Ireland
--:--IST
Starting soon Phase 3

Reduced Post-transplant Cyclophosphamide Dose in Patients Undergoing Haploidentical Hematopoietic Stem Cell Transplantation for Hematological Malignancies

NCT07193420 · tracked via the Priya Life Science France tracker
Phase
Phase 3
Started
2026-06
Last updated
2026-05-12

Condition(s) studied

GVHD - Graft-Versus-Host DiseaseHSCTHaploidentical Stem Cell Transplantation

Investigational drug(s) / intervention(s)

Cyclophosphamide 35mg/kg/day →Cyclophosphamide 50mg/kg/day →

Cyclophosphamide 35mg/kg/day: Cyclophosphamide will be administered intravenously (IV) post-HSCT at the experimental dose (70 mg/kg, divided into two doses of 35 mg/kg/day (Adjusted Body Weight) on days +3 and +4).

Cyclophosphamide 50mg/kg/day: Cyclophosphamide will be administered intravenously (IV) post-HSCT at the standard dose (100 mg/kg, divided into two doses of 50 mg/kg/day (Adjusted Body Weight) on days +3 and +4).

Study summary

Phase III comparative, open-label, randomized (1:1) trial designed to evaluate the efficacy of reducing the total dose of PTCy to 70 mg/kg on GREFS compared to the standard dose of 100 mg/kg, in patients undergoing haploidentical HSCT for the treatment of a hematological malignancy, two years after HSCT.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * Age ≥ 18 years * Confirmed hematological malignancy with an indication for allogeneic HSCT * Presence of a haploidentical donor willing to donate PBSC * Patient planned to receive a thiotepa-based conditioning regimen * Provision of written informed consent Affiliation to a social security system (excluding "Aide Médicale d'État") Exclusion Criteria: * Karnofsky performance status \< 70% * Life expectancy \< 1 month, as determined by the attending physician * Acute or chronic heart failure, defined as left ventricular ejection fraction \< 40% * Pulmonary dysfunction with diffusion capacity \< 50% of predicted values * Renal impairment with estimated glomerular filtration rate (eGFR) \< 45 mL/min (calculated using the CKD-EPI formula) * Decompensated hemolytic anemia * Fanconi anemia and other DNA breakage repair disorders * Acute urothelial toxicity due to cytotoxic chemotherapy or radiotherapy * Obstruction of urinary outflow * Concomitant use with yellow fever vaccine and with live virus and bacterial vaccines * Combination with products containing Hypericum perforatum * Combination with medicines that are substrates for the multidrug efflux transporter P-glycoprotein (P-gp) or the organic anion transporter proteins (OATP) and for which elevated plasma concentrations are associated with serious and/or life-threatening events, e.g., bosentan, dabigatran etexilate and aliskiren * Active non-controlled infectious disease * Positive HIV status * Pregnancy, breast-feeding, or refusal to use effective contraception for the duration of the study and 6 months after the last treatment dose * Individuals under legal protection measures or unable to provide consent (e.g., severe neurological or psychiatric disorders, or deprivation of liberty by judicial or administrative decision) * Hypersensitivity to the active substance or any of the excipients * Concurrent participation in another investigational therapeutic study * Inability to comply with study procedures as assessed by the investigator based on objective criteria, including but not limited to: * Significant language barrier in the absence of adequate translation support * Social or geographic situation preventing follow-up and adherence to visit schedule * Ongoing substance abuse likely to interfere with protocol compliance * Documented cognitive or functional impairment not otherwise covered under legal protection

Primary outcome measure(s)

  • GVHD-free, relapse-free, event-free survival (GREFS) — Day 0 to first occurrence of acute grade III-IV GVHD, severe chronic GVHD, relapse, death, grade 3-4 cardiac event, or grade 3-4 BK virus-associated HC (up to 24 months post-transplant); platelet recovery (>50 × 10^9/L) assessed until Day +60
    The primary endpoint is the assessment of the GREFS at 2 years after HSCT, a composite endpoint defined as the probability of survival without severe GVHD, relapse/progression of the hematological malignancy, or PTCy-associated adverse event, whichever comes first from transplantation

Trial sites (1)

FacilityCityRegionStatus
Saint Antoine Hospital - Hematology Department Paris France
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07193420 on ClinicalTrials.gov ↗ ← All trials in France