Table 1:
Summary of Clinical Trials Reviewed
| Study | Design | Population | OS Endpoint | Treatment Groups | Consolidation Arms | FDA Review Consideration |
|---|---|---|---|---|---|---|
| E1910 | Phase 3 Randomized | Adults 30 to 70 years old with newly-diagnosed Ph-negative B-ALL | Primary | MRD− | Blin + chemo (n=112) vs chemo (n=112) | Efficacy |
| MRD+ | Blin + chemo (n=40) vs chemo (n=22) | Supportive | ||||
| 20120215 | Phase 3 Randomized | Pediatric patients 28 days to 18 years old with first relapse of Ph-negative B-ALL | Key Secondary | High-risk | Blin (n=54) vs chemo (n=57) | Efficacy |
| AALL1331 | Phase 3 Randomized | Pediatric and young adult patients 1 to < 31 years old with first relapse of Ph-negative B-ALL | Key Secondary | Low-risk | Blin + chemo (n=127) vs chemo (n=129) | Supportive |
| High- or intermediate-risk | Blin (n=107) vs chemo (n=109) | Supportive | ||||
| NL59901.078.17 | Single-Arm with Historical Control | Infant patients < 1 year old with newly-diagnosed KMT2Ar (Ph-negative) B-ALL | Secondary | KMT2Ar Infant B-ALL | Blin + chemo (n=30) vs chemo historical control (n=214) | Supportive |
Source: FDA Analysis
Abbreviations: Ph: Philadelphia chromosome; B-ALL: B cell precursor acute lymphoblastic leukemia; MRD: minimal residual disease; KMT2Ar: KMT2A-rearranged
See Supplementary Figures S1–S4 for additional details on the trial designs