Health TrialsbyHealthPanel

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

ConditionsGVHD - Graft-Versus-Host DiseaseHSCTHaploidentical Stem Cell Transplantation
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18+
SponsorAssistance Publique - Hôpitaux de Paris

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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

Trial design

Design model

Parallel

Treatments tested in this trial

  • 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).

Treatment groups

180 Participants
are divided into 2 treatment groups
Group A: Reduced doseExperimental treatment 1 intervention
Group B: Standard doseActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

GVHD-free, relapse-free, event-free survival (GREFS)

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

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

Secondary outcomes

1

Overall survival (OS)

Overall survival (OS) at 2 years is defined as survival irrespective of disease status

Time frame
From transplantation until death from any cause or up to 24 months, whichever occurs first
2

Quality of life FACT-BMT

Quality of life compared to baseline using questionnaire: FACT-BMT (Functional Assessment of Cancer Therapy - Bone Marrow Transplant, version 4)

Time frame
At 1, 3, 6, 12, and 24 months after HSCT
3

Quality of life EQ-5D-5L

Quality of life compared to baseline using questionnaire: EQ-5D-5L (EuroQol 5-Dimension, 5-Level questionnaire).

Time frame
At 1, 3, 6, 12, and 24 months after HSCT
4

Toxicities, infection and hematogical recovery

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

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Assistance Publique - Hôpitaux de Paris

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: