Question | n (%) |
---|---|
What immune-mediated inflammatory disease(s) was the trial conducted in?a | |
Rheumatoid arthritis | 30 (30.9) |
Systemic lupus erythematosus | 10 (10.3) |
Psoriasis | 9 (9.3) |
Psoriatic arthritis | 9 (9.3) |
Juvenile idiopathic arthritis | 4 (4.1) |
Multiple sclerosis | 4 (4.1) |
Sjögren’s syndrome | 3 (3.1) |
Systemic sclerosis | 3 (3.1) |
Other (see Supplementary Materials) | 25 (25.8) |
How many treatment arms were in the trial? | |
1 | 5 (5.2) |
2 | 63 (64.9) |
3 | 13 (13.4) |
4 | 11 (11.3) |
5 | 2 (2.1) |
6 | 3 (3.1) |
What was the total planned sample size according to the sample size calculation? | Median: 214 IQR: [113, 400] Range: [10, 5400] |
How was the trial funded? | |
Industry | 73 (75.3) |
Academic | 16 (16.5) |
Mixed | 4 (4.1) |
Not reported | 3 (3.1) |
Charity | 1 (1.0) |
Was any innovative design used?b | |
Yes | 19 (19.6) |
Group-sequential design/futility interim analysis | 7 (7.2) |
Sequential multiple assignment randomised trial design | 6 (6.2) |
Bayesian methods used | 4 (4.1) |
Sample size re-estimation | 2 (2.1) |
Basket trial design | 1 (1.0) |
Use of an innovative design by trial funding | |
Industry | 16/73 (21.9) |
Other | 3/24 (12.5) |
Did the trial design report the involvement of any evidence from routinely collected data, cohorts, or biobanks? | |
Yes | 2 (2.1) |
What was the length of patient recruitment (in weeks)?c | Median: 96 IQR: [55, 120] Range: [16, 296] |
What was the primary endpoint timepoint (in weeks)?d | Median: 24 IQR: [12, 48] Range: [4, 240] |
Did the exclusion criteria explicitly include the presence of another autoimmune disease?e | |
Yes | 58 (59.8) |
Were any endpoints based on dichotomizing continuous information? | |
Primary | 66 (68.0) |
Secondary | 81 (83.5) |
How were dichotomized responder endpoints analysed?f | |
Cochrane-Mantel-Haenszel test | 27 (27.8) |
Logistic regression | 21 (21.6) |
Chi-square test | 9 (9.3) |
Cox model | 9 (9.3) |
Fisher’s exact test | 9 (9.3) |
Log rank | 5 (5.2) |
Other | 16 (16.5) |
Any high-dimensional data collected at baseline (gene expression, GWAS, synovial biopsies etc.)?g | |
Yes | 8 (8.2) |