Not applicableWithdrawnNCT06634836What this trial is testingPatient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & BWho this might be right forHemophilia AHemophilia B Pfizer
Not applicableLooking for participantsNCT04728841What this trial is testingGene Therapy for Chinese Hemophilia AWho this might be right forHemophilia AGene Therapy Institute of Hematology & Blood Diseases Hospital, China 12
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
Early research (Phase 1)Study completedNCT05265767What this trial is testingHematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia AWho this might be right forHemophilia A Christian Medical College, Vellore, India 6
Not applicableLooking for participantsNCT06147414What this trial is testingDevelopment of Non-Invasive Prenatal Diagnosis for Single Gene DisordersWho this might be right forInvasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, IncludingSickle Cell DiseaseCystic Fibrosis+12 more Assistance Publique - Hôpitaux de Paris 550
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07226206What this trial is testingA Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia AWho this might be right forHemophilia A Hoffmann-La Roche 5
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
Very early researchLooking for participantsNCT05152732What 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
Large-scale testing (Phase 3)Study completedNCT03569891What this trial is testingHOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B PatientsWho this might be right forHemophilia B CSL Behring 67
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
Testing effectiveness (Phase 2)Study completedNCT02576795What this trial is testingGene Therapy Study in Severe Haemophilia A Patients (270-201)Who this might be right forSevere Haemophilia A BioMarin Pharmaceutical 15
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
Early research (Phase 1)Active Not RecruitingNCT03818763What this trial is testingGene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia AWho this might be right forHemophilia A Medical College of Wisconsin 5
Large-scale testing (Phase 3)Active Not RecruitingNCT06224907What this trial is testingPhase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia AWho this might be right forHemophilia A BioMarin Pharmaceutical 6
Testing effectiveness (Phase 2)Study completedNCT03003533What this trial is testingA Gene Transfer Study for Hemophilia AWho this might be right forHemophilia A Spark Therapeutics, Inc. 25
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)Ended earlyNCT03520712What this trial is testingGene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5Who this might be right forHemophilia AGene TherapyClotting Disorders+1 more BioMarin Pharmaceutical 3
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 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 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