Table 2.

Completed, active in follow-up, and recruiting GT trials in SCID, WAS, and CGD

Promoter/transgeneNCT no.StatusVector typeRef.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/transgeneNCT no.StatusVector typeRef.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.

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