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 |