Phase 2
Completed N=78
A Research Study of Bone Marrow Transplantation From Unrelated or Partially Matched Related Donors
Hematological Malignancies
Source: ClinicalTrials.gov NCT01349101 ↗
Enrolled (actual)
78
Serious AEs
12.8%
Results posted
Dec 2025
Primary outcomePrimary: Successful Engraftment Using Cyclophosphamide Post-Transplant — 1.00; 0.93 Cumulative Probability of Engraftment
Summary
It is hypothesized that engraftment when administering cyclophosphamide post the stem cell infusion will increase, the incidence of graft versus host disease (GVHD) and day 100 mortality will decrease, and the use of cyclophosphamide post stem cell infusion with alternative donors will be as safe and as effective as traditional matched transplants.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Successful Engraftment Using Cyclophosphamide Post-Transplant |
1.00; 0.93 | — |
| SECONDARY Cumulative Incidence of Grade III-IV GVHD |
0.5152; 0.3111 | — |
| SECONDARY Incidence of GVHD Unresponsive to Corticosteroids and Photopheresis |
0.0625; 0.0233 | — |
| SECONDARY Transplant-Related Mortality |
0.0909; 0.1951 | — |
Eligibility Criteria
Inclusion Criteria
- Any patient with a hematological or oncological diagnosis in which allogeneic hematopoietic stem cell transplantation (HSCT) is thought to be beneficial.
- Patients without morphological or molecular evidence of disease
- For patients with "indolent diseases, " if the patient has evidence of disease the disease burden must be minimal (at least PR) and the disease must be chemoresponsive. Thus for example patients with acute leukemia (not an indolent disease) must be in a morphological CR or CRp.
- For patients with MDS the inclusion criteria is specifically as follows:
- For patients with RA or RARS or isolated 5q- they can proceed to transplant without any treatment.
- For patients with RAEB-1, RCMD+/-RS, or MDS NOS must have stable disease for 6 months (as documented by serial bone marrow examinations) in the absence of any therapy but growth factors or transfusion support. Patients who require treatment to "control their disease" must show chemo-responsiveness
- For patients with CMML or RAEB-2 they must demonstrate chemo-responsiveness
- Chemo-responsiveness is defined as a blast percentage decrease by at least 5 percentage points and there must be less than 10% blasts after treatment and at the time of transplant, if there are more than 10% blasts at any point during the disease course
- Chemo-responsiveness must also include at least one of the following if applicable:
- A cytogenetic response
- A well-documented decrease in transfusion requirements.
- Patients must have a related donor who is zero, one, two, three, or four antigen mismatched at the HLA-A; B; C; DR loci or an unrelated donor up to a two antigen mismatch. DNA will be retained by the tissue typing laboratory for possible typing for DQ and DP. When multiple related donor options are available donor selection will be determined the same as in the TJU two-step protocols. When multiple unrelated donors are available care will be made to avoid HLA-A and HLA-B mismatches if possible based on data from the Japanese Marrow Donor Registry studies. An HLA antibody screen will be performed on each patient.
- All patients must have adequate organ function:
- Patients with related donors must have an LVEF of >35%. Patients with unrelated donors must have an LVEF >45%. Patients with LVEF ≤50% and all patients with symptoms or history of heart failure or coronary artery disease must have a stress echo or equivalent test and a cardiological evaluation.
- Patients with related donors must have a DLCO >35% of predicted corrected for hemoglobin. Patients with unrelated donors must have a DLCO >45% of predicted corrected for hemoglobin. For related donors if the DLCO is less than 45% the EF must be greater than 45% and vice versa.
- Patients with related donors must have an adequate liver function as defined by a serum bilirubin 60 ml/min/1.73 m^2.
- Patients with related donors must have a performance status > 60% (TJU Karnofsky14) (Appendix A). Patients with unrelated donors must have a Performance status > 70% (TJU Karnofsky).
- Patients with related donors must have a HCT-CI Score 2 μg/ml. Patients on systemic corticosteroids at a dose equivalent of prednisone 7.5mg/day or higher.
- Patients who cannot receive cyclophosphamide.
- Patients with evidence of another malignancy, exclusive of a skin cancer that requires only local treatment, should not be enrolled on this protocol.
- Patients with refractory disease.
- Patients with clinically significant preformed antibodies to their donors.
- Patients who require supplemental oxygen other than for sleep apnea will be excluded.
Data sourced from ClinicalTrials.gov (NCT01349101). 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.