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Table 3 Study design of included trials

From: Clinical trials of stem cell-based therapies for pediatric diseases: a comprehensive analysis of trials registered on ClinicalTrials.gov and the ICTRP portal site

Study type Study design Number (n) Percent (%)
Interventional    
Phases
  Phase 0 5 2.7
  Phase 1 49 26.3
  Phase 1 Phase 2 45 24.2
  Phase 2 43 23.1
  Phase 3 10 5.4
  Phase 2 Phase 3 6 3.2
  Phases 4 2 1.1
  NA 26 14.0
Allocation
  Randomized 49 26.3
  Non-randomized 40 21.5
  NA 97 52.2
Intervention model
  Single group assignment 103 55.4
  Parallel assignment 57 30.7
  Crossover assignment 7 3.8
  Factorial assignment 1 0.5
  NA 18 9.7
Masking
  None (Open Label) 131 70.4
  Single 9 4.8
  Triple 9 4.8
  Quadruple 8 4.3
  Double 5 2.7
  NA 24 12.9
Primary purpose
  Treatment 151 81.2
  Prevention 7 3.8
  Basic science 2 1.1
  Device feasibility 1 0.5
  Supportive care 1 0.5
  Other 1 0.5
  NA 23 12.4
Observational
Observational model
  Case-Only/series 6 37.5
  Cohort 3 18.8
  Other 3 18.8
  NA 4 25.0
Time perspective
  Prospective 4 25.0
  Other 2 12.5
  NA 10 62.5
  1. *NA, not available