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

NCT ID: NCT05167370

Last Updated: 2022-01-25

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 2 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-12-13
• Study Completion Date: 2012-04-24

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.

Conditions

Solid Tumors; Brain Tumors

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Amifostine

Group Type: EXPERIMENTAL

Amifostine

Intervention Type: DRUG

Amifostine (Ethyol) 1125 mg/m2 will be given intravenously daily over 5 minutes starting 30 minutes prior to chemotherapy (melphalan or thiotepa) on days -5, -4, -3, -2, and on day -1 twenty four hours after prior dose of amifostine.

Interventions

Amifostine

Amifostine (Ethyol) 1125 mg/m2 will be given intravenously daily over 5 minutes starting 30 minutes prior to chemotherapy (melphalan or thiotepa) on days -5, -4, -3, -2, and on day -1 twenty four hours after prior dose of amifostine.

Intervention Type: DRUG

Other Intervention Names

Ethyol

Eligibility Criteria

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

• Maximum Eligible Age: 30 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Sonata Jodele, MD

Role: PRINCIPAL_INVESTIGATOR

CCHMC

Locations

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Countries

United States

Other Identifiers

Amifostine

Identifier Type: -

Identifier Source: org_study_id