Phase 2
N=78
Reduced Intensity (RIC) Conditioning And Transplantation of HLA-Haplo-HCT
Hematologic Malignancies
Bottom Line
View on ClinicalTrials.gov: NCT02988466 ↗Enrolled (actual)
78
Serious AEs
38.5%
Results posted
May 2025
Primary outcome: Primary: Disease-free Survival (DFS) — 57; 29; 38; 57 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Haplo HCT <55 years old (Biological); Haplo HCT ≥55 years old (Biological); GVHD Prophylaxis (Drug); Haplo HCT ≥55 and < 65 years old (Biological); Haplo HCT ≥65 and ≤75 years old (Biological)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- May 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Disease-free Survival (DFS) |
57; 29; 38; 57 | — |
| SECONDARY Incidence of Grade II-IV and Grade III-IV Acute Graft Versus-host-disease (GVHD) |
21; 33; 13; 31; 0; 19 | — |
| SECONDARY Treatment Related Mortality (TRM) |
21; 57; 13; 26; 21; 57 | — |
| SECONDARY Relapse Incidence |
29; 29; 75; 26; 29; 30 | — |
| SECONDARY Incidence of Serious Fungal and Viral Infection |
7; 10; 38; 6; 14; 10 | — |
Summary
This is a single institution phase II study of a reduced intensity conditioning (RIC) followed by a haploidentical hematopoietic cell transplant (haplo-HCT) in persons with diagnosis of hematologic malignancy. Conditioning will consists of fludarabine, cyclophosphamide, melphalan and total body irradiation (TBI) preparative regimen with a melphalan dose reduction for patients ≥55 years old and those with HCT Comorbidity Index (CI) >3. This study uses a two-stage phase II design with accrual goal of 84 patients, using 28 patients separately for arms A, C and D
Eligibility Criteria
Inclusion Criteria
- Karnofsky performance status of ≥70% or Lansky play score ≥ 70%
- A related haploidentical bone marrow donor with up to 2 or 3 HLA locus-mismatches
- The donor and recipient must be HLA identical for at least one haplotype (using high resolution DNA based typing) at the following genetic loci: HLA-A, HLA-B, HLA-C, and HLA-DRB1.
- Adequate liver and renal function
- Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction ≥ 40%
- Diffusion capacity corrected (DLCOcorr) > 40% predicted, and absence of O2 requirements
- > 6 months after prior autologous transplant (if applicable)
- Agrees to use contraception during study treatment
- Voluntary written consent (adult or parent/guardian with presentation of the minor information sheet, if appropriate)
- Patients who are HIV+ must have undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation
Exclusion Criteria
- 5% blasts in normocellular bone marrow OR any % blasts if blasts have unique morphologic markers (e.g. Auer rods).
- CML in blast crisis
- Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressive on salvage therapy.
- stable non-bulky disease is acceptable.
- Active central nervous system malignancy
Criteria For Donor Selection:
- Donors must be HLA-haploidentical relatives of the patient, defined as having a shared HLA haplotype between donor and patient at HLA-A, -B, -C, and -DRB1.
- Eligible donors (14-70 years old) include biological children, siblings or half siblings, or parents, able and willing to undergo bone marrow harvesting.
- For donors <18 years, the maximum recipient weight (actual body weight) should not exceed 1.25 times the donor weight (actual body weight)1 In addition, bone marrow product volume should be limited to 20 ml/kg donor weight for donors <18 years.
Data sourced from ClinicalTrials.gov (NCT02988466). 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.