Completed, active in follow-up, and recruiting GT trials in SCID, WAS, and CGD
Promoter/transgene . | NCT no. . | Status . | Vector type . | Ref. . | Center(s) . | Nonacademic sponsorship or commercialization . |
---|---|---|---|---|---|---|
ADA-deficient SCID | ||||||
γRV-ADA | NCT00599781 | Completed | γRV | 15,16 | OSR-TIGET | Marketed as Strimvelis in Europe |
γRV-ADA | NCT01279720 | Completed | γRV | 17 | GOSH | |
MND-γRV-ADA | NCT00794508 | Completed | γRV | 18,19 | UCLA | |
GCsapM-γRV-ADA | NCT00018018 | Completed | γRV | NHGRI | ||
EFS-codon optimized ADA | NCT01852071 | Completed | LV | 31 | UCLA, NHGRI | Sponsored by Orchard Therapeutics, OTL-101 |
NCT02999984 | Active, not recruiting | LV | UCL, UCLA | |||
NCT01380990 | Active, not recruiting | LV | GOSH | |||
NCT03765632 | Recruiting | LV | GOSH | |||
X-linked SCID | ||||||
EFS-IL2RG | NCT01129544 | Active, not recruiting | SIN-γRV | 14 | BCH, UCLA, CCHMC | |
NCT01410019 | Unknown | HNEM | ||||
NCT01175239 | Unknown | GOSH | ||||
EFS codon-optimized IL2RG | NCT03311503 | Recruiting | LV | BCH, UCLA | ||
NCT03601286 | Recruiting | LV | GOSH | |||
EFS codon-optimized IL2RG with insulator | NCT01306019 | Recruiting | LV | 36 | NIAID | |
NCT01512888 | Recruiting | LV | 37 | SJCRH | ||
UCSF | ||||||
SCRI | ||||||
EFS codon-optimized IL2RG with insulator | NCT03315078 | Recruiting | LV | NIAID | ||
IL2RG, not specified | NCT03217617 | Recruiting | LV | SG-IMI | ||
WAS | ||||||
1.6-kb WAS promoter-hWAS | NCT01515462 | Active, not recruiting | LV | 40 | OSR-TIGET | Sponsored by Orchard Therapeutics, OTL-103 |
NCT03837483 | Recruiting | LV | OSR-TIGET | Sponsored by Orchard Therapeutics, OTL-103 | ||
NCT01347346 | Completed | LV | 41 | HNEM | Sponsored by Genethon | |
NCT01347242 | Recruiting | LV | GOSH | Sponsored by Genethon | ||
NCT01410825 | Active, not recruiting | LV | BCH | |||
X-linked CGD | ||||||
γRV-CYBB | NCT00927134 | Completed | γRV | 53,54 | UCHZ | |
Cathepsin G/c-fes chimeric promoter codon-optimized CYBB | NCT02234934 | Recruiting | LV | UCLA, BCH, NIAID | ||
NCT02757911 | Recruiting | LV | HNEM | Sponsored by Genethon | ||
NCT01855685 | Recruiting | LV | UCL, RFH, GOSH | Sponsored by Genethon | ||
CYBB, not specified | NCT03645486 | Recruiting | LV | SG-IMI | ||
CYBB, not specified | NCT01906541 | Recruiting | SIN-γRV | UHF | ||
CYBB, not specified | NCT00778882 | Active, not recruiting | γRV | SNU | Sponsored by Helixmith |
Promoter/transgene . | NCT no. . | Status . | Vector type . | Ref. . | Center(s) . | Nonacademic sponsorship or commercialization . |
---|---|---|---|---|---|---|
ADA-deficient SCID | ||||||
γRV-ADA | NCT00599781 | Completed | γRV | 15,16 | OSR-TIGET | Marketed as Strimvelis in Europe |
γRV-ADA | NCT01279720 | Completed | γRV | 17 | GOSH | |
MND-γRV-ADA | NCT00794508 | Completed | γRV | 18,19 | UCLA | |
GCsapM-γRV-ADA | NCT00018018 | Completed | γRV | NHGRI | ||
EFS-codon optimized ADA | NCT01852071 | Completed | LV | 31 | UCLA, NHGRI | Sponsored by Orchard Therapeutics, OTL-101 |
NCT02999984 | Active, not recruiting | LV | UCL, UCLA | |||
NCT01380990 | Active, not recruiting | LV | GOSH | |||
NCT03765632 | Recruiting | LV | GOSH | |||
X-linked SCID | ||||||
EFS-IL2RG | NCT01129544 | Active, not recruiting | SIN-γRV | 14 | BCH, UCLA, CCHMC | |
NCT01410019 | Unknown | HNEM | ||||
NCT01175239 | Unknown | GOSH | ||||
EFS codon-optimized IL2RG | NCT03311503 | Recruiting | LV | BCH, UCLA | ||
NCT03601286 | Recruiting | LV | GOSH | |||
EFS codon-optimized IL2RG with insulator | NCT01306019 | Recruiting | LV | 36 | NIAID | |
NCT01512888 | Recruiting | LV | 37 | SJCRH | ||
UCSF | ||||||
SCRI | ||||||
EFS codon-optimized IL2RG with insulator | NCT03315078 | Recruiting | LV | NIAID | ||
IL2RG, not specified | NCT03217617 | Recruiting | LV | SG-IMI | ||
WAS | ||||||
1.6-kb WAS promoter-hWAS | NCT01515462 | Active, not recruiting | LV | 40 | OSR-TIGET | Sponsored by Orchard Therapeutics, OTL-103 |
NCT03837483 | Recruiting | LV | OSR-TIGET | Sponsored by Orchard Therapeutics, OTL-103 | ||
NCT01347346 | Completed | LV | 41 | HNEM | Sponsored by Genethon | |
NCT01347242 | Recruiting | LV | GOSH | Sponsored by Genethon | ||
NCT01410825 | Active, not recruiting | LV | BCH | |||
X-linked CGD | ||||||
γRV-CYBB | NCT00927134 | Completed | γRV | 53,54 | UCHZ | |
Cathepsin G/c-fes chimeric promoter codon-optimized CYBB | NCT02234934 | Recruiting | LV | UCLA, BCH, NIAID | ||
NCT02757911 | Recruiting | LV | HNEM | Sponsored by Genethon | ||
NCT01855685 | Recruiting | LV | UCL, RFH, GOSH | Sponsored by Genethon | ||
CYBB, not specified | NCT03645486 | Recruiting | LV | SG-IMI | ||
CYBB, not specified | NCT01906541 | Recruiting | SIN-γRV | UHF | ||
CYBB, not specified | NCT00778882 | Active, not recruiting | γRV | SNU | Sponsored by Helixmith |
BCH, Boston Children’s Hospital, Boston, MA; CCHMC, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH; EFS, elongation factor 1a short; γRV, gammaretrovirus; GOSH, Great Ormond Street Hospital, London, United Kingdom; HNEM, Hopital Necker Enfants Malades, Paris, France; LV, lentivirus; NHGRI, National Human Genome Research Institute, Bethesda, MD; NIAID, National Institute of Allergy and Infectious Diseases, Bethesda, MD; OSR-TIGET, Ospedale San Raffaele–Telethon Institute for Gene Therapy, Milan, Italy; RFH, Royal Free Hospital, London, United Kingdom; SCRI, Seattle Children’s Research Institute, Seattle, WA, USA; SG-IMI, Shenzhen Geno-Immune Medical Institute, Shenzhen, People’s Republic of China; SIN, self-inactivating; SJCRH, St. Jude Childhood Research Institute, Memphis, TN; SNU, Seoul National University, Seoul, Republic of Korea; UCHZ, University Children’s Hospital Zurich, Zurich, Switzerland; UCL, University College London, London, United Kingdom; UCLA, University of California, Los Angeles, CA; UCSF, University of California, San Francisco, CA; UHF, University Hospital Frankfurt, Frankfurt, Germany.