Table 4

General characteristics of clinical trials in personalised medicine

Trial designClinical trial*Recruitment status of clinical trial as for March 2021Disease areaPhases
OngoingCompletednf†Unknown‡CancerNo cancerIIII/IIIIIIIVn/a§nf†
n=131
(%)
n=63
(%)
n=60
(%)
n=1
(%)
n=7
(%)
n=113
(%)
n=18 (%)n=75
(%)
n=13
(%)
n=28 (%)n=2
(%)
n=12
(%)
n=1
(%)
Adaptive biomarker design0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)
Adaptive parallel Simon two-stage design1 (0.8)0 (0)1 (1.7)0 (0)0 (0)1 (0.9)0 (0)1 (1.3)0 (0)0 (0)0 (0)0 (0)0 (0)
Adaptive enrichment design4 (3.1)0 (0)4 (6.7)0 (0)0 (0)0 (0)4 (22.2)0 (0)0 (0)4 (14.3)0 (0)0 (0)0 (0)
Adaptive signature design0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)
Adaptive strategy for biomarker with measurement error1 (0.8)1 (1.6)0 (0)0 (0)0 (0)1 (0.9)0 (0)0 (0)0 (0)0 (0)0 (0)1 (8.3)0 (0)
Basket35 (26.7)19 (30.2)13 (21.7)0 (0)3 (42.9)34 (30.1)1 (5.6)32 (42.7)0 (0)2 (7.1)0 (0)1 (8.3)0 (0)
Basket of basket design1 (0.8)1 (1.6)0 (0)0 (0)0 (0)1 (0.9)0 (0)1 (1.3)0 (0)0 (0)0 (0)0 (0)0 (0)
Biomarker strategy design with biomarker assessment in the control arm3 (2.3)0 (0)3 (5.0)0 (0)0 (0)2 (1.8)1 (5.6)0 (0)0 (0)2 (7.1)1 (50.0)0 (0)0 (0)
Biomarker strategy design with treatment randomisation in the control arm0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)
Biomarker strategy design without biomarker assessment in the control arm4 (3.1)2 (3.2)2 (3.3)0 (0)0 (0)0 (0)4 (22.2)0 (0)0 (0)0 (0)0 (0)4 (33.3)0 (0)
Hybrid design1 (0.8)0 (0)1 (1.7)0 (0)0 (0)1 (0.9)0 (0)0 (0)0 (0)1 (3.6)0 (0)0 (0)0 (0)
Marker stratified design15 (11.5)0 (0)14 (23.3)1 (100)0 (0)15 (13.3)0 (0)0 (0)0 (0)14 (50.0)0 (0)0 (0)1 (100.0)
Modified biomarker strategy design3 (2.3)0 (0)2 (3.3)0 (0)1 (14.3)3 (2.7)0 (0)2 (2.7)0 (0)1 (3.6)0 (0)0 (0)0 (0)
Multiarm multistage design7 (5.3)3 (4.8)3 (5.0)0 (0)1 (14.3)5 (4.4)2 (11.1)4 (5.3)2 (15.4)1 (3.6)0 (0)0 (0)0 (0)
Outcome-based adaptive randomisation design4 (3.1)2 (3.2)2 (3.3)0 (0)0 (0)3 (2.7)1 (5.6)2 (2.7)1 (7.7)1 (3.6)0 (0)0 (0)0 (0)
Platform18 (13.7)13 (20.6)4 (6.7)0 (0)1 (14.3)14 (12.4)4 (22.2)11 (14.7)4 (30.8)1 (3.6)1 (50.0)1 (8.3)
Reverse marker biased strategy0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)0 (0)
Sequential multiple assignment randomised trial1 (0.8)0 (0)1 (1.7)0 (0)0 (0)0 (0)1 (5.6)0 (0)0 (0)0 (0)0 (0)1 (8.3)0 (0)
Tandem two-stage design1 (0.8)0 (0)1 (1.7)0 (0)0 (0)1 (0.9)0 (0)1 (1.3)0 (0)0 (0)0 (0)0 (0)0 (0)
Umbrella30 (22.9)20 (31.7)9 (15.0)0 (0)1 (14.3)30 (26.5)0 (0)19 (25.3)6 (46.2)1 (3.6)0 (0)4 (33.3)0 (0)
Umbrella-basket hybrid2 (1.5)2 (3.2)0 (0)0 (0)0 (0)2 (1.8)0 (0)2 (2.7)0 (0)0 (0)0 (0)0 (0)0 (0)
  • *If the same clinical trial was labelled differently across articles, we considered the trial as example of the design reported in the paper. For instance, I-SPY 2 has been labelled as outcome-based adaptive randomisation,15 platform36 or umbrella design37 and it was considered as an example for each of those trial designs.

  • †Not found.

  • ‡Unknown is used to indicate a trial status that has not been verified within the past 2 years on the ClinicalTrials.gov website.

  • §Not applicable is used on the ClinicalTrials.gov website to describe trials without FDA-defined phases including trials of devices or behavioural interventions.

  • FDA, U.S. Food and Drug Administration; n/a, Not applicable; nf, Not found.