Phase I study of CDK8 inhibitor RVU120 in combination with everolimus in children with recurrent or progressive Group 3 or 4 medulloblastoma; MEDWAY

Condition studied

Medulloblastoma (recurrent or progressive)

Investigational medicinal product(s)

Eligibility

Primary endpoint

1. Frequency and nature of AEs, SAEs and DLTs according to CTCAE v5.0 in RVU120 monotherapy and combination therapy with everolimus 2. MTD and/or RP2D of RVU120 as a single agent and in combination with everolimus

Endpoint detail

1. PK parameters of RVU120 as a single agent and in combination with everolimus, including Cmax, AUCτ, tmax, AUC0 - ∞, t1/2 2. Ctrough of everolimus in combination with RVU120 3. Overall response rate (ORR), duration of response, progression-free survival (PFS), and overall survival (OS) in accordance with RAPNO guidelines, where applicable, stratified by molecular subgroup (G3 vs. G4) and MYC or MYCN expression in combination therapy of RVU120 and everolimus