A Phase I Study to Examine the Toxicity of Allogeneic Stem Cell Transplantation for Relapsed or Therapy Refractory Ewing Sarcoma

Phase 1

Withdrawn

• Conditions: Ewing Sarcoma
• Interventions: Biological: allogeneic stem cell transplantation; Drug: Busulfan; Drug: Clyclophosphamide; Drug: Melphalan; Drug: Fludarabina

• Registration Number: NCT01969942
• Lead Sponsor: University of Louisville

Brief Summary

The purpose of this study is to examine the toxicity of using allogeneic stem cell transplantation for treatment of subjects with relapsed or refractory Ewing Sarcoma (ES). Donors will consist of either Human Leukocyte Antigen identical (HLA)or 9/10 (A, B, C, DR, DQ \[A, B, C, are Class I markers for HLA and DR and DQ are Class II markers for Matching for Transplant for Donors to match with recipient\]) matched related or unrelated donors. Specifically, we will examine:

* The toxicity of allogeneic stem cell transplant (SCT) in this patient population, as related to incidence of grade 3-4 acute Graft Verse Host Disease (GVHD).

* The incidence of transplant related mortality at 100 days.

Detailed Description

Not available

Study Timeline

• Study Start: 2013-04
• Study First Submitted: 2013-10-21
• Study First Submitted QC: 2013-10-24
• Study First Posted: 2013-10-25
• Primary Completion: 2016-10
• Study Completion: 2016-10
• Status Verified: 2017-05
• Last Update Submitted: 2017-05-08
• Last Update Posted: 2017-05-09

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• Patients aged 0-30 years with relapsed or therapy refractory Ewings sarcoma, excluding patients with brain metastases. Patients who have received a prior autologous stem cell transplant are eligible.
• Related and unrelated marrow and peripheral blood stem donors must be 9/10 or 10/10 (HLA A, B, C, DR, DQ) matched with the recipient.

Exclusion Criteria

• Organ dysfunction: Patients who have the following levels of organ system dysfunction are not eligible:

  • Cardiac: Ejection Fraction less than 50 percent
  • Renal: Est. Creatinine Clearance less than 50
  • Hepatic: Bilirubin greater than 3.0
  • Pulmonary: Diffusing Capacity for Carbon Monoxide (DLCO) less than 70 percent, or for patient who cannot cooperate with pulmonary function testing, O2 saturation less than 95 percent on room air.

• Performance status: Lansky performance less than 70; Eastern Cooperative Oncology Group (ECOG) status greater than or equal to 2

• Patients with an isolated recurrence of their tumor (in the site of primary tumor) greater than 1 year after completing therapy are excluded as these patients could be cured with local therapy alone.

  • As part of the standard of care for pre-transplant evaluation subjects will be tested for exposure to viral agents such as hepatitis B, C, HTLV-1/2 and HIV. Subjects testing positive for HI may be rejected as candidates for transplantation, based on the clinical judgement of the stem cell transplant physician.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
allogeneic stem cell transplantation Plan A	allogeneic stem cell transplantation	Subjects will receive the vaccine, Busulfan and Melphalan.
allogeneic stem cell transplantation Plan A	Busulfan	Subjects will receive the vaccine, Busulfan and Melphalan.
allogeneic stem cell transplantation Plan A	Melphalan	Subjects will receive the vaccine, Busulfan and Melphalan.
allogeneic stem cell transplantation Plan B	allogeneic stem cell transplantation	If subject have already received a stem cell transplant using Busulfan and Melphalan, they will receive Clysophosohamide and Fludarabine.
allogeneic stem cell transplantation Plan B	Clyclophosphamide	If subject have already received a stem cell transplant using Busulfan and Melphalan, they will receive Clysophosohamide and Fludarabine.
allogeneic stem cell transplantation Plan B	Fludarabina	If subject have already received a stem cell transplant using Busulfan and Melphalan, they will receive Clysophosohamide and Fludarabine.

Primary Outcome Measures

Name	Time	Method
Development of Grade 3-4 GVHD	12 months

Trial Locations

Locations (1)

Pediatric Hematology/Oncology University of Louisville; 🇺🇸 Louisville, Kentucky, United States