Phase 2
N=41
Allogeneic Hematopoietic Stem Cell Transplantation for High-Risk Hematologic Malignancies Using One Haploidentical Donor
Hematopoietic/Lymphoid Cancer
Bottom Line
View on ClinicalTrials.gov: NCT01982682 ↗Enrolled (actual)
41
Serious AEs
80.0%
Results posted
Jun 2018
Primary outcome: Primary: Count of Participants That Experience 1 Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplantation (HSCT) Using the Thomas Jefferson University 2 Step Approach — 27 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Total-Body Irradiation (TBI) (Radiation); Donor Lymphocyte Infusion (DLI) (Biological); Cyclophosphamide (Drug); Allogeneic hematopoietic stem cell transplantation (HSCT) (Procedure); Mycophenolate mofetil (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
- Primary completion
- Mar 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Count of Participants That Experience 1 Year Relapse Free Survival After Undergoing Hematopoietic Stem Cell Transplantation (HSCT) Using the Thomas Jefferson University 2 Step Approach |
27 | — |
| SECONDARY Number of Participants With Successful Engraftment |
37 | — |
| SECONDARY Count of Participants That Experienced Death as a Result of Graft-versus-host Disease (GVHD) |
2 | — |
| SECONDARY Median Pace of T Cell Immune Recovery at 28 Days Post Hematopoietic Stem Cell Transplantation (HSCT) |
41.3; 48 | — |
| SECONDARY Median Pace of T Cell Immune Recovery at 90 Days Post Hematopoietic Stem Cell Transplantation (HSCT) |
141; 384 | — |
Summary
This phase II trial studies how well total-body irradiation, donor lymphocyte infusion, and cyclophosphamide before donor stem cell transplant works in treating patients with high-risk hematologic malignancies. Giving total-body irradiation, donor lymphocyte infusion, and chemotherapy before a donor stem cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's stem cells. When certain 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 cells. Removing the T cells from the donor cells before transplant and giving tacrolimus and mycophenolate mofetil may stop this from happening.
Eligibility Criteria
Inclusion Criteria
- This treatment is for patients with high risk hematologic malignancies. High risk is defined as:
- Any patient with a hematologic malignancy with residual disease after treatment with 1 or more chemotherapy regimens in whom achievement of remission with additional chemoradiotherapy is felt to be unlikely
- Patients without morphologic evidence of disease but with high risk features which would predict for relapse despite remission at HSCT such as adverse cytogenetics, 3rd or greater CR (complete response), or failure to recover peripheral blood counts to normal ranges. While these patients do not have detectable disease by current methods, like all patients they have non-detectable disease which in their case is highly aggressive.
- Patients must have one related donor who is HLA (human leukocyte antigen) mismatched in the GVHD direction at two or more HLA loci
- Patients must adequate organ function:
- LVEF (left ventricular ejection fraction) of >50 %
- Diffusing capacity of the lungs for carbon monoxide (DLCO) (adjusted for hemoglobin) >50 % of predicted and forced expiration to the full FEV-1 >50 %
- Adequate liver function as defined by a serum bilirubin 60 ml/min
- Karnofsky Performance Status (KPS) of > 80% on the modified (KPS) tool
- Patients must be willing to use contraception if they have childbearing potential
- Able to give informed consent
Exclusion Criteria
- Modified (KPS) Karnofsky Performance status of 5 Comorbidity Points on the Hematopoietic cell transplantation - specific comorbidity (HCT-CI) Index (See Appendix B)
- Class I or II antibodies against donor human leukocyte antigens (HLA)
- HIV positive
- Active involvement of the central nervous system with malignancy
- Psychiatric disorder that would preclude patients from signing an informed consent
- Pregnancy, or unwillingness to use contraception if they have child bearing potential
- Patients with life expectancy of 2 ugm/ml
- Patients with active inflammatory processes including T max >101 or active tissue inflammation are excluded
- Inability to tolerate cyclophosphamide or undergo total body irradiation at the doses specified in the treatment plan
Data sourced from ClinicalTrials.gov (NCT01982682). 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.