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

Cincinnati Children's Hospital Medical Center

Status and phase: Terminated; Phase 2

Conditions: Brain Tumors; Solid Tumors

Treatments: Drug: Amifostine

Study type: Interventional

Funder types: Other

Identifiers

NCT05167370

Amifostine

Details and patient eligibility

About

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.

Full 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.

Enrollment: 2 patients

Sex: All

Ages: Under 30 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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2 participants in 1 patient group

Amifostine

Experimental group

Treatment:

Drug: Amifostine