Survey: projected enrollment on the Inhibitor Prevention Trial and Inhibitor Eradication Trial
| Potential subject pool . | Percent needed to enroll in 6-y trial . | Sample size and power for 6-y trial . |
|---|---|---|
| Inhibitor Prevention Trial | ||
| Severe HA births | ||
| PUPs | ||
| n = 210 in 3 y (2016-2018) | ||
| n = 420 in 6-y trial | 16% of 420 = 66 | n = 66; 1 − β = 0.80, α = 0.05 |
| Inhibitor Eradication Trial | ||
| Severe HA-I | ||
| n = 12 from Prevention Trial plus | ||
| n = 274 ITI-refractory/ITI-naive | 29% of 274 = 78 | n = 90 (12+78); 1 − β = 0.80, α = 0.05 |
| Potential subject pool . | Percent needed to enroll in 6-y trial . | Sample size and power for 6-y trial . |
|---|---|---|
| Inhibitor Prevention Trial | ||
| Severe HA births | ||
| PUPs | ||
| n = 210 in 3 y (2016-2018) | ||
| n = 420 in 6-y trial | 16% of 420 = 66 | n = 66; 1 − β = 0.80, α = 0.05 |
| Inhibitor Eradication Trial | ||
| Severe HA-I | ||
| n = 12 from Prevention Trial plus | ||
| n = 274 ITI-refractory/ITI-naive | 29% of 274 = 78 | n = 90 (12+78); 1 − β = 0.80, α = 0.05 |
The projections are based on a survey of HTCs. The data indicate that the trials are possible if ≥16% of available PUPs with severe hemophilia A (HA) enroll on the Inhibitor Prevention Trial and if ≤29% of PTPs with severe HA with inhibitors (HA-I) enroll on the Inhibitor Eradication Trial.