP1265, a coagulation factor IX gene insertion therapy, in pe…
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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 research · 2026-04-13Clinical Trial Update: A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A [Phase 3]
A Phase 3 clinical trial evaluating NXT007 versus Factor VIII prophylaxis in Hemophilia A is currently recruiting.
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A.
JOIHA is an observational study assessing the effectiveness of Jivi in preventing joint problems in adults with Hemophilia A; the study is a…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: Joint Health, Balance and Quality of Life in Adults With Hemophilia A
This clinical trial update indicates a study assessing joint health, balance, and quality of life in adults with Hemophilia A is currently r…
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CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding
A long-term safety study update for Jivi (emicizumab) in patients with Hemophilia A who are already receiving Jivi to prevent bleeds.
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CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
This clinical trial update describes evaluating the effectiveness and long-term safety of damoctocog alfa pegol in people with Hemophilia A,…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: Post Approval Observational Study to Learn More About How Safe Octocog Alfa is and How Well it Works in Patients With Severe Hemophilia A in India
A post-approval observational study in India to evaluate the safety and effectiveness of Octocog alfa (Kovaltry) in patients with severe Hem…
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CLINICAL_TRIALS research · 2026-04-08Clinical Trial Update: Central Pain Mechanisms and Clinical and Psychological Factors Associated With Pain Interference in Daily Life in Adults With Hemophilia and Hemophilic Arthropathy
This clinical trial update outlines a study evaluating central pain mechanisms and psychological factors related to pain interference in dai…
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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 research · 2026-04-07Clinical Trial Update: A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A [Phase 3]
Phase 3 clinical trial comparing NXT007 to Emicizumab prophylaxis in people with Hemophilia A; the study is currently recruiting.
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CLINICAL_TRIALS withdrawal · 2026-04-07Clinical Trial Update: POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies [Phase 4]
A Phase 4 clinical trial investigating hemostatic potential and joint health in severe Hemophilia A patients on novel replacement therapies …
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CLINICAL_TRIALS research · 2026-04-07Clinical Trial Update: Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively [Phase 3]
Phase 3 trials are evaluating the safety and effectiveness of Giroctocogene Fitelparvovec for Hemophilia A and Fidanacogene Elaparvovec for …
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A [Phase 1, Phase 2]
This is an ongoing Phase 1/2 clinical trial evaluating NXT007 in adults with Hemophilia A, assessing safety, tolerability, pharmacokinetics,…
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CLINICAL_TRIALS research · 2026-04-06Clinical Trial Update: Pharmacokinetics, Pharmacodynamics, and Safety Profile of Understudied Drugs Administered to Children Per Standard of Care (POPS)
Duke University-sponsored trial evaluating pharmacokinetics, pharmacodynamics, and safety of understudied drugs given to children as part of…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A [Phase 1]
A Phase 1 clinical trial is evaluating Emicizumab (Hemlibra) for severe Von Willebrand disease (type 3) and for patients with concomitant Vo…
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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 research · 2026-03-31Clinical Trial Update: A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A [Phase 1, Phase 2]
A phase 1/2 gene therapy study (SPK-8011QQ) in adults with severe or moderately severe Hemophilia A, sponsored by Hoffmann-La Roche, is not …
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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.