Hemophilia
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2024-07-19Clinical Trial Update: Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B [Phase 1]
Phase 1 ascending-dose study of SB-FIX, a zinc finger nuclease–based genome editing therapy for Hemophilia B, was terminated with results po…
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CLINICAL_TRIALS trial_result · 2024-07-12Clinical Trial Update: Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A [Phase 1]
A Phase 1 clinical trial update on hematopoietic stem cell transplantation gene therapy for severe Hemophilia A, sponsored by Christian Medi…
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CLINICAL_TRIALS trial_result · 2024-06-25Clinical Trial Update: Retrospective Experience Assessing the Real-World Utilization and Effectiveness of SEVENFACT®
A retrospective real-world study evaluating the utilization and effectiveness of SEVENFACT in people with hemophilia. The study has complete…
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CLINICAL_TRIALS trial_result · 2024-06-17Clinical Trial Update: ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD)
AHEAD is a completed clinical trial evaluating outcomes in Hemophilia A patients treated with ADVATE and ADYNOVI as part of an outcome datab…
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CLINICAL_TRIALS trial_result · 2024-06-14Clinical Trial Update: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Subcutaneous Emicizumab in Participants From Birth to 12 Months of Age With Hemophilia A Without Inhibitors [Phase 3]
Phase 3 trial results evaluating subcutaneous emicizumab in infants from birth to 12 months with Hemophilia A, covering efficacy, safety, ph…
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CLINICAL_TRIALS trial_result · 2024-06-05Clinical Trial Update: An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B [Phase 1, Phase 2]
Open-label extension study of investigational fitusiran in adults with moderate to severe Hemophilia A or B; trial completed with results po…
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CLINICAL_TRIALS trial_result · 2024-05-24Clinical Trial Update: Changes in Intra-articular Parameters by Doppler Ultrasound in Patients With Hemophilic Ankle Arthropathy [NA]
A completed clinical trial evaluating changes in intra-articular Doppler ultrasound parameters in patients with hemophilic ankle arthropathy…
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CLINICAL_TRIALS trial_result · 2024-05-16Clinical Trial Update: Evaluation of a Recombinant Factor IX Product, APVO101, in Previously-Treated Pediatric Patients With Hemophilia B [Phase 3]
Phase 3 clinical trial evaluating APVO101, a recombinant factor IX product, in previously-treated pediatric patients with Hemophilia B has c…
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CLINICAL_TRIALS trial_result · 2024-05-03Clinical Trial Update: Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
Long-term follow-up study update for subjects treated with Sangamo's SB-318, SB-913, or SB-FIX, evaluating ongoing outcomes in hemophilia B …
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CLINICAL_TRIALS trial_result · 2024-04-04Clinical Trial Update: Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A [Phase 3]
A Phase 3 trial of valoctocogene roxaparvovec in Hemophilia A with prophylactic steroids has completed and posted results (status COMPLETED)…
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CLINICAL_TRIALS trial_result · 2024-03-27Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B [Phase 3]
Results from a Phase 3 study of PF-06838435 gene therapy for Hemophilia B have been posted, evaluating efficacy and safety in adult males wi…
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CLINICAL_TRIALS trial_result · 2024-03-20Clinical Trial Update: Pharmacokinetic-guided Dosing of Emicizumab [Phase 4]
Phase 4 clinical trial updating pharmacokinetic-guided dosing of emicizumab in people with Hemophilia A (with and without inhibitors) across…
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CLINICAL_TRIALS trial_result · 2024-03-07Clinical Trial Update: A Study of ADVATE in People With Hemophilia A in India [Phase 4]
A Phase 4 trial of ADVATE in people with Hemophilia A in India has completed with results posted.
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CLINICAL_TRIALS trial_result · 2024-03-07Clinical Trial Update: A Clinical Study of Recombinant Human Coagulation Factor VIII for Injection in Patients With Severe Hemophilia A [Phase 3]
Phase 3 clinical study evaluating recombinant human coagulation factor VIII for injection in severe hemophilia A.
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CLINICAL_TRIALS trial_result · 2024-03-06Clinical Trial Update: Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants [Phase 3]
Phase 3 clinical trial update evaluating prophylactic emicizumab versus no prophylaxis in Hemophilia A, with results and pharmacokinetic dat…
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CLINICAL_TRIALS trial_result · 2024-03-05Clinical Trial Update: Comparison of Outcomes Between Low Dose Emicizumab and Factor VIII in Clinically Severe Hemophilia A [Phase 2, Phase 3]
A clinical trial update comparing low-dose emicizumab to Factor VIII in patients with clinically severe hemophilia A without inhibitor, focu…
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CLINICAL_TRIALS trial_result · 2024-02-29Clinical Trial Update: Combination Regimen With Sodium Valproate for Severe Hemophilia: a Single-arm, Phase 1, Pilot Trial. [Phase 1, Phase 2]
A single-arm Phase 1/2 pilot trial evaluating a combination regimen with sodium valproate for severe hemophilia.
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CLINICAL_TRIALS trial_result · 2024-02-28Clinical Trial Update: Phase 3 Clinical Project of Pegylated Recombinant Human Coagulation Factor VIII-Fc Fusion Protein [Phase 3]
Phase 3 clinical trial update for a pegylated recombinant Factor VIII-Fc fusion protein in severe Hemophilia A is currently recruiting.
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CLINICAL_TRIALS trial_result · 2024-02-23Clinical Trial Update: Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors [Phase 1, Phase 2]
A dose-finding study of SPK-8016 gene therapy in Hemophilia A has completed Phase 1/2 testing to support evaluation in patients with FVIII i…
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CLINICAL_TRIALS trial_result · 2024-02-13Clinical Trial Update: Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A [Phase 3]
A Phase 3 study of BIVV001 in pediatric patients with Hemophilia A has completed and posted results on safety, efficacy, and pharmacokinetic…
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CLINICAL_TRIALS trial_result · 2024-01-02Clinical Trial Update: Safety and Efficacy of STSP-0601 in Adult Patients With Hemophilia A or B Without Inhibitor [Phase 2]
Phase 2 trial update reporting safety and efficacy of STSP-0601 in adults with hemophilia A or B without inhibitors.
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CLINICAL_TRIALS trial_result · 2023-12-13Clinical Trial Update: An Open Label Study to Determine the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A. [Phase 3]
A Phase 3 open-label study evaluated the safety and efficacy of recombinant human Coagulation Factor VIII-Fc fusion protein for injection in…
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CLINICAL_TRIALS trial_result · 2023-12-12Clinical Trial Update: Health Related Quality of Life of Youth and Young Adults With Haemophilia A [Phase 4]
A Phase 4 clinical trial evaluated health-related quality of life in youth and young adults with Hemophilia A, completed recruitment.
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CLINICAL_TRIALS trial_result · 2023-11-28Clinical Trial Update: Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301) [Phase 3]
Phase 3, single-arm trial evaluating valoctocogene roxaparvovec for Hemophilia A has completed, with results posted on 2023-11-28.
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CLINICAL_TRIALS trial_result · 2023-11-18Clinical Trial Update: rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study [Phase 3]
Phase 3 pharmacokinetic study of rFVIII-Fc produced by AryoGen Pharmed Co. for severe hemophilia A has completed.