Know Cancer

or
forgot password

A Phase II Trial of a Chemotherapy Based Regimen of Intravenous Busulfan (Busulfex), Melphalan and Thiotepa as Myeloablative Regimen Followed by a T- Cell Depleted Allogeneic Hematopoietic Stem Cell Transplant From and HLA-Compatible Donor in the Treatment of High Risk Ewing's Sarcoma Family Tumors


Phase 2
N/A
40 Years
Not Enrolling
Both
Sarcoma

Thank you

Trial Information

A Phase II Trial of a Chemotherapy Based Regimen of Intravenous Busulfan (Busulfex), Melphalan and Thiotepa as Myeloablative Regimen Followed by a T- Cell Depleted Allogeneic Hematopoietic Stem Cell Transplant From and HLA-Compatible Donor in the Treatment of High Risk Ewing's Sarcoma Family Tumors


OBJECTIVES:

- Evaluate disease-free and overall survival of patients with high-risk tumors of the
Ewing's family treated with unmodified T-cell depleted allogeneic hematopoietic stem
cell transplantation after cytoreduction comprising busulfan, melphalan, and thiotepa.

- Determine the regimen-related morbidity and mortality in these patients.

- Determine the incidence of acute and chronic graft-vs-host disease in patients treated
with this regimen.

- Determine the biologic response of minimal residual disease in patients treated with
this regimen.

OUTLINE: This is a prospective study.

- Myeloablative preparative regimen: Patients receive busulfan IV over 2 hours every 6
hours on days -8 to -6, melphalan IV over 20 minutes on days -5 to -3, and thiotepa IV
over 4 hours on day -2.

- Allogeneic hematopoietic stem cell transplant: Patients undergo allogeneic bone marrow
or T-cell depleted peripheral blood stem cell transplantation on day 0.

- Graft-vs-host disease (GVHD) prophylaxis: Patients receive treatment according to
institutional guidelines and are given treatment against infection.

After completion of study treatment, patients are followed periodically for at least 3
years.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Diagnosis of high-risk tumors of the Ewing's family as defined by 1 of the following:

- Biopsy-proven disease with distant metastases to sites other than the lung

- Relapsed disease after completion of prior standard front-line therapy or
high-dose chemotherapy

- Currently in complete remission (CR) with no evidence of disease (with or without
minimal residual disease) or very good partial remission (i.e., CR with an abnormal
bone scan) after prior standard or high-dose chemotherapy with local control

- HLA-compatible stem cell donor available

- Compatible donors include those matched at both HLA-A, -B, -C, -DR and 1 of 2
-DQ alleles by high-resolution molecular typing

- Related or unrelated donor

PATIENT CHARACTERISTICS:

- Karnofsky performance status (PS) 70-100% (≥ 16 years old) OR Lansky PS 70-100% (< 16
years old)

- LVEF > 50% at rest

- SGOT < 3 times upper limit of normal

- Bilirubin < 2.0 mg/dL (unless liver is involved with disease)

- Creatinine normal AND/OR creatinine clearance > 60 mL/min

- Lung diffusion capacity > 50% of predicted (corrected for hemoglobin) OR asymptomatic
with a room air oxygen saturation of ≥ 98%

- Not pregnant or nursing

- Negative pregnancy test

- Fertile patients must use effective contraception

- No active uncontrolled viral, bacterial, or fungal infection

- No HIV-1 or -2 positivity

PRIOR CONCURRENT THERAPY:

- See Disease Characteristics

- No prior therapy with 100 mg/m² of melphalan

- No prior high-dose chemotherapy requiring autologous stem cell rescue

- No prior radiotherapy to > 50% of the pelvic marrow space

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Disease-free survival at 1 year

Outcome Time Frame:

2 years

Safety Issue:

No

Principal Investigator

Susan Prockop, MD

Investigator Role:

Study Chair

Investigator Affiliation:

Memorial Sloan-Kettering Cancer Center

Authority:

United States: Institutional Review Board

Study ID:

05-059

NCT ID:

NCT00357396

Start Date:

June 2005

Completion Date:

October 2009

Related Keywords:

  • Sarcoma
  • metastatic Ewing sarcoma/peripheral primitive neuroectodermal tumor
  • recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor
  • Sarcoma, Ewing's
  • Neuroectodermal Tumors, Primitive, Peripheral
  • Sarcoma

Name

Location

Memorial Sloan-Kettering Cancer Center New York, New York  10021