Donor Stem Cell Transplant After Chemotherapy for the Treatment of Recurrent or Refractory High-Risk Solid Tumors in Pediatric and Adolescent-Young Adults

Study Results

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

1 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-08-19

Study Completion Date

2024-08-26

Brief Summary

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This phase II trial investigates side effects and how well donor stem cell transplant after chemotherapy works in treating pediatric and adolescent-young adults with high-risk solid tumor that has come back (recurrent) or does not respond to treatment (refractory). Chemotherapy drugs, such as fludarabine, thiotepa, etoposide, melphalan, and rabbit anti-thymocyte globulin work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving chemotherapy before a donor stem cell transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When the healthy stem cells from a donor are infused into a patient, they may help the patient's bone marrow make more healthy cells and platelets and may help destroy any remaining cancer cells.

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Detailed Description

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PRIMARY OBJECTIVE:

I. To assess tolerability of allogeneic hematopoietic stem cell transplantation (HCT) for patients with chemo-responsive recurrent/refractory solid tumors as defined by transplant-related mortality (TRM) at day 30 and the rate of grade III or higher organ toxicity (Bearman Regimen-Related Toxicities Scale) attributable to conditioning occurring within 30 days.

SECONDARY OBJECTIVES:

I. Assess median time to platelet and neutrophil engraftment. II. Assess incidence of acute graft-versus-host disease (aGVHD) by day 100. III. Assess incidence of chronic GVHD (cGVHD) at day 100 and one year. IV. Assess rate of grade II organ toxicity through day 100. V. Assess rate of graft failure (primary and secondary) through day 100. VI. Assess rate of infectious complications through day 100. VII. Assess progression free survival (PFS) at day 100,180 and 365. VIII. Assess cumulative incidence of relapse, overall survival (OS) at 100 days and 1 year.

OUTLINE:

CONDITIONING REGIMEN: Patients receive thiotepa intravenously (IV) over 2-4 hours and etoposide IV over 60 minutes on days -8 to -6, melphalan IV over 20 minutes on days -5 and -4, and fludarabine phosphate IV over 1 hour on days -5 to -3 in the absence of disease progression or unacceptable toxicity. Patients receiving umbilical cord transplant also receive rabbit anti-thymocyte globulin IV on days -4 and -3.

TRANSPLANT: Patients undergo HSCT on day 0.

GVHD PROPHYLAXIS: Beginning day -2, patients receive tacrolimus or cyclosporine IV continuously until able to receive orally (PO). Patients continue tacrolimus or cyclosporine PO to day 60 and tapered to day 100. Patients also receive mycophenolate mofetil PO or IV every 8 hours until day 40 and tapered to day 90.

After completion of HSCT, patients are followed up for up to 1 year.

Conditions

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Desmoplastic Small Round Cell Tumor Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor Recurrent Desmoplastic Small Round Cell Tumor Recurrent Malignant Peripheral Nerve Sheath Tumor Recurrent Malignant Solid Neoplasm Recurrent Neuroblastoma Recurrent Rhabdomyosarcoma Refractory Desmoplastic Small Round Cell Tumor Refractory Malignant Peripheral Nerve Sheath Tumor Refractory Malignant Solid Neoplasm Refractory Neuroblastoma Refractory Rhabdomyosarcoma

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Intervention Type DRUG

Given IV

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Allogeneic Hematopoietic Cell Transplantation Allogeneic Stem Cell Transplantation HSC HSCT Stem Cell Transplantation, Allogeneic 27-400 Ciclosporin CsA Cyclosporin Cyclosporin A Cyclosporine Modified Gengraf Neoral OL 27-400 Sandimmune SangCya Demethyl Epipodophyllotoxin Ethylidine Glucoside EPEG Lastet Toposar Vepesid VP 16 VP 16-213 VP-16 VP-16-213 VP16 2-F-ara-AMP 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)- Beneflur Fludara SH T 586 Anti-Thymocyte Globulin Rabbit Grafalon Rabbit Anti-Human Thymocyte Globulin (RATG) Rabbit Anti-Thymocyte Globulin Rabbit Antithymocyte Globulin Rabbit ATG rATG Thymoglobulin Alanine Nitrogen Mustard CB-3025 L-PAM L-Phenylalanine Mustard L-Sarcolysin L-Sarcolysin Phenylalanine mustard L-Sarcolysine Melphalanum Phenylalanine Mustard Phenylalanine Nitrogen Mustard Sarcoclorin Sarkolysin WR-19813 CellCept MMF FK 506 Fujimycin Hecoria Prograf Protopic 1,1'',1''''-Phosphinothioylidynetrisaziridine Girostan N,N'', N''''-Triethylenethiophosphoramide Oncotiotepa STEPA Tepadina TESPA Tespamin Tespamine Thio-Tepa Thiofosfamide Thiofozil Thiophosphamide Thiophosphoramide Thiotef Tifosyl TIO TEF Tio-tef Triethylene Thiophosphoramide Triethylenethiophosphoramide Tris(1-aziridinyl)phosphine sulfide TSPA WR 45312

Eligibility Criteria

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Inclusion Criteria

* Pathological criteria, including malignant recurrent/refractory solid tumors. This would include:

* Ewing's/peripheral primitive neuroectodermal tumor (PNET)
* Malignant peripheral nerve sheath tumor, neurofibrosarcoma
* Rhabdomyosarcoma
* Neuroblastoma (patients who are ineligible for tandem autologous transplant or who are at least 3 months post autologous HCT)
* Desmoplastic small round cell tumor (DSRCT)- both new diagnoses as well as recurrent/refractory disease
* Patients must have chemo-responsive disease, defined as; 30% or greater decrease in the tumor target lesions when compared to its pre-treatment evaluation. Patients with complete response will be eligible to participate
* Available suitable HCT donor
* Creatinine clearance or glomerular filtration rate (GFR) \>= 50 ml/min/1.73m\^2, and not requiring dialysis
* Diffusing capacity of lung for carbon monoxide (DLCO) (corrected for hemoglobin) \>= 50% predicted. If unable to perform pulmonary function tests, then oxygen (O2) saturation \>= 92% in room air
* Bilirubin =\< 3 x upper limit of normal (ULN) and alanine aminotransferase (ALT) and aspartate aminotransferase (AST) =\< 5 x for age (with the exception of isolated hyperbilirubinemia due to Gilbert's syndrome)
* DONOR: Matched related donor bone marrow (10 of 10 HLA alleles \[HLA-A, B, C, DR, and DQ\]. Matched related donor peripheral blood stem cell (PBSC) is allowed only if collection of bone marrow (BM) is not available or refused by parents/donor
* DONOR: Matched allogeneic umbilical cord blood (UCB): related

* High-resolution matching at A,B, DRB1 (minimum 4/6)
* KIR major histocompatibility complex (MHC) class 1 preferential mismatch (minimum 4/6)
* DONOR: Matched allogeneic umbilical cord blood: unrelated

* High-resolution matching at A,B, DRB1 (minimum 4/6) •\*KIR MHC class 1 preferential mismatch (minimum 4/6)

Exclusion Criteria

* Lack of histocompatible suitable related donor/ graft source
* End-organ failure that precludes the ability to tolerate the transplant procedure, including conditioning regimen
* Renal failure requiring dialysis
* Congenital heart disease resulting in congestive heart failure
* Ventilatory failure: requires invasive mechanical ventilation
* Human immunodeficiency virus (HIV) infection
* A female of reproductive potential who is pregnant, planning to become pregnant during the study, or is nursing a child

Maximum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No