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Phase 2 Completed N=34 Treatment

Reduced Intensity Flu/Mel/TBI Conditioning for HAPLO HCT Patients With Hematologic Malignancies

Source: ClinicalTrials.gov NCT04191187 ↗
Enrolled (actual)
34
Serious AEs
61.8%
Results posted
Mar 2025
Primary outcomePrimary: Disease Free Survival — 70.6 percentage of participants — p=0.002

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.

Outcome Measures

OutcomeResultp-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 —

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
View full record on ClinicalTrials.gov →

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. Informational only — not medical advice.

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