Phase 2
N=34
Reduced Intensity Flu/Mel/TBI Conditioning for HAPLO HCT Patients With Hematologic Malignancies
Acute Myeloid Leukemia · Acute Lymphoblastic Leukemia · Biphenotypic Acute Leukemia · Undifferentiated Leukemia · Prolymphocytic Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT04191187 ↗Enrolled (actual)
34
Serious AEs
61.8%
Results posted
Mar 2025
Primary outcome: Primary: Disease Free Survival — 70.6 percentage of participants — p=0.002
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Fludarabine (Drug); Melphalan (Drug); Total Body Irradiation (Radiation)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- H. Lee Moffitt Cancer Center and Research Institute
- Primary completion
- Feb 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Disease Free Survival |
70.6 | 0.002 sig |
| SECONDARY Percentage of Participants With Graft vs Host Disease (GVHD) Free Survival |
61.8 | — |
| SECONDARY Percentage of Participants Overall Survival (OS) |
73.3 | — |
| SECONDARY Percentage of Participants With Treatment Related Mortality (TRM) at 6 Months |
17.6 | — |
| SECONDARY Percentage of Participants With Treatment Related Mortality (TRM) at 18 Months |
17.6 | — |
| SECONDARY Percentage of Participants With Relapse Free Survival (RFS) |
70.6 | — |
Summary
This is a single arm, phase II trial of HLA-haploidentical related hematopoietic cells transplant (Haplo-HCT) using reduced intensity conditioning (fludarabine and melphalan and total body irradiation). Peripheral blood is the donor graft source. This study is designed to estimate disease-free survival (DFS) at 18 months post-transplant.
Eligibility Criteria
Inclusion Criteria
- Age ≥ 55 years or HCT Co-Morbidity score (HCT-CI) >/=3
- Lack of a suitable 8/8 HLA-matched sibling donor
- Adequate performance status is defined as Karnofsky score ≥ 70%
- Patients and selected donor must be HLA typed at high resolution using DNA based typing at the following HLA-loci: HLA-A, -B, -C and DRB1. Donors must be HLA-haploidentical relatives including, but not limited to, children, siblings, or parents, defined as having a shared HLA haplotype between donor and patient at HLA-A, -B, -C, and -DRB1.
- Acute Myeloid Leukemia (AML): Must be in remission with morphology ( 12 months are eligible after at least two prior therapies
- Patients with primary, refractory disease. Bulky disease and an estimated tumor doubling time of less than one month require debulking therapy prior to transplant.
- Lymphoplasmacytic lymphoma is eligible after initial therapy if chemotherapy sensitive
- Adequate organ function as defined per protocol
- Sexually active females of child bearing potential and males with partners of child bearing potential must agree to use adequate birth control during study treatment
Exclusion Criteria
- Pregnant or breastfeeding
- Untreated active infection
- Active HIV infection
- Prior allogenic transplant at any time prior or less than 6 months since prior autologous transplant (if applicable)
- Active central nervous system malignancy
- Favorable risk AML defined as per protocol
- Active central nervous system malignancy
- Favorable risk AML defined as having one of the following:
- t(8, 21) without cKIT mutation or evidence of immunophenotypic, cytogenetic or molecular minimal residual disease (MRD)
- inv(16) or t(16;16) without cKIT mutation or evidence of MRD
- Normal karyotype with mutated NPM1 but FLT3-ITD wild type without evidence of MRD
- Normal karyatype with double mutated CEBPA without evidence of MRD
Data sourced from ClinicalTrials.gov (NCT04191187). 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.