Phase 2
N=176
Mismatched Transplantation Using High-dose Post-transplant Cyclophosphamide
Blood Stem Cell Transplant Failure · Leukemia · Hematologic Malignancies
Bottom Line
View on ClinicalTrials.gov: NCT01010217 ↗Enrolled (actual)
176
Serious AEs
18.2%
Results posted
Jan 2020
Primary outcome: Primary: Number of Participants With Non-relapse Mortality (NRM) — 8; 7; 2; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Cyclophosphamide (Drug); Fludarabine (Drug); Melphalan (Drug); Mesna (Drug); Rituximab (Drug); Stem Cell Transplantation (Procedure); Thiotepa (Drug); Tacrolimus (Drug); Mycofenolate mofetil (Drug); G-CSF (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- M.D. Anderson Cancer Center
- Primary completion
- Oct 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Non-relapse Mortality (NRM) |
8; 7; 2; 0; 0 | — |
| SECONDARY Number of Participants With Non Related Mortality (NRM) |
17; 15; 4; 0; 0 | — |
| SECONDARY Engraftments |
80; 50; 21; 0; 0 | — |
| SECONDARY Grade III-IV aGVHD |
11; 7; 1; 0; 0 | — |
| SECONDARY cGVHD |
11; 7; 0; 0; 0 | — |
| SECONDARY Disease Free Survival |
50; 24; 12; 0; 0 | — |
Summary
The goal of this clinical research study is to learn about the safety of giving a stem cell transplant from a tissue-mismatched donor, followed by cyclophosphamide, to patients with certain types of blood disorders or blood cancers. Melphalan, thiotepa, and fludarabine will also be given before the transplant.
Researchers will study the health status of these patients at 3 months after the transplant.
Eligibility Criteria
Inclusion Criteria
- Patients 55 and 3 abnormalities], peripheral blood blasts 55 years and /=1st chronic phase, after failed >/=2 lines of tyrosine kinase inhibitors; in accelerated or blast phase with > 30% bone marrow blasts;
- Prior allogeneic stem cell transplant more than 6 months from the first transplant, in remission.
- Chemotherapy-sensitive relapsed lymphoma (Complete or partial response), Hodgkin's or non-Hodgkin's lymphoma, no evidence of "bulky" disease (> 10 cm in diameter);
- Patients with chemo-sensitive CLL with persistent or recurrent disease after fludarabine-based regimens, no evidence of "bulky" disease (> 10 cm in diameter)
- Patients with poor prognosis multiple myeloma by cytogenetics (del13, del 17p, t(1;14) or t(14;16) or hypodiploidy, with advanced disease (stage>/=2) and /or relapsed after autologous stem cell transplant.
- Patients with myelofibrosis (Lille >0, transfusion dependency, progression to blast phase; however, in remission from AML) or chronic myelomonocytic leukemia (CMML). These patients will be treated with the reduced-intensity conditioning regimen #2 and will be subject to the same stopping rule as the group >/= 55 years or with comorbidities.
- Zubrod performance status 0-1 or Lansky PS greater or equal to 70%.
- Patients above >/=65 years old should have an age-adjusted co-morbidity index of /=50 ml/min (calculated with Cockcroft-Gault formula); Creatinine for children /= 45% predicted corrected for hemoglobin. For pediatric patients, if unable to perform pulmonary function, >/= 92% oxygen saturation with pulse oximetry.
- Left ventricular ejection fraction (LVEF) >/= 40%.
- Patient or patient's legal representative, parent(s) or guardian should provide written informed consent. Assent of a minor if participant's age is at least seven and less than eighteen years.
Exclusion Criteria
- HIV positive; active hepatitis B or C
- Patients with active infections. The PI is the final arbiter of the eligibility.
- Liver cirrhosis with greater than grade 1 stage 1 inflammation/fibrosis
- Uncontrolled central nervous system (CNS) involvement by tumor cells
- Patients with AML must have less than 30% bone marrow blasts and no peripheral blood blasts.
- History of another primary malignancy that has not been in remission for at least 3 years. (The following are exempt from the 3-year limit: non-invasive nonmelanoma skin cancer, fully excised melanoma in situ [Stage 0], curatively treated localized prostate cancer, and cervical carcinoma in situ on biopsy or a squamous intraepithelial lesion on PAP smear.)
- Positive Beta human chorionic gonadotropin (HCG) test in a woman with child bearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization.
- Inability to comply with medical therapy or follow-up.
Data sourced from ClinicalTrials.gov (NCT01010217). 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.