Phase 2
Completed N=38
Clofarabine, Melphalan, and Thiotepa Followed By a Donor Stem Cell Transplant in Treating Patients With High-Risk and/or Advanced Hematologic Cancer or Other Disease
Source: ClinicalTrials.gov NCT00423514 ↗Enrolled (actual)
38
Serious AEs
29.0%
Results posted
Dec 2022
Primary outcomePrimary: Response to Therapy — 1; 0; 27; 4 Participants
Summary
RATIONALE: Giving chemotherapy, such as clofarabine, melphalan, and thiotepa, before a donor stem cell transplant helps stop the growth of cancer or abnormal cells. It also helps stop the patient's immune system from rejecting the donor's 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 also make an immune response against the body's normal cells. Giving tacrolimus and mycophenolate mofetil before the transplant may stop this from happening.
PURPOSE: This phase I/II trial is studying the side effects and best dose of clofarabine when given together with melphalan and thiotepa, followed by a donor stem cell transplant and to see how well it works in treating patients with high-risk and/or advanced hematologic cancer or other disease.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Response to Therapy |
1; 0; 27; 4; 3; 3 | — |
| PRIMARY Overall Survival |
19; 4; 12; 3 | — |
| SECONDARY Participants Evaluated for Early Post-transplant Regimen-related Severe Morbidity (Grade III to IV Nonhematologic Toxicity) and Mortality as Measured by the NCI Cancer Therapy Evaluation Program CTCAE v 3.0 |
31; 7 | — |
Eligibility Criteria
DISEASE CHARACTERISTICS:
- Histologically or cytologically confirmed diagnosis of 1 of the following:
- Acute myelogenous leukemia, meeting 1 of the following criteria:
- In first complete remission (CR), meeting 1 of the following criteria:
- Poor risk [no t(15, 17), inv 16, or t(8,21)]
- Not a candidate for total body irradiation (TBI)
- Any infant in first CR
- In second CR, meeting the following criteria:
- All patients
- In more than second CR OR relapsed/refractory disease, meeting the following criteria:
- All patients
- Blast percentage > 5% and 5% and 5% and RAEB1
- Secondary high risk disease
- All patients
- Any stage
- Juvenile myelomonocytic leukemia
- All patients
- No doubling of peripheral blast counts within a period of 2 weeks
- No active CNS disease
- HLA-compatible donor available meeting 1 of the following criteria:
- Related donor
- Genotypically or phenotypically matched at ≥ 7 or 8 of HLA-A, -B, -C and -DRB1 alleles
- Unrelated donor meeting 1 of the following criteria:
- 8 of 8 alleles matched
- For patients 60 mL/min
- LVEF > 50% at rest OR shortening fraction ≥ 29%
- Patients with asymptomatic pulmonary disease with no prior risk factors OR symptomatic pulmonary disease with diffusion capacity > 50% of predicted (corrected for hemoglobin) are eligible
- No active uncontrolled viral, bacterial, or fungal infection
- No known HIV I or II positivity
- No known human T-cell lymphotrophic virus I or II positivity
- Not pregnant or nursing
- Negative pregnancy test
- Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY:
- No hydroxyurea within the past 2 weeks
- No allogeneic or autologous stem cell transplantation within the past 6 months
Data sourced from ClinicalTrials.gov (NCT00423514). 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. Informational only — not medical advice.