Phase 2
N=10
Busulfan, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk Ewing's Tumors
Sarcoma
Bottom Line
View on ClinicalTrials.gov: NCT00357396 ↗Enrolled (actual)
10
Serious AEs
40.0%
Results posted
Nov 2015
Primary outcome: Primary: Overall Objective Response — 4; 1 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- graft versus host disease prophylaxis/therapy (Biological); busulfan (Drug); melphalan (Drug); thiotepa (Drug); allogeneic bone marrow transplantation (Procedure); allogeneic hematopoietic stem cell transplantation (Procedure); peripheral blood stem cell transplantation (Procedure)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Memorial Sloan Kettering Cancer Center
- Primary completion
- Oct 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Objective Response |
4; 1 | — |
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.
Eligibility 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% ( 50% at rest
- SGOT 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
Data sourced from ClinicalTrials.gov (NCT00357396). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.