High Dose Chemotherapy With Amifostine and Autologous Stem Cell Transplantation for High Risk Relapsed Pediatric Solid Tumors and Brain Tumors
Primary Purpose
Solid Tumors, Brain Tumors
Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Amifostine
Sponsored by
About this trial
This is an interventional treatment trial for Solid Tumors focused on measuring Solid Tumors, Brain Tumors
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
Sites / Locations
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Amifostine
Arm Description
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
Full Information
NCT ID
NCT05167370
First Posted
August 1, 2012
Last Updated
December 29, 2021
Sponsor
Children's Hospital Medical Center, Cincinnati
1. Study Identification
Unique Protocol Identification Number
NCT05167370
Brief Title
High Dose Chemotherapy With Amifostine and Autologous Stem Cell Transplantation for High Risk Relapsed Pediatric Solid Tumors and Brain Tumors
Official Title
High Dose Chemotherapy With Amifostine and Autologous Stem Cell Transplantation for High Risk Relapsed Pediatric Solid Tumors and Brain Tumors
Study Type
Interventional
2. Study Status
Record Verification Date
December 2021
Overall Recruitment Status
Terminated
Why Stopped
The study was terminated as recommended by the DSMB due to inability to meet enrollment goals within the time frame allotted (target of 25 patients over 5 years).
Study Start Date
December 13, 2010 (Actual)
Primary Completion Date
April 29, 2011 (Actual)
Study Completion Date
April 24, 2012 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Solid Tumors, Brain Tumors
Keywords
Solid Tumors, Brain Tumors
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Amifostine
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Amifostine
Other Intervention Name(s)
Ethyol
Intervention Description
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.
Primary Outcome Measure Information:
Title
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
Time Frame
3 months
10. Eligibility
Sex
All
Maximum Age & Unit of Time
30 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sonata Jodele, MD
Organizational Affiliation
CCHMC
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
12. IPD Sharing Statement
Learn more about this trial
High Dose Chemotherapy With Amifostine and Autologous Stem Cell Transplantation for High Risk Relapsed Pediatric Solid Tumors and Brain Tumors
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