High Dose Chemotherapy With Amifostine and Autologous Stem Cell Transplantation for High Risk Relapsed Pediatric Solid Tumors and Brain Tumors

Phase 2

Terminated

• Conditions: Solid Tumors; Brain Tumors
• Interventions: Drug: Amifostine

• Registration Number: NCT05167370
• Lead Sponsor: Children's Hospital Medical Center, Cincinnati

Brief Summary

This is a study of amifostine to determine how effective it is in the reduction of infection in a high dose chemotherapy regimen with autologous stem cell rescue in children with high risk, relapsed or refractory pediatric solid tumors.

Detailed Description

Autologous stem cell transplant (ASCT) permits chemotherapy dose-escalation to exploit the steep dose-response of solid tumors to alkylating agents. Although ASCT regimens have activity in some high risk pediatric solid tumors, non-hematological regimen-related morbidity and mortality are major barriers to additional dose escalation. We hypothesized that the chemoprotectant amifostine (Ethyol®) would reduce the toxicity of ASCT without compromising anti-tumor efficacy. This is a study of amifostine at 1125 mg/m2 to determine the efficacy of it's chemoprotection in the reduction of bacteremia in a high dose busulfan, melphalan and thiotepa chemotherapy regimen with autologous stem cell rescue in children with high risk, relapsed or refractory pediatric solid tumors.

Study Timeline

• Study Start: 2010-12-13
• Primary Completion: 2011-04-29
• Study Completion: 2012-04-24
• Study First Submitted: 2012-08-01
• Status Verified: 2021-12
• Study First Submitted QC: 2021-12-09
• Study First Posted: 2021-12-22
• Results First Submitted: 2021-12-29
• Results First Submitted QC: 2021-12-29
• Last Update Submitted: 2021-12-29
• Results First Posted: 2022-01-25
• Last Update Posted: 2022-01-25

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 2

Inclusion Criteria

• High risk Ewing's Sarcoma Family Tumors (ESFT) including Ewing's Sarcoma, Askin's tumor, peripheral PNET
• High risk desmoplastic small round cell tumors (DSRCT)
• Relapsed Wilm's tumor, diffuse anaplastic Wilm's tumor
• High risk brain tumors including PNET/Medulloblastomas/germinomas
• Relapsed germ cell tumors
• Metastatic or relapsed rhabdoid tumors
• Other relapsed/refractory pediatric embryonal tumors
• Less than 30 years of age
• Performance >= 50%
• Cancer Diagnosis verification and staging
• Disease Response and Recovery
• Adequate Organ Function (Renal, Liver, Cardiac)

Exclusion Criteria

• Uncontrolled Infection
• Pregnancy or Breastfeeding (For Females)
• Disease Progression
• Uncontrolled Intercurrent Illness
• HIV Positive
• Receiving other Investigational Agents
• Amifostine Allergy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Amifostine	Amifostine	-

Primary Outcome Measures

Name	Time	Method
Incidence of Bacteriemia in a High Dose Busulfan, Melphalan and Thiotepa Chemotherapy Regimen With Autologous Peripheral Blood Stem Cell Rescue in Patients With High Risk and Relapsed or Refractory Pediatric Solid Tumors	3 months

Trial Locations

Locations (1)

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States