VENETOCLAX for Myelodysplastic syndrome | Chronic myelomonocytic leukemia | Secondary acute myeloid leukemia

Inclusion criteria

• {"criterion_text":"- MDS, CMML or sAML according to WHO classification (revised version 2016) with a marrow blast count >5% and/or high-risk genetic features (eg. bad risk karyotype according to the IPSS-R / ELN classification or presence of unfavorable somatic mutations (e.g. TP53, RUNX1, IDH1, IDH2, KMT2A, DEK-NUP214 or RAS pathway mutations including NRAS, KRAS, PTPN11, CBL, NF1, RIT1 or KIT), falling into the \"high\" or \"very high\" risk category of the IPSS-R or IPSS M) any time between diagnosis and inclusion."}

Exclusion criteria

• {"criterion_text":"- previous cytotoxic therapy exceeding oral Hydroxyurea or >2 courses of treatment with Azacytidine, Decitabine or low dose Ara-C alone or in combination with Venetoclax"}

Primary endpoints

• {"endpoint_text":"- safety, defined as the maximal organ toxicity to each organ system according to CTC criteria as well as the number of AEs of grade III or greater until day +30 (± 3) after transplantation","definition_or_measurement_approach":"Measured as maximal organ toxicity per organ system according to CTC criteria and number of adverse events (AEs) grade III or greater up to day +30 (±3) after transplantation."}

Secondary endpoints

• {"endpoint_text":"- safety, defined as the maximal organ toxicity to each organ system according to CTC criteria as well as the number of AEs of grade III or greater until day +100 (± 7) after transplantation","definition_or_measurement_approach":"Measured as maximal organ toxicity per organ system according to CTC criteria and number of AEs grade III or greater up to day +100 (±7) after transplantation."}
• {"endpoint_text":"- graft failure defined as no donor chimerism at day +30 (± 3) after transplantation","definition_or_measurement_approach":"Graft failure defined by absence of donor chimerism at day +30 (±3) post-transplant."}
• {"endpoint_text":"- incidence, course und severity of aGvHD and cGvHD during the first 2 years after transplantation","definition_or_measurement_approach":"Incidence, time course and severity of acute and chronic graft-versus-host disease assessed during 2-year follow-up after transplantation."}
• {"endpoint_text":"- incidence, course and severity of VOD","definition_or_measurement_approach":"Incidence, time course and severity of veno-occlusive disease (VOD) recorded post-transplant."}
• {"endpoint_text":"- time (days from day 0) to hematopoietic reconstitution (ANC>500/µl, PLT>20000/µl and PLT>50000/µl)","definition_or_measurement_approach":"Measured as days from day 0 to achieve ANC >500/µl and platelet thresholds >20,000/µl and >50,000/µl."}
• {"endpoint_text":"- time (days from day 0) to transfusion independence","definition_or_measurement_approach":"Measured as days from day 0 to cessation of transfusion requirement (transfusion independence)."}
• {"endpoint_text":"- best disease response within the first 100 days (± 7) after transplantation (according to IWG-criteria for MDS and ELN-criteria for AML)","definition_or_measurement_approach":"Best response per IWG criteria for MDS and ELN criteria for AML within 100 days (±7) after transplant."}
• {"endpoint_text":"- disease response and donor chimerism at day +30 (± 3), +60 (± 7) and +100 (± 7) after transplantation (according to IWG-criteria for MDS and ELN-criteria for AML)","definition_or_measurement_approach":"Disease response per IWG/ELN criteria and donor chimerism measured in blood/marrow at specified timepoints."}
• {"endpoint_text":"- time (days from day 0) to complete donor chimerism in blood and marrow","definition_or_measurement_approach":"Measured as days from day 0 to attainment of complete donor chimerism in blood and bone marrow."}
• {"endpoint_text":"- disappearance of molecular markers of disease (yes/no, time in days from day 0 )","definition_or_measurement_approach":"Assessment of clearance of molecular disease markers (yes/no) and time in days from day 0."}
• {"endpoint_text":"- event-free survival (EFS, death, relapse and disease progression will be recorded as event)","definition_or_measurement_approach":"EFS measured with events defined as death, relapse or disease progression."}
• {"endpoint_text":"- cumulative incidence of relapse (CIR, disease progression and relapse will be recorded as event)","definition_or_measurement_approach":"CIR measured with disease progression and relapse recorded as events."}
• {"endpoint_text":"- overall survival (OS, death will be recorded as event)","definition_or_measurement_approach":"Overall survival measured; death recorded as event."}

Germany

Earliest CTIS Part Ii Submission Date

22-07-2024

Latest Decision Or Authorization Date

06-05-2026

Processing Time Days

653

Number Of Sites

6

Number Of Participants

38

Primary sponsor

Full Name

Heinrich-Heine-Universitaet Duesseldorf

Organisation Type

Educational Institution

Country Of Registered Address

Germany

Third parties

• {"country":"","full_name":"AbbVie Deutschland GmbH & Co. KG","duties_or_roles":"Source of monetary support","organisation_type":""}
• {"country":"","full_name":"Eurocept International B.V.","duties_or_roles":"Source of monetary support","organisation_type":""}
• {"country":"","full_name":"Medac GmbH","duties_or_roles":"Source of monetary support","organisation_type":""}