Phase 2
Completed N=28
Bone Marrow Transplantation of Patients in Remission Using Partially Matched Relative Donor
Source: ClinicalTrials.gov NCT01350245 ↗Enrolled (actual)
28
Serious AEs
67.9%
Results posted
Oct 2014
Primary outcomePrimary: Disease-Free Survival (DFS) — 78.6 percentage of patients
Summary
The primary hypothesis of this research study is that patients in remission undergoing myeloablative haploidentical hematopoietic stem cell transplantation (HSCT) on the Thomas Jefferson University (TJU) 2 Step treatment regimen will have a disease-free survival (DFS) rate at 1 year that is the same or better than the historical DFS of patients with similar diagnoses and ages undergoing matched sibling HSCT. Based on a review of the literature a DFS rate of 50% or better at 1 year would meet the criterion for an effective alternative therapy. A DFS rate of 75% or better would imply superior efficacy of the TJU 2 Step approach over T-replete matched sibling HSCT.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Disease-Free Survival (DFS) |
78.6 | — |
| PRIMARY Probability of Overall Survival at 15 Months Post-treatment |
85 | — |
Eligibility Criteria
Inclusion Criteria
- Any patient with a hematologic or oncologic diagnosis without morphological evidence of disease in which allogeneic HSCT is thought to be beneficial.
- Diagnoses include:
Acute Myeloid Leukemia Myelodysplastic Syndromes Biphenotypic Leukemia Acute Lymphocytic Leukemia Chronic Myeloid Leukemia Chronic Lymphocytic Leukemia Plasma Cell Neoplasms Lymphoma Hodgkin Disease Aplastic Anemia
- Patients must have a related donor who is a two or more allele mismatch at the HLA-A; B; C; DR loci.
- Patients must adequate organ function:
- LVEF of > or = 50%
- DLCO > or = 50% of predicted corrected for hemoglobin
- Adequate liver function as defined by a serum bilirubin or = 60 ml/min
- Performance status > or = 70% (TJU Karnofsky)
- HCT-CI Score 5 Points
- Combination of Performance status of or = 2 ugm/ml
- 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.
Data sourced from ClinicalTrials.gov (NCT01350245). 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.