CYCLOPHOSPHAMIDE for Hematological malignancy

Inclusion criteria

• {"criterion_text":"- Age ≥ 18 years"}
• {"criterion_text":"- Confirmed hematological malignancy with an indication for allogeneic HSCT"}
• {"criterion_text":"- Presence of a haploidentical donor willing to donate PBSC"}
• {"criterion_text":"- Patient planned to receive a thiotepa-based conditioning regimen"}
• {"criterion_text":"- Provision of written informed consent"}
• {"criterion_text":"- Affiliation to a social security system (excluding “Aide Médicale d’État”)"}

Exclusion criteria

• {"criterion_text":"- Karnofsky performance status < 70%"}
• {"criterion_text":"- Concurrent participation in another investigational therapeutic study"}
• {"criterion_text":"- Inability to comply with study procedures"}
• {"criterion_text":"- Life expectancy < 1 month, as determined by the attending physician"}
• {"criterion_text":"- Acute or chronic heart failure, defined as left ventricular ejection fraction < 40%"}
• {"criterion_text":"- Pulmonary dysfunction with diffusion capacity < 50% of predicted values"}
• {"criterion_text":"- Renal impairment with estimated glomerular filtration rate (eGFR) < 45 mL/min (calculated using the CKD-EPI formula)"}
• {"criterion_text":"- Positive HIV status"}
• {"criterion_text":"- Pregnancy, breast-feeding, or refusal to use effective contraception"}
• {"criterion_text":"- 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)"}
• {"criterion_text":"- Hypersensitivity to the active substance or any of the excipients"}

Primary endpoints

• {"endpoint_text":"- 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","definition_or_measurement_approach":"Composite endpoint defined as the probability of survival without severe GVHD, relapse/progression of the hematological malignancy, or PTCy-associated adverse event, assessed at 2 years after HSCT."}

Secondary endpoints

• {"endpoint_text":"- Overall survival (OS) at 2 years: From transplantation until death from any cause or up to 24 months, whichever occurs first","definition_or_measurement_approach":"From transplantation until death from any cause or up to 24 months."}
• {"endpoint_text":"- Quality of life at 1, 3, 6, 12, and 24 months after HSCT compared to baseline using two questionnaires: FACT-BMT (Functional Assessment of Cancer Therapy - Bone Marrow Transplant, version 4) and EQ-5D-5L (EuroQol 5-Dimension, 5-Level questionnaire).","definition_or_measurement_approach":"Assessed using FACT-BMT v4 and EQ-5D-5L at 1, 3, 6, 12 and 24 months versus baseline."}
• {"endpoint_text":"- 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 109/L) or platelet recovery (platelet count > 50 x 109/L) after HSCT 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","definition_or_measurement_approach":"Organ toxicities by CTCAE v5.0; infections cumulative incidence; hematological recovery defined as ANC > 0.5 x10^9/L and platelets >50 x10^9/L. Timeframes: up to event, day +60 for hematological recovery, up to 24 months for organ toxicities/infections."}
• {"endpoint_text":"- Cumulative incidence and severity of acute and chronic GVHD: 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","definition_or_measurement_approach":"Cumulative incidence and grade of acute GVHD (up to 180 days) and chronic GVHD (up to 24 months) from transplantation until event or death."}
• {"endpoint_text":"- Non-relapse mortality (NRM) at 2 years: From transplantation until death without evidence of relapse or up to 24 months, whichever occurs first","definition_or_measurement_approach":"Death without relapse from transplantation up to 24 months."}
• {"endpoint_text":"- Cumulative incidence of relapse at 2 years: From transplantation until the occurrence of relapse or up to 24 months, whichever occurs first","definition_or_measurement_approach":"Occurrence of relapse from transplantation up to 24 months."}
• {"endpoint_text":"- Disease-free survival (DFS) at 2 years: From transplantation until relapse or progression of the hematological disease, death, or up to 24 months, whichever occurs first","definition_or_measurement_approach":"Time from transplantation to relapse/progression, death, or up to 24 months."}
• {"endpoint_text":"- GVHD-free, relapse-free survival (GRFS) at 2 years: From transplantation until the occurrence of acute grade III-IV GVHD, severe chronic GVHD, relapse, death, or up to 24 months, whichever occurs first","definition_or_measurement_approach":"Composite endpoint: time to acute grade III-IV GVHD, severe chronic GVHD, relapse, or death, assessed up to 24 months."}
• {"endpoint_text":"- Incremental cost-effectiveness (cost/life year) and cost-utility (cost/quality-adjusted life-year, QALY) ratios at 2 years.","definition_or_measurement_approach":"Health economic evaluation using cost per life-year and cost per QALY at 2 years."}

France

Earliest CTIS Part Ii Submission Date

16-06-2025

Latest Decision Or Authorization Date

11-07-2025

Processing Time Days

25

Number Of Sites

16

Number Of Participants

180

Primary sponsor

Full Name

Assistance Publique Hopitaux De Paris

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

France