NCT Number Title Status Interventions Sponsor/ Phases Funder Type
NCT04418414 Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A Not yet recruiting Gene therapy Gene therapy Expression Therapeutics, LLC Phase 1 Industry
NCT03818763 Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A Recruiting Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII Parameswaran Hari | Medical College of Wisconsin Phase 1 Other
NCT04370054 Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A adults Not yet recruiting Biological: Recombinant AAV2/6 Human Factor VIII Gene therapy Biological: Recombinant AAV2/6 Human Factor VIII Gene therapy UniQure Biopharma B.V. Phase 1 | Phase 2 Industry
NCT03001830 Gene Therapy for Haemophilia A. Recruiting AAV2/8-HLP-FVIII-V3 AAV2/8-HLP-FVIII-V3 University College, London | Medical Research Council Phase 1 Other
NCT03520712 Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5 Enrolling by invitation Valoctocogene Roxaparvovec Valoctocogene Roxaparvovec BioMarin Pharmaceutical Phase 1| Phase 2 Industry
NCT03061201 Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A Recruiting Biological: SB-525 Biological: SB-525 Pfizer Phase 2 Industry
NCT03003533 A Gene Transfer Study for Hemophilia A Recruiting Genetic: SPK-8011 Genetic: SPK-8011 Spark Therapeutics Phase 1| Phase 2 Industry
NCT03370913 Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients Active, not recruiting Biological: valoctocogene roxaparvovec Biological: valoctocogene roxaparvovec BioMarin Pharmaceutical Phase 3 Industry
NCT03370172 Safety and Dose Escalation Study of an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia A Active, not recruiting Drug: BAX 888 Drug: BAX 888 Baxalta now part of Shire Phase 1 | Phase 2 Industry
NCT02576795 Gene Therapy Study in Severe Haemiphilia A patients Active, not recruiting Genetic: BMN 270 Genetic: BMN 270 BioMarin Pharmaceutical Phase 1 | Phase 2 Industry
NCT04323098 Study to Evaluate the Efficacy and Safety of Volactocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A Not yer recruiting Valoctocogene Roxaparvovec Valoctocogene Roxaparvovec BioMarin Pharmaceutical Phase 1 | Phase 2 Industry
NCT03391974 Single-Arm Study to Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A patients at a Dose of 4E13 vg/kg Valoctocogene Roxaparvovec Valoctocogene Roxaparvovec BioMarin Pharmaceutical Phase 3 Industry
NCT03588299 Study to Test the Safety in Patients With Severe Hemophilia 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”. Recruiting Drug: BAY2599023 (DTX201) Drug: BAY2599023 (DTX201) Baye | Ultragenix pharmaceutical Phase 1 | Phase 2 Industry | Other
NCT03217032 Lentiviral FVIII Gene Therapy Not yet recruiting Biological: YUVA-GT-F801 Biological: YUVA-GT-F801 Shenzhen Geno-Immune Medical Institute Phase 1 Other
NCT03734588 Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors Active, not recruiting Genetic: SPK-8016 Genetic: SPK-8016 Spark Therapeutics Phase 1 | Phase 2 Industry
NCT03861273 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 Recruiting Biological: PF-06838435/fidanacogene elaparvovec Biological: PF-06838435/fidanacogene elaparvovec Pfizer Phase 3 Industry
NCT01687608 Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B Active, not recruiting Biological: AskBio009 Biological: AskBio009 Baxalta now part of Shire Phase 1 | Phase 2 Industry
NCT04135300 Gene Therapy for Chinese Hemophilia B Recruiting Genetic: Single dose intravenous injection of BBM-H901 Genetic: Single dose intravenous injection of BBM-H901 Institute of Hematology & Blood Diseases Hospital | East China University of Science and Technology Not Applicable Other
NCT03961243 Lentiviral FIX Gene Therapy Not yet recruiting Biological: YUVA-GT-F901 Biological: YUVA-GT-F901 Shenzhen Geno-Immune Medical Institute Phase 1 Other
NCT03641703 A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy Recruiting Biological: FLT180a Biological: FLT180a Freeline Therapeutics Phase 2| Phase 3 Industry
NCT02396342 Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B Active, not recruiting Genetic: AAV5-hFIX Genetic: AAV5-hFIX UniQure Biopharma B.V. Phase 1 | Phase 2 Industry
NCT03369444 A Factor IX Gene Therapy Study (FIX-GT) Recruiting Biological: FLT180a Biological: FLT180a University College, London Phase 1 Other
NCT03307980 Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B Recruiting Biological: SPK-9001 Biological: SPK-9001 Pfizer Phase 2 Industry
NCT04394286 A Phase 1/2 Study of SHP648, an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects Recruiting Genetic: SHP648 Genetic: SHP648 Baxalta now part of Shire Phase 1 | Phase 2 Industry
NCT02695160 Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B Active, not recruiting Biological: SB-FIX Biological: SB-FIX Sangamo Therapeutics Phase 1 Industry
NCT03569891 HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients Active, not recruiting Genetic: AAV5-hFIXco-Padua Genetic: AAV5-hFIXco-Padua UniQure Biopharma B.V. Phase 3 Industry
NCT00979238 Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B Active, not recruiting Genetic: Gene Transfer|scAAV2/8-LP1-hFIXco Genetic: Gene Transfer|scAAV2/8-LP1-hFIXco St. Jude Children’s Research Hospital | National Heart, Lung, and Blood Institute (NHLBI) | Hemophilia of Georgia, Inc. | Children’s Hospital of Philadelphia | University College, London Phase 1 Other | NIH