Summary of active gene therapy clinical trials for hemophilia registered on ClinicalTrials.gov
| NCT Number . | Status . | Interventions . | Sponsor/Collaborators . |
|---|---|---|---|
| Hemophilia A active gene therapy clinical trials | |||
| NCT03370172 | Recruiting | BAX 888, AAV8-co-BDD-F8 | Baxalta now part of Shire/Shire |
| NCT02576795 | Active, not recruiting | BMN 270, AAV5-co-BDD-F8 | BioMarin Pharmaceutical |
| NCT03370913 | Recruiting | Valoctocogene Roxaparvovec (BMN 270) | BioMarin Pharmaceutical |
| NCT03392974 | Recruiting | Valoctocogene Roxaparvovec (BMN 270) | BioMarin Pharmaceutical |
| NCT03520712 | Enrolling by invitation | Valoctocogene Roxaparvovec (BMN 270) | BioMarin Pharmaceutical |
| NCT03061201 | Recruiting | SB-525, AAV6-co-BDD-F8 | Sangamo Therapeutics |
| NCT03003533 | Recruiting | SPK-8011, LK03-co-BDD-F8 | Spark Therapeutics |
| NCT03432520 | Not yet recruiting | SPK-8011, LK03-co-BDD-F8 | Spark Therapeutics |
| NCT03001830 | Recruiting | AAV2/8-HLP-FVIII-V3 | University College, London/Medical Research Council |
| NCT03217032 | Not yet recruiting | YUVA-GT-F801- Lentiviral vector F8 HSC | Shenzhen Geno-Immune Medical Institute |
| Hemophilia B active gene therapy clinical trials | |||
| NCT03217032 | Not yet recruiting | YUVA-GT-F901, Lentiviral vector F9 HSC | Shenzhen Geno-Immune Medical Institute |
| NCT01687608 | Active, not recruiting | AskBio009, AAV8-coF9-Padua | Baxalta now part of Shire/Shire |
| NCT02695160 | Recruiting | SB-FIX, AAV6-zinc-finger nuclease Alb locus and AAV6-F9 donor template | Sangamo Therapeutics |
| NCT00515710 | Active, not recruiting | AAV2-FIX16 | Spark Therapeutics/Children's Hospital of Philadelphia/The Hemophilia Center of Western Pennsylvania/Royal Prince Alfred Hospital, Sydney, Australia |
| NCT02484092 | Active, not recruiting | SPK-9001, mutant AAV8-coF9-Padua | Spark Therapeutics/Pfizer |
| NCT03307980 | Recruiting | SPK-9001, mutant AAV8-coF9-Padua | Spark Therapeutics/Pfizer |
| NCT00979238 | Active, not recruiting | scAAV2/8-LP1-hFIXco | St. Jude Children's Research Hospital/National Heart, Lung, and Blood Institute/Hemophilia of Georgia, Inc./Children's Hospital of Philadelphia |
| NCT02396342 | Active, not recruiting | AAV5-hFIX (AMT-060) | UniQure Biopharma B.V. |
| NCT03489291 | Recruiting | AAV5-hFIXco-Padua (AMT-061) | UniQure Biopharma B.V. |
| NCT03369444 | Recruiting | FLT180a | University College, London |
| NCT Number . | Status . | Interventions . | Sponsor/Collaborators . |
|---|---|---|---|
| Hemophilia A active gene therapy clinical trials | |||
| NCT03370172 | Recruiting | BAX 888, AAV8-co-BDD-F8 | Baxalta now part of Shire/Shire |
| NCT02576795 | Active, not recruiting | BMN 270, AAV5-co-BDD-F8 | BioMarin Pharmaceutical |
| NCT03370913 | Recruiting | Valoctocogene Roxaparvovec (BMN 270) | BioMarin Pharmaceutical |
| NCT03392974 | Recruiting | Valoctocogene Roxaparvovec (BMN 270) | BioMarin Pharmaceutical |
| NCT03520712 | Enrolling by invitation | Valoctocogene Roxaparvovec (BMN 270) | BioMarin Pharmaceutical |
| NCT03061201 | Recruiting | SB-525, AAV6-co-BDD-F8 | Sangamo Therapeutics |
| NCT03003533 | Recruiting | SPK-8011, LK03-co-BDD-F8 | Spark Therapeutics |
| NCT03432520 | Not yet recruiting | SPK-8011, LK03-co-BDD-F8 | Spark Therapeutics |
| NCT03001830 | Recruiting | AAV2/8-HLP-FVIII-V3 | University College, London/Medical Research Council |
| NCT03217032 | Not yet recruiting | YUVA-GT-F801- Lentiviral vector F8 HSC | Shenzhen Geno-Immune Medical Institute |
| Hemophilia B active gene therapy clinical trials | |||
| NCT03217032 | Not yet recruiting | YUVA-GT-F901, Lentiviral vector F9 HSC | Shenzhen Geno-Immune Medical Institute |
| NCT01687608 | Active, not recruiting | AskBio009, AAV8-coF9-Padua | Baxalta now part of Shire/Shire |
| NCT02695160 | Recruiting | SB-FIX, AAV6-zinc-finger nuclease Alb locus and AAV6-F9 donor template | Sangamo Therapeutics |
| NCT00515710 | Active, not recruiting | AAV2-FIX16 | Spark Therapeutics/Children's Hospital of Philadelphia/The Hemophilia Center of Western Pennsylvania/Royal Prince Alfred Hospital, Sydney, Australia |
| NCT02484092 | Active, not recruiting | SPK-9001, mutant AAV8-coF9-Padua | Spark Therapeutics/Pfizer |
| NCT03307980 | Recruiting | SPK-9001, mutant AAV8-coF9-Padua | Spark Therapeutics/Pfizer |
| NCT00979238 | Active, not recruiting | scAAV2/8-LP1-hFIXco | St. Jude Children's Research Hospital/National Heart, Lung, and Blood Institute/Hemophilia of Georgia, Inc./Children's Hospital of Philadelphia |
| NCT02396342 | Active, not recruiting | AAV5-hFIX (AMT-060) | UniQure Biopharma B.V. |
| NCT03489291 | Recruiting | AAV5-hFIXco-Padua (AMT-061) | UniQure Biopharma B.V. |
| NCT03369444 | Recruiting | FLT180a | University College, London |