Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors

Conditions

• Diffuse Intrinsic Pontine Glioma
• Ewing Sarcoma
• Medulloblastoma
• Neuroblastoma
• Rhabdoid Tumor
• Rhabdomyosarcoma
• Solid Tumors

Eligibility Criteria

Sex

ALL

Age

2 Years - 20 Years

Healthy Volunteers

Not accepted

Interventions

• Palbociclib — DRUG
  Phase 1: Administered (oral) at 55 mg/m2, 75 mg/m2, or 40 mg/m2, or 95 mg/m2 or 115 mg/m2 on days 1-14 of a 21-day cycle Phase 1 Tumor specific cohort-Neuroblastoma and Phase 2 : Administered (oral) at 75 mg/m2 on days 1-14 of a 21-day cycle
• Temozolomide — DRUG
  Phase 1 and Phase 2: Administered at 100 mg/m2 (oral or intravenous), on days 1-5 of a 21-day cycle
• Irinotecan — DRUG
  Phase 1 and Phase 2: Administered at 50 mg/m2 (intravenous), on days 1-5 of a 21-day cycle
• Topotecan — DRUG
  Phase 1 only : Administered at 0.75 mg/m2 (intravenous), on days 1-5 of a 21-day cycle
• Cyclophosphamide — DRUG
  Phase 1 only: Administered at 250 mg/m2 (intravenous), on days 1-5 of a 21-day cycle

Study Details

A study to learn about safety and find out maximum tolerable dose of palbociclib given in combination with chemotherapy (temozolomide with irinotecan or topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors (phase 1). Neuroblastoma tumor specific cohort to further evaluate antitumor activity of palbociclib in combination with topotecan and cyclophosphamide in children, adolescents, and young adults with recurrent or refractory neuroblastoma. Phase 2 to learn about the efficacy of palbociclib in combination with irinotecan and temozolomide when compared with irinotecan and temozolomide alone in the treatment of children, adolescents, and young adults with recurrent or refractory Ewing sarcoma (EWS).

Key Dates

Start date

May 24, 2019

Status verified

Jan 2025

Primary completion

Aug 26, 2024

Completion

Oct 18, 2025

Study Design

Enrollment

128 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Phase 2 Arm A
  Palbociclib in combination with irinotecan and temozolomide.
• Experimental: Phase 1
  Palbociclib in combination with temozolomide and irinotecan and/or with topotecan and cyclophosphamide.
• Active Comparator: Phase 2 Arm B
  Irinotecan and temozolomide alone.
• Experimental: Phase 1 Tumor specific cohort - Neuroblastoma
  Palbociclib in combination with topotecan and cyclophosphamide.

Primary Outcome Measure

Phase 2 open-label, randomized: Event-free survival (EFS) based on Investigator assessment. [ Time Frame: Baseline to Month 24. ]

Locations (63)

Find similar trials in Birmingham, AL

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