Busulfan, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk Ewing's Tumors

NCT00357396 | Completed Phase 2 Results

• 2 other identifiers: 05-059MSKCC-05059
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

RATIONALE: Giving chemotherapy drugs, such as busulfan, melphalan, and thiotepa, before a donor stem cell transplant helps stop the growth of tumor cells and prepares the patient's bone marrow for the stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal tissues. Giving tacrolimus, sirolimus, and mycophenolate mofetil may stop this from happening. PURPOSE: This phase II trial is studying how well giving busulfan together with melphalan and thiotepa followed by a donor stem cell transplant works in treating patients with high-risk Ewing's tumors.

Conditions

Sarcoma

Keywords

metastatic Ewing sarcoma/peripheral primitive neuroectodermal tumor; recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor

Outcome Measures

Primary Outcomes (1)

• Overall Objective Response

  2 years

Study Arms (1)

Chemo followed by DSCT

EXPERIMENTAL

* 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.

Biological: graft versus host disease prophylaxis/therapy; Drug: busulfan; Drug: melphalan; Drug: thiotepa; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation

Interventions

graft versus host disease prophylaxis/therapy BIOLOGICAL

Chemo followed by DSCT

busulfan DRUG

Chemo followed by DSCT

melphalan DRUG

Chemo followed by DSCT

thiotepa DRUG

Chemo followed by DSCT

allogeneic bone marrow transplantation PROCEDURE

Chemo followed by DSCT

allogeneic hematopoietic stem cell transplantation PROCEDURE

Chemo followed by DSCT

peripheral blood stem cell transplantation PROCEDURE

Chemo followed by DSCT

Eligibility Criteria

• Age: Up to 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Memorial Sloan Kettering Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

Memorial Sloan-Kettering Cancer Center

New York, New York, 10065, United States

Location

MeSH Terms

Conditions

Sarcoma; Neuroectodermal Tumors, Primitive, Peripheral

Interventions

Therapeutics; Busulfan; Melphalan; Thiotepa; Peripheral Blood Stem Cell Transplantation

Condition Hierarchy (Ancestors)

Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Butylene Glycols; Glycols; Alcohols; Organic Chemicals; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Amino Acids, Peptides, and Proteins; Phosphoramides; Organophosphorus Compounds; Triethylenephosphoramide; Aziridines; Azirines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Hematopoietic Stem Cell Transplantation; Stem Cell Transplantation; Cell Transplantation; Cell- and Tissue-Based Therapy; Biological Therapy; Transplantation; Surgical Procedures, Operative

Results Point of Contact

• Title: Dr. Susan Prockop
• Organization: Memorial Sloan Kettering Cancer Center

prockops@mskcc.org

212-639-6715

Study Officials

• Susan Prockop, MD

  Memorial Sloan Kettering Cancer Center

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 26, 2006
• First Posted: July 27, 2006
• Study Start: June 1, 2005
• Primary Completion: October 1, 2009
• Study Completion: October 1, 2009
• Last Updated: November 25, 2015
• Results First Posted: November 25, 2015

Record last verified: 2015-10

Locations

US (1)