drugset / Trial / NCT05429502
Study of Efficacy and Safety of Ribociclib (LEE011) in Combination With Topotecan and Temozolomide (TOTEM) in Pediatric Patients With Relapsed or Refractory Neuroblastoma and Other Solid Tumors
Phase 1
Terminated
12 enrolled
Novartis Pharmaceuticals
Innovative Therapies For Children with Cancer Consortium · collab
NaSequentialOpen-labelTreatment
Summary
This is a Phase I/II study to assess the efficacy and safety of ribociclib in combination with topotecan and temozolomide (TOTEM) in pediatric patients with relapsed or refractory (r/r) neuroblastoma (NB), and other solid tumors, including medulloblastoma (MB), high-grade glioma (HGG), malignant rhabdoid tumors (MRT), and rhabdomyosarcoma (RMS).
Timeline
- Start
- 2022-12-27
- Primary completion
- 2025-02-26
- Completion
- 2025-02-26
Outcome
Outcome not reported
Stopped: “Study halted early due to high toxicity risk (from BHLRM model) in combo treatment and inability to identify recommended Phase 2 dose in initial phase (Phase 1-Part A), making continuation to subsequent phases (Phase 1-Part B and Phase 2) unfeasible.”
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ribociclib | Small molecule | 100 mg/m2 | — |
| Subject | Ribociclib | Small molecule | 200 mg/m2 | — |
| Subject | Temozolomide | Small molecule | 100 mg/m2 | — |
| Subject | Temozolomide | Small molecule | 150 mg/m2 | — |
| Subject | Topotecan | Small molecule | 0.75 mg/m2 | — |