Large-scale testing (Phase 3)WithdrawnNCT06297486What this trial is testingStudy of a Gene Therapy Treatment for Hemophilia AWho this might be right forHemophilia A Spark Therapeutics, Inc.
Not applicableUnknownNCT05523128What this trial is testingThe Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.Who this might be right forHemophilia A Institute of Hematology & Blood Diseases Hospital, China 6
Testing effectiveness (Phase 2)Study completedNCT04684940What this trial is testingSafety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior InhibitorsWho this might be right forHemophilia A With InhibitorHemophilia A With Anti Factor VIII BioMarin Pharmaceutical 10
Large-scale testing (Phase 3)Study completedNCT03370913What this trial is testingSingle-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)Who this might be right forHemophilia A BioMarin Pharmaceutical 144
Testing effectiveness (Phase 2)Study completedNCT03061201What this trial is testingRecombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia AWho this might be right forHemophilia A Pfizer 13
Testing effectiveness (Phase 2)Study completedNCT03489291What this trial is testingDose Confirmation Trial of AAV5-hFIXco-PaduaWho this might be right forHemophilia B CSL Behring 3
Large-scale testing (Phase 3)Study completedNCT03392974What this trial is testingSingle-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kgWho this might be right forHemophilia A BioMarin Pharmaceutical 1
Testing effectiveness (Phase 2)Study completedNCT03734588What this trial is testingDose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII InhibitorsWho this might be right forAdeno-Associated Virus (AAV)Blood Coagulation DisorderBlood Coagulation Disorders, Inherited+14 more Spark Therapeutics, Inc. 4
Testing effectiveness (Phase 2)Ended earlyNCT04541628What this trial is testingSafety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia AWho this might be right forHemophilia A Eli Lilly and Company 3
Large-scale testing (Phase 3)Study completedNCT03587116What this trial is testingEvaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)Who this might be right forHemophilia BHemophilia A Pfizer 212
Testing effectiveness (Phase 2)Study completedNCT03307980What this trial is testingLong-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia BWho this might be right forHemophilia B Pfizer 21
Not applicableLooking for participantsNCT04817462What this trial is testingLiver Biopsy In Haemophilia Gene TherapyWho this might be right forHemophilia B, SevereHemophilia A, Severe University College, London 10
Not applicableNot Yet RecruitingNCT04883710What this trial is testingThe World Federation of Hemophilia Gene Therapy RegistryWho this might be right forHemophilia World Federation of Hemophilia 5,000
Early research (Phase 1)Ended earlyNCT01620801What this trial is testingHemophilia B Gene Therapy With AAV8 VectorWho this might be right forHemophilia B Spark Therapeutics, Inc. 4
Not applicableEnrolling By InvitationNCT05768386What this trial is testingA Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)Who this might be right forHemophilia A BioMarin Pharmaceutical 172
Not applicableStudy completedNCT04723680What this trial is testingAn Exploration of the Impact of Gene Therapy on the Lives of People With Haemophilia and Their FamiliesWho this might be right forHemophilia Haemnet 69
Not applicableNot Yet RecruitingNCT05932914What this trial is testingLiver Biopsy Following Gene Therapy For HemophiliaWho this might be right forHemophilia AHemophilia B St. Jude Children's Research Hospital 8
Very early researchUnknownNCT05152732What this trial is testingSafety and Tolerability of VGB-R04 in Patients With Haemophilia BWho this might be right forHemophilia B Institute of Hematology & Blood Diseases Hospital, China 3
Not applicableActive Not RecruitingNCT03432520What this trial is testingLong-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia AWho this might be right forHemophilia A Spark Therapeutics, Inc. 29
Early research (Phase 1)Not Yet RecruitingNCT04418414What this trial is testingHematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia AWho this might be right forHemophilia A Expression Therapeutics, LLC 7