Efficacy and Safety of Canakinumab for the Treatment of Anemia in LR-MDS Patients

NCT05237713 | Terminated Phase 2

• 1 other identifier: CANFIRE
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 3

Brief Summary

Hematologic improvement of erythrocytes after 6 months of canakinumab treatment.

Conditions

Anemia; Myelodysplastic Syndromes

Keywords

anemia; low risk MDS; inflammation; IL-1beta blockade; NLRP3 activation

Outcome Measures

Primary Outcomes (1)

• HI-E after 6 months of treatment

  Erythroid response rate (HI-E) of canakinumab

  6 months

Secondary Outcomes (5)

• HI-E response duration

  up to three years

• Number of (serious) adverse events

  up to three years

• Disease progression

  after 24 weeks

• Impact of canakinumab on quality of life by using the validated Quality of Life in Myelodysplasia Scale (QUALMS)

  From the date of treatment start until the end of study, assessed up to 36 months

• Impact of canakinumab on quality of life by using the validated European Organisation for Research and Treatment of Cancer Core Quality of Life questionnaire (EORTC QLQ-C30)

  From the date of treatment start until the end of study, assessed up to 36 months

Study Arms (1)

Canakinumab treatment

OTHER

200 mg canakinumab subcutaneously every three weeks

Drug: Canakinumab Injection

Interventions

Canakinumab Injection DRUG

Administration for a duration of 6 months for all patients and in case of response further treatment for up to three years

Also known as: Ilaris(R)

Canakinumab treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of
• Lower-risk myelodysplastic syndrome (MDS) OR
• Myelodysplastic/myeloproliferative neoplasm (MDS/MPN) including MDS/MPN-RS-T, MDS/MPNu, aCML, or CMML (as per the World Health Organization \[WHO\] 2016 classification) Note: Diagnoses will be confirmed by central morphological review during screening assessment
• Very low, low or intermediate risk disease MDS with up to 3.5 points according to the revised International Prognostic Scoring System (IPSS-R) classification (to be confirmed during screening assessment). For MDS/MPN \< 10% bone marrow blasts at screening. For CMML low or intermediate risk according to CMML-Specific Prognostic Scoring System (CPSS Score).
• the hemoglobin mean over the baseline period will be less than 10 g/dL OR three or more RBC-transfusions will have been given during the baseline period documenting transfusion dependence.
• Documented transfusion strategy: A transfusion trigger threshold is needed which characterizes the transfusion strategy - ideally for the whole baseline period, but at least for the time from registration to the end of the study.
• Relapsed / refractory / intolerant / ineligible (endogenous serum erythropoietin levels ≥ 200 U/L) to ESA treatment
• Age ≥ 18 years
• Written informed consent

You may not qualify if:

• Compliance with major study procedures
• Patient does not accept bone marrow sampling during screening and treatment period.
• Patient does not accept peripheral blood sampling for screening and during treatment.
• Patient does not accept subcutaneous application of canakinumab every three weeks
• Contraindications
• Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
• a. iron deficiency must be determined by calculated transferrin saturation (iron/total iron binding capacity) ≤ 20%, or serum ferritin ≤ 15 µg/L
• Prior allogeneic or autologous stem cell transplant
• Known history of diagnosis of AML
• Safety
• Severe neutropenia defined by ANC ≤ 0.5 Gpt/l
• Severe thrombocytopenia with platelets (PLT) \< 30 Gpt/L
• Serum creatinine \> 1.5x ULN OR measured or calculated creatinine clearance \< 40 ml/min (NOTE: creatinine clearance should be calculated per institutional standard. GFR can also be used)
• Serum aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT) or alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) ≥ 3.0 x upper limit of normal (ULN) - both have to be measured
• Eastern Cooperative Oncology Group (ECOG) \> 2

Sponsors & Collaborators

• University of Leipzig: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (3)

Medizinisches Versorgungszentrum "Onkologischer Schwerpunkt am Oskar- Helene-Heim"

Berlin, 14195, Germany

Location

Universitätsklinikum Carl Gustav Carus an der Technischen Universität Dresden Medizinischen Klinik und Poliklinik I / Hämatologie

Dresden, 01307, Germany

Location

Klinik und Poliklinik für Hämatologie und Zelltherapie, Hämostaseologie

Leipzig, 04318, Germany

Location

MeSH Terms

Conditions

Anemia; Myelodysplastic Syndromes; Inflammation

Interventions

canakinumab

Condition Hierarchy (Ancestors)

Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Anne Sophie Kubasch, Dr.

  University Leipzig

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Coordinating Investigator

Study Record Dates

• First Submitted: January 21, 2022
• First Posted: February 14, 2022
• Study Start: April 26, 2022
• Primary Completion: February 29, 2024
• Study Completion: February 29, 2024
• Last Updated: May 6, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

DE (3)