Hemophilia
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: A Safety and Efficacy Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor [Phase 1, Phase 2]
Phase 1/2 safety and efficacy study of STSP-0601 in adult patients with Hemophilia A or B with inhibitors has been completed.
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: A Safety Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor [Phase 1]
Phase 1 safety study of STSP-0601 in adults with Hemophilia A or Hemophilia B with inhibitors has completed.
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST)
Psychometric validation results for the Hemophilia Functional Ability Scoring Tool (Hemo-FAST) are reported for patients with Hemophilia A a…
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
This completed clinical trial evaluated the real-world effectiveness and usage of Alprolix in patients with Hemophilia B in France.
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CLINICAL_TRIALS trial_result · 2024-09-19Clinical Trial Update: Impact on French Physician's Haemophilia Treatment Management Decision Based on Systematic Joint Examination [NA]
This entry reports the completion of NCT04133883, a trial assessing how systematic joint examination influences French physicians' managemen…
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CLINICAL_TRIALS trial_result · 2024-09-04Clinical Trial Update: Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A [Phase 1, Phase 2]
Phase 1/2 trial evaluating an encapsulated allogeneic FVIII cell therapy for Hemophilia A was terminated, with results posted in September 2…
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CLINICAL_TRIALS research · 2024-08-30Clinical Trial Update: Trial of an Exercise Intervention for Children With Haemophilia [NA]
An update on an ongoing clinical trial evaluating an exercise intervention for children with hemophilia; the study is currently recruiting.
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CLINICAL_TRIALS withdrawal · 2024-08-19Clinical Trial Update: A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV) [NA]
A prospective study evaluating seroprevalence and seroconversion of antibodies against adeno-associated virus (AAV) in Hemophilia A patients…
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CLINICAL_TRIALS trial_result · 2024-08-13Clinical Trial Update: Vitamin D Deficiency and Body Composition in the Patients With Hemophilia
A completed clinical study evaluating vitamin D deficiency and body composition in patients with hemophilia, including relationships to sarc…
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CLINICAL_TRIALS trial_result · 2024-08-09Clinical Trial Update: Immune Tolerance Induction in Haemophilia A Patients Using Wilate or Nuwiq
The trial evaluating immune tolerance induction in Hemophilia A patients using Wilate or Nuwiq has been terminated, with results posted on 2…
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CLINICAL_TRIALS trial_result · 2024-07-22Clinical Trial Update: RIXUBIS Drug Use-Result Survey (Japan)
A Japan drug-use survey for RIXUBIS in Hemophilia B with results posted.
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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 research · 2024-07-12Clinical Trial Update: Evaluation of the Inter-center Variability of the Measurement of Thrombin Generation by the ST Genesia System
Completed clinical trial evaluating inter-center variability in thrombin generation measurements using the ST Genesia system.
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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 research · 2024-06-20Clinical Trial Update: Study of Efficacy and Safety of FRSW107 in Pediatric Patients With Severe Hemophilia A [Phase 3]
Phase 3 clinical trial evaluating the efficacy and safety of FRSW107 in pediatric patients with Hemophilia A; the study is not yet recruitin…
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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 research · 2024-05-31Clinical Trial Update: Gaining Insight Into the Complexity of Pain in Patients With Haemophilia
A clinical trial conducted by Universiteit Antwerpen investigated pain in patients with Hemophilia A and Hemophilia B; the study has complet…
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CLINICAL_TRIALS research · 2024-05-24Clinical Trial Update: Conditioned Pain Modulation in Patients With Hemophilia
This clinical trial update describes a study evaluating conditioned pain modulation in patients with hemophilia. The study status is recruit…
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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)…