A Phase II, Single-Arm, Open-Label Study to Assess the Efficacy and Safety of Canakinumab for the Treatment of Anemia in Patients with IPSS-RVery Low, Low, or Intermediate Risk Myelodysplastic Syndromes or MDS/MP

Phase 1

Conditions: ower-risk MDS or MDS/MPN
Therapeutic area: Not possible to specify

Registration Number: CTIS2024-515886-33-00

Lead Sponsor: niversitaet Leipzig

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 11

Inclusion Criteria

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), 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, Symptomatic anemia (based on complete Hb time course and transfusion history), Relapsed / refractory / intolerant / ineligible (sEPO = 200 U/L) to ESA treatment, Age = 18 years, Written informed consent

Exclusion Criteria

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To study the erythroid response rate (HI-E) of canakinumab in patients with IPSS-R very low, low, or intermediate risk MDS or MDS/MPN.;Secondary Objective: To study the HI-E response duration, To study the safety and toxicity of single agent canakinumab, To study the proportion of disease progression, To assess the impact of canakinumab on quality of life (QoL) over 6 months post screening;Primary end point(s): 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.

Secondary Outcome Measures