Phase 2
N=27
A Two-Step Approach to Bone Marrow Transplant Using Cells From A Partially-Matched Relative
Hematologic Malignancies
Bottom Line
View on ClinicalTrials.gov: NCT00429143 ↗Enrolled (actual)
27
Serious AEs
85.2%
Results posted
May 2013
Primary outcome: Primary: Overall Survival of Participants — 13 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Total Body Irradiation (TBI) (Radiation); Donor Lymphocyte Infusion (DLI) (Biological); Cyclophosphamide (CY) (Drug); Tacrolimus (Drug); Mycophenolate Mofetil (MMF) (Drug); Hematopoietic Stem Cell Transplant (HSCT) (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
- Primary completion
- Aug 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Overall Survival of Participants |
13 | — |
| PRIMARY Optimal Dose of CD3+ Donor Lymphocytes (T-cells) for Consistent Engraftment Without GVHD |
2 | — |
| SECONDARY Engraftment Rates |
23 | — |
| SECONDARY Lymphoid Recovery |
23 | — |
| SECONDARY Incidence of Grades III-IV GVHD |
2 | — |
Summary
The purpose of this study is to develop a way of treating patients who do not have a completely matched family donor or a readily available unrelated donor with bone marrow transplant by using a partially-matched family donor. Patients receiving this type of transplant will receive chemotherapy and/or radiation to treat their disease. They will also receive their donor's cells in 2 parts. During the first part, the donor's lymphocytes will be exposed to one of the chemotherapy agents to help the patient become tolerant to the lymphocytes. In the second part of the transplant, the patient will receive their donor's stem cells to help recover their peripheral blood counts and establish long-term engraftment. The hypothesis of this study is that in partially-matched allogeneic transplant, there is a defined number of donor T-cells that can be treated and given to the recipient to avoid post-transplant infection without causing severe graft-versus-host disease.
Eligibility Criteria
Inclusion Criteria
- Any patient with a hematologic or oncologic diagnosis in which allogeneic HSCT is thought to be beneficial, and in whom front-line therapy has already been applied.
- Patients must have a related donor who is either a one, two or three out of six antigen mismatch at the HLA-A;B;DR loci.
- Patients without a well-matched unrelated donor or those who have a disease status that precludes a wait for an identified unrelated donor.
- Patients must adequate organ function:
- LVEF of >45%
- FVC or FEV1 >45% of predicted
- Adequate liver function as defined by a serum bilirubin 40 ml/min
- Performance status > 60% (Karnofsky)
- Patients must be willing to use contraception if they have childbearing potential
- Able to give informed consent
Exclusion Criteria
- An eligible HLA-identical sibling donor.
- Performance status < 60% (Karnosfsky)
- HIV positive
- Active involvement of the central nervous system with malignancy
- Psychiatric disorder that would preclude patients from signing an informed consent
- Pregnancy
- Patients with life expectancy of < 6 months for reasons other than their underlying hematologic/oncologic disorder.
Data sourced from ClinicalTrials.gov (NCT00429143). 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.