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Table 1 Design of 81 trials on genome editing in humans registered in WHO ICTRP

From: Inadequate reporting quality of registered genome editing trials: an observational study

Trial design features No. (%) of trials
Study type:
 Interventionala 78 (96.3)
 Long-term follow-upb 3 (3.7)
Primary purpose:c
 Treatmentd 63 (80.8)
 Other 2 (2.6)
 Not providede 13 (16.7)
Study phase:c
 Phase 0 3 (3.8)
 Early phase 1 4 (5.1)
 Phase 1 37 (47.4)
 Phase 2 7 (9.0)
 Phase 1/2 20 (25.6)
 Stated “not applicable” 6 (7.7)
 Not providedf 1 (1.3)
Allocation:c
 Randomized controlled trialg 6 (7.7)
 Non-randomized trial 23 (29.5)
 Not precisely stated, but single arm reported 7 (9.0)
 Stated “not applicable” 38 (48.7)
 Not provided 4 (5.1)
Intervention study model:c
 Single group 47 (60.3)
 Parallel 14 (17.9)
 Sequential 15 (19.2)
 Not provided 2 (2.6)
Masking:c
 Open-label 61 (78.2)
 Single-blindh 3 (3.8)
 Double-blindi 1 (1.3)
 Stated “not applicable” 5 (6.4)
 Not provided 8 (10.3)
Placebo:c
 Placebo comparator notedj 3 (3.8)
 Not provided 75 (96.2)
  1. Abbreviation: WHO ICTRP World Health Organization International Clinical Trial Registry Platform
  2. aAmong 78 trials, 11 (14.1%) were incorrectly classified as observational in their registration protocol (1 trial from CT.gov, NCT02867345, and 10 trials from ChiCTR), and 1 (1.3%) ChiCTR trial had “Cause/Relative factors study” stated under its study type (ChiCTR1800019378)
  3. bNCT04208529 was registered as an observational study to evaluate the long-term safety and efficacy in subjects who received CTX001 in 2 trials (NCT03655678 and NCT03745287) analyzed in this study as interventional trials, whilst other 2 LTFU trials, NCT04201782 and NCT02735083, had an interventional study type in their registration protocol and did not specify to which trials they referred to
  4. cCharacteristics related only to trials with an administered intervention (n = 78)
  5. dOne trial had “therapy” among registered terms under the trial scopes in EudraCT
  6. eAmong these 13 trials, 12 were registered in the Chinese trial registry, without the specific field for primary purpose, and 1 trial had an inappropriately registered observational type in CT.gov (NCT02867345)
  7. fCT.gov trial mentioned previously (NCT02867345). This trial is among trials without provided data for all parameters in the table signed with the superscript “c”
  8. gOne parallel ClinicalTrials.gov trial reported “randomized” under the Study Design field used for data analysis, but under the Detailed Description field also reported “non-randomized” (NCT03298828)
  9. hThree trials from ClinicalTrials.gov (NCT03525652, NCT03525782, and NCT03706326)
  10. iOne ClinicalTrials.gov trial (NCT03666871)
  11. jTwo trials were open-label (NCT02863913 and NCT02867332), and one was single-blind (NCT03525782)