Table 2 Trial characteristics of the Pediatric Prevention Trial and the Harmony Trial.
Trial Name | Pediatric Prevention Trial | Harmony Trial | |||||
|---|---|---|---|---|---|---|---|
Trial number | NCT00272948 | NCT02851407 | |||||
Trial design | Phase 3, open-label, randomized study | Phase 3, open-label, randomized study | |||||
Primary outcome parameter | SOS/VOD incidence by Day 30 post-HSCT | SOS/VOD-free survival by Day 30 post-HSCT | |||||
Conducted between | January 2006 and January 2009 | August 2016 and March 2022 | |||||
Planned sample size | 290 high-risk pediatric patients (later amended to 360 patients) | 400 adult and pediatric patients (trial was stopped with 372 patients) | |||||
Sample size calculation | 25% with a reduction to 12.5% | 28% with a reduction to 16% | |||||
Assessment of SOS/VOD | Investigator | Endpoint Adjudication Committee (EPAC) | |||||
Inclusion criteria | • 2nd myeloablative HSCT • Allogeneic HSCT for leukemia > 2nd relapse • Pre-existing Liver disease • Prior Abdominal irradiation • Prior treatment with Gemtuzumab • Busulfan/Melphalan Conditioning • Osteopetrosis • Familiar macrophage activating syndrome • Adrenoleukodystrophy (ALD) | • Myeloablative conditioning (MAC) with ○ ≥2 alkylating agents ○ TBI and ≥1 alkylating agent • ≥1 of the following criteria: ○ ≥1 hepatic-related EBMT risk factor1 at screening ○ Advanced-stage neuroblastoma requiring MAC • Osteopetrosis and undergoing MAC • Primary immunodeficiency syndromes • Prior treatment with an OMAB • Class III, high-risk TDT | |||||
Outcome | Incidence of SOS/VOD | ||||||
Control Arm | Experimental Arm | Control Arm | Experimental Arm | Control Arm | |||
|---|---|---|---|---|---|---|---|
EPAC | Investigator | EPAC | Investigator | ||||
20% | 12% | 20% | 25% | 12% | 21% | 16% | |
