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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