Phase 2
Completed N=60
Thiotepa-Clofarabine-Busulfan With Allogeneic Stem Cell Transplant for High Risk Malignancies
Source: ClinicalTrials.gov NCT00857389 ↗Enrolled (actual)
60
Serious AEs
89.7%
Results posted
Apr 2020
Primary outcomePrimary: Number of Participants With Survival Rate at 100 Days Post-transplant — 45 Participants
Summary
Any time the words "you," "your," "I," or "me" appear, it is meant to apply to the potential participant.
The goal of this clinical research study is to learn if thiotepa, busulfan, and clofarabine, when given before an allogeneic (bone marrow , blood, or cord blood cells) or haploidentical (bone marrow) stem cell transplantation can help to control cancers of the bone marrow and lymph node system. The safety of this treatment will also be studied.
This is an investigational study. Thiotepa and clofarabine are FDA approved and commercially available for the treatment of leukemia. Busulfan is FDA approved and commercially available for use in stem cell transplantation. The combination of thiotepa, clofarabine, and busulfan together with a stem cell transplant is investigational.
Up to 60 participants will take part in this study. All will be enrolled at M. D. Anderson.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Survival Rate at 100 Days Post-transplant |
45 | — |
| SECONDARY Number of Participants With Disease Free Survival |
32 | — |
| SECONDARY Overall Survival Rate |
320 | — |
| SECONDARY Graft vs Host Disease (GVHD) |
34 | — |
| SECONDARY Engraftment |
52 | — |
| SECONDARY Number of Participants With Serious Adverse Events |
52 | — |
| SECONDARY Relapse Rate of Participants Treated With Thiotepa, Busulfan, and Clofarabine |
26 | — |
Eligibility Criteria
Inclusion Criteria
- Diagnosed with one of the following diseases:
- Acute myelogenous leukemia (AML) in induction failure, relapse, past first remission, or CR1 considered at risk for relapse
- Myelodysplastic syndromes with International Prognostic Scoring System score (IPSS score) >/= 2 or myelodysplasia that has not responded to chemotherapy
- Biphenotypic leukemia
- Acute lymphocytic leukemia with induction failure, first complete remission with high risk cytogenetics (e.g. Philadelphia positive chromosome, t(4:11) Remission requiring more than 2 chemotherapy to achieve remission, or any stage beyond CR1
- Chronic Myelogenous Leukemia (CML): second chronic phase, accelerated phase or blast crises with less than 10% blasts in the bone marrow, or CR1 and resistance to Gleevec or other tyrosine kinase inhibitors
- Non-Hodgkin's Lymphoma - induction failures, second or third complete remission, or relapse (including relapse post autologous hematopoietic stem cell transplant)
- Hodgkin's disease - induction failure, second or later complete remission, or relapse (including relapse post autologous hematopoietic stem cell transplant).
- Chronic Lymphocytic Leukemia that has failed induction therapy or Rai Stages 2-4
- Related or unrelated donor which is HLA-matched or mismatched in 1 HLA A, B, C, DR, or DQ locus is acceptable (i.e. >/= 9/10 matched related or unrelated donor, matched with molecular high-resolution technique per current std. for BMT program). Cord blood units must match patient at 4, 5, or 6/6 HLA class 1 serological & II molecular antigens with a min. of 2 x 10e7 TNC/kg recipient weight in the pre-thawed fraction. For patient lacking a matched related or unrelated donor or acceptable cord blood unit(s), a related haploidentical donor ( /= 50% for patients 16 years of age.
- Cardiac function - left ventricular ejection fraction >/= 40%.
- Pulmonary function - diffusion capacity of at least 50% predicted. Children unable to perform pulmonary function tests (e.g. less than 7 years old) pulse oximetry of >/= 92% on room air.
- Serum creatinine /= 50 ml/min.
- SGPT </= 200 IU/mL, serum bilirubin < 1.5 x normal.
- Written informed consent and assent as is age appropriate.
- No active infection.
Exclusion Criteria
- Pregnancy in women of child bearing potential (pregnancy test performed within 2 weeks of study entry).
- HIV positive (highly immunosuppressive treatment)
- Active CNS leukemia
- Chronic or active Hepatitis B or Hepatitis C. If questions about liver health discuss with PI and strongly consider liver biopsy.
Data sourced from ClinicalTrials.gov (NCT00857389). 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.