Safety and Efficacy of Cabazitaxel in Pediatric Patients With Refractory Solid Tumors Including Central Nervous System Tumors

Inclusion Criteria

• •Phase 1 Part (dose escalation): Participants with a histologically confirmed solid tumor including tumors of the central nervous system that was recurrent or refractory and for which no further effective standard treatment was available. All participants must had measurable disease. Participants with diffuse pontine glioma were eligible without a biopsy after evidence of progressive disease post radiation therapy.
• •Phase 2 Part (safety and activity): Participants with recurrent or refractory high grade glioma or diffuse intrinsic pontine glioma for whom no further effective therapy was available. All participants must had measurable disease. Participants with diffuse pontine glioma were eligible without a biopsy after evidence of progressive disease post radiation therapy. Participants with a grade III or grade IV glioma must had pathologic confirmation either at the time of initial diagnosis or at the time of recurrence.
• •Participants aged ≥2 years and ≤18 years
• •Participants met the body surface area (BSA) requirements to be eligible:
• •1. Minimal BSA requirements for a particular dose level;
• •2. During the Phase 1 part participants must had a BSA \<2.1 m² at the time of enrollment
• •3. During the Phase 2 part participants with a BSA ≥2.1 m² were eligible, however the actual dose of cabazitaxel for these participants were adjusted to a maximum dose calculated with (capped at) the BSA of 2.1 m²
• •Performance status by:
• •1. Lansky score ≥60 (participants ≤10 years of age)
• •2. Karnofsky score ≥60% (participants \>10 years of age) Participants who were unable to walk because of paralysis, but who were mobile in a wheelchair, were considered ambulatory for the purpose of assessing the performance score.
• •Participants must had adequate liver, renal and marrow function as defined below:
• •1. Total bilirubin ≤1.0 x the upper limit of normal (ULN) for age
• •2. AST (SGOT) and ALT (SGPT) ≤2.5 x ULN
• •3. Serum creatinine ≤1.5 x ULN for age or creatinine clearance ≥60 mL/min/1.73 m²
• •4. Absolute neutrophil count ≥1.0x10\^9 /L
• •5. Platelets ≥75x10\^9/L (transfusion independent)
• •6. Hemoglobin ≥8.0 g/dL (could be transfused)
• •Female participants of child-bearing potential must had a negative pregnancy test ≤7 days before starting cabazitaxel treatment.
• •Male and female participants of reproductive potential must agreed to use adequate contraception prior to study entry, for the duration of study participation and for 6 months following the last dose of cabazitaxel.
• •Written informed consent/assent prior to any study-specific procedures. Consent must be obtained from the participant and/or parent(s) or legal guardian(s) and the signature of at least one parent or guardian was required. Investigators also obtained assent of participants according to local, regional or national guidelines.
• •Participants must have recovered from the acute toxic effects of all prior therapy to ≤ grade 1 before entering the study.