Canakinumab for Myelodysplastic syndromes (lower-risk) | Myelodysplastic/myeloproliferative neoplasm (MDS/MPN) | MDS/MPN-RS-T | MDS/MPNu | Atypical chronic myeloid leukaemia (aCML) | Chronic myelomonocytic leukaemia (CMML)

Inclusion criteria

• {"criterion_text":"- Cytomorphology based confirmed diagnosis of lower-risk MDS or MDS/MPN including MDS/MPN-RS-T, MDS/MPNu, aCML or CMML (as per the World Health Organization [WHO] 2016 classification)\n- IPSS-R up to 3.5: very low/low/intermediate risk disease (MDS); MDS/MPN < 10% bone marrow blasts; CPSS-Score low/intermediate for CMML\n- Symptomatic anemia (based on complete Hb time course and transfusion history)\n- Relapsed / refractory / intolerant / ineligible (sEPO ≥ 200 U/L) to ESA treatment\n- Age ≥ 18 years\n- Written informed consent"}

Exclusion criteria

• {"criterion_text":"- Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding\n- Active second malignancy\n- Prior or concomitant treatment with anticancer medications or disease modifying agents\n- Previously treated with canakinumab\n- Prior allogeneic or autologous stem cell transplant\n- Known history of diagnosis of AML\n- Severe neutropenia (ANC  0.5 Gpt/L)\n- Severe thrombocytopenia (PLT <30 Gpt/L)\n- Serum creatinine > 1.5x ULN OR measured or calculated creatinine clearance < 40 ml/min\n- AST or ALT ≥ 3.0 x ULN\n- ECOG > 2\n- Total bilirubin ≥ 2.0 x ULN"}

Primary endpoints

• {"endpoint_text":"- The primary clinical endpoint “documented achievement of erythroid response (HI-E)” is based on internationally accepted and validated IWG 2018 response criteria and defined as an increase in hemoglobin levels by at least 1.5 g/dL, persistent for at least 8 weeks over the baseline hemoglobin level (mean over 16 weeks prior to inclusion) for NTD patients or no transfusions within a 8 week-window for LTB and HTB (major response), and will be determined after 8 cycles of canakinumab treatment.","definition_or_measurement_approach":"Based on IWG 2018 response criteria: increase in hemoglobin ≥1.5 g/dL sustained for ≥8 weeks over baseline (mean over 16 weeks prior to inclusion) for non-transfusion-dependent (NTD) patients; or no transfusions within an 8-week window for low/transfusion burden (LTB) and high/transfusion burden (HTB) (major response). Determination after 8 cycles of canakinumab treatment."}

Germany

Earliest CTIS Part Ii Submission Date

26-06-2024

Latest Decision Or Authorization Date

20-05-2025

Processing Time Days

328

Number Of Sites

2

Number Of Participants

11

Primary sponsor

Full Name

Universitaet Leipzig

Organisation Type

Educational Institution

Country Of Registered Address

Germany