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

• Sponsor: Children's Hospital Medical Center, Cincinnati (Other)
• Overall Status: Terminated
• CT.gov ID: NCT05167370
• Collaborator: (none)
• Enrollment: 2
• Location: 1
• Arm: 1
• Actual Duration (Months): 16.4
• Patients Per Site Per Month: 0.1

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Solid Tumors; Brain Tumors	Drug: Amifostine	Phase 2

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 Design

• Study Type: Interventional
• Actual Enrollment: 2 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: High Dose Chemotherapy With Amifostine and Autologous Stem Cell Transplantation for High Risk Relapsed Pediatric Solid Tumors and Brain Tumors
• Actual Study Start Date: Dec 13, 2010
• Actual Primary Completion Date: Apr 29, 2011
• Actual Study Completion Date: Apr 24, 2012

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Amifostine	Drug: 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.; Other Names: Ethyol

Outcome Measures

Primary Outcome Measures

1. 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]

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 30 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229

Sponsors and Collaborators

• Children's Hospital Medical Center, Cincinnati

Investigators

• Principal Investigator: Sonata Jodele, MD, CCHMC

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Children's Hospital Medical Center, Cincinnati
• ClinicalTrials.gov Identifier: NCT05167370
• Other Study ID Numbers: Amifostine
• First Posted: Dec 22, 2021
• Last Update Posted: Jan 25, 2022
• Last Verified: Dec 1, 2021

• 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
• Keywords provided by Children's Hospital Medical Center, Cincinnati: Solid Tumors; Brain Tumors
• Additional relevant MeSH terms: Neoplasms; Brain Neoplasms; Amifostine

Study Results

Participant Flow

Arm/Group Title	Amifostine
Arm/Group Description	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.
Period Title: Overall Study
STARTED	2
COMPLETED	2
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Amifostine
Arm/Group Description	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.
Overall Participants	2
Age (Count of Participants)
<=18 years	2 100%
Between 18 and 65 years	0 0%
>=65 years	0 0%
Sex: Female, Male (Count of Participants)
Female	2 100%
Male	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	2 100%
More than one race	0 0%
Unknown or Not Reported	0 0%
Region of Enrollment (participants) [Number]
United States	2 100%

Outcome Measures

1. Primary Outcome

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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Amifostine
Arm/Group Description	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.
Measure Participants	2
Count of Participants [Participants]	0 0%

Adverse Events

Time Frame	Through 30 days after the end of protocol therapy
Arm/Group Title	Amifostine
Arm/Group Description	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.
All Cause Mortality
	Amifostine
	Affected / at Risk (%)	# Events
Total	0/2 (0%)
Serious Adverse Events
	Amifostine
	Affected / at Risk (%)	# Events
Total	0/2 (0%)
Other (Not Including Serious) Adverse Events
	Amifostine
	Affected / at Risk (%)	# Events
Total	2/2 (100%)
Blood and lymphatic system disorders
Febrile Neutropenia	2/2 (100%)	2
Cardiac disorders
Hypotension	2/2 (100%)	2
Gastrointestinal disorders
Mucositis	2/2 (100%)	2
Nausea	2/2 (100%)	2
Vomiting	2/2 (100%)	2
General disorders
Disease Progression	1/2 (50%)	1
Metabolism and nutrition disorders
Hypocalcemia	2/2 (100%)	2
Hypokalemia	1/2 (50%)	1
Hypophosphatemia	1/2 (50%)	1

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.