Apatinib Mesylate Combined With IT Regimen for the Treatment of Recurrent or Refractory Pediatric Neuroblastoma: A Single-arm, Phase I/II，Multi-center, Clinical Study.

Sponsor
Sun Yat-sen University
Study ID
NCT05027386
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
5 Years - 18 Years
Healthy Volunteers
Not accepted

Interventions

  • Apatinib, Irinotecan, Temozolomide — DRUG
    In the Phase I stage, apatinib was administered using a 3+3 dose escalation design with three dose cohorts. The IT regimen was maintained at fixed doses, with patients receiving up to 6 cycles of chemotherapy. The recommended Phase II dose (RP2D) was determined from the Phase I dose-escalation phase. In the Phase II stage, the study included an apatinib combination therapy phase and an apatinib maintenance therapy phase. During the combination therapy phase, apatinib was administered at the RP2D in combination with the IT regimen (at fixed doses) for up to 6 cycles. In the maintenance therapy phase, apatinib was administered orally as a single agent at the RP2D until disease progression or intolerable toxicity occurred.

Study Details

The survival rate of recurrent and refractory pediatric neuroblastoma is low and the prognosis is poor. Apatinib mesylate is a highly selective small-molecule vasoendothelial growth factor receptor-2 (VEGFR-2) tyrosine kinase inhibitor. Apatinib mesylate has been shown to be safe and effective in recurrent or refractory pediatric neuroblastoma in Sun Yat-sen University Cancer Center. Apatinib mesylate combined with IT regimen is expected to further improve the efficacy and survival rate of recurrent or refractory pediatric neuroblastoma.

Key Dates

Start date
Aug 26, 2021
Status verified
Apr 2025
Primary completion
Feb 4, 2026
Completion
Feb 4, 2026

Study Design

Enrollment
125 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: The first dose level
    The first dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (100 mg/m²/dose infused IV 90 min) for up to six courses.
  • Experimental: The second dose level
    The second dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (100 mg/m²/dose infused IV 90 min) for up to six courses.
  • Experimental: The third dose level
    The third dose level of apatinib combined with fixed-dose 5-day courses of irinotecan (50 mg/m²/dose infused IV 90 min) plus temozolomide (100 mg/m²/dose infused IV 90 min) for up to six courses.
  • Experimental: Phase 2: The RPIID group
    Based on the Phase I stage of apatinib, the recommended phase II dose was administered in combination with the IT regimen every three weeks for up to six cycles, followed by maintenance therapy with apatinib until tumor progression recurrence or unacceptable toxicity.

Primary Outcome Measure

Objective response rate [ Time Frame: up to 6 courses of therapy, or about 6 months ]

Central Contacts

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