Reduced Post-transplant Cyclophosphamide Dose in Patients Undergoing Haploidentical Hematopoietic Stem Cell Transplantation for Hematological Malignancies
GVHD - Graft-Versus-Host Disease · HSCT · Haploidentical Stem Cell Transplantation
What this study involves
The registry says this study is recruiting. Individual locations may have different availability.
- Study type
- Interventional
- Phase
- Phase 3
- Age range
- 18 years - Not specified
- Sex eligible
- All sexes
- Healthy volunteers
- Not accepted
- Participants
- 180 Estimated
These are starting points, not an eligibility decision. The research team must review the full requirements.
About the research
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.
Read the full description
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.
Who may be able to participate?
The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.
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
Treatment, groups and participation
The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.
- Assignment
- Random assignment
- Masking
- No masking
Reduced dose
cyclophosphamide administered at 35mg/kg/day (Adjusted body weight) on days +3 and +4
Drug: Cyclophosphamide 35mg/kg/day
Standard dose
cyclophosphamide administered at 50mg/kg/day (Adjusted Body Weight) on days +3 and +4
Drug: Cyclophosphamide 50mg/kg/day
Cyclophosphamide 35mg/kg/day DRUG
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 DRUG
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).
What researchers are measuring
Outcome measures describe what is being studied. They are not proof that a treatment works.
Primary measures
GVHD-free, relapse-free, event-free survival (GREFS)
Time frame: 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
Secondary measures
Overall survival (OS)
Time frame: From transplantation until death from any cause or up to 24 months, whichever occurs first
Overall survival (OS) at 2 years is defined as survival irrespective of disease status
Quality of life FACT-BMT
Time frame: At 1, 3, 6, 12, and 24 months after HSCT
Quality of life compared to baseline using questionnaire: FACT-BMT (Functional Assessment of Cancer Therapy - Bone Marrow Transplant, version 4)
Quality of life EQ-5D-5L
Time frame: At 1, 3, 6, 12, and 24 months after HSCT
Quality of life compared to baseline using questionnaire: EQ-5D-5L (EuroQol 5-Dimension, 5-Level questionnaire).
Toxicities, infection and hematogical recovery
Time frame: From transplantation until the occurrence of the event, day +60 for hematological recovery, or up to 24 months for organ damage toxicities and infections, whichever occurs first
Organ damage toxicities assessed by the common terminology criteria for adverse events (CTCAE) v5.0, cumulative incidences of bacterial, viral, and fungal infections, and failure to achieve neutrophil recovery (absolute neutrophil count \> 0.5 x 10\^9/L) or platelet recovery (platelet count \> 50 x 10\^9/L) after HSCT
Cumulative incidence and severity of acute and chronic GVHD assessed according to the 2014 NIH criteria
Time frame: From transplantation until the occurrence of GVHD or death from any cause, or up to 180 days after transplantation for acute GVHD, or up to 24 months for chronic GVHD, whichever occurs first
Acute GVHD grading should be performed by the MAGIC criteria, for chronic GVHD; the time of onset of chronic GVHD will be recorded, as well as the requirement for a systemic immunosuppressive therapy and the maximum grade achieved according to the NIH Consensus Criteria
Non-relapse mortality
Time frame: From transplantation until death without evidence of relapse or up to 24 months, whichever occurs first Description: NRM refers to death without evidence of disease relapse.
Not reported in this registry record.
Cumulative incidence of relapse
Time frame: From transplantation until the occurrence of relapse or up to 24 months, whichever occurs first
Cumulative incidence functions (CIFs) will estimate outcomes such as relapse
Disease-free survival (DFS)
Time frame: At two years
DFS is defined as survival without relapse or progression, the endpoints will be censored at two years to address differences in follow-up between groups
GVHD-free, relapse-free survival (GRFS)
Time frame: From transplantation until the occurrence of acute grade III-IV GVHD, severe chronic GVHD, relapse, death, or up to 24 months, whichever occurs first
GRFS encompasses survival free from acute grade III-IV GVHD, chronic GVHD requiring systemic immunosuppression, or relapse
Cost-effectiveness
Time frame: From transplantation until 2 years
The endpoint of the medico-economic analysis is the incremental cost-utility ratio (ICUR) at 24 months of reducing the total dose of PTCy to 70 mg/kg on GREFS compared to the standard dose of 100 mg/kg. The incremental cost-utility ratio will be calculated in cost per QALY gained.
The secondary endpoint is the incremental cost-effectiveness ratio (ICER) at 24 months. The incremental cost-effectiveness ratio will be calculated in cost per life-years gained.
Cytokine profiles
Time frame: Inclusion, Day 0, Day 15-35, Day 90, Day 365
This ancillary study will evaluate cytokine profiles in relation to PTCy doses using a multiplex test based on fluorescence-coded beads
Gut microbiota
Time frame: Inclusion, Day 0, Day 15-35, Day 90
This ancillary study will evaluate gut microbiota: richness based on α-diversity indexes
Review the complete result tables and limitations in the official record.
Open official results ↗Research locations (1)
The overall study status and a local site’s status can differ. Confirm that location’s availability before traveling.
1 locations
Saint Antoine Hospital - Hematology DepartmentParis, FranceRecruiting
Study team and timeline
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Study start
- Sep 29, 2026 Actual
- Primary completion
- 2030-09 Estimated
- Study completion
- 2030-09 Estimated
- First posted
- Sep 25, 2025 Actual
- Last updated
- Nov 10, 2026 Actual
Official study contacts
Official study title
Reduced Post-transplant Cyclophosphamide Dose in Patients Undergoing Haploidentical Hematopoietic Stem Cell Transplantation for Hematological Malignancies: a Phase III Randomized Study
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Source: ClinicalTrials.gov API v2. Information is submitted by sponsors and investigators. NLM does not approve the safety or science of all listed studies. Registry disclaimer.
Retrieved: Nov 12, 2026 · Last registry update: Nov 10, 2026