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AnemiaPhase 2NCT05237713

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

A Phase 2 study testing Canakinumab Injection in people with Anemia and Myelodysplastic Syndromes, run by University of Leipzig at 3 sites in Germany.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

Hematologic improvement of erythrocytes after 6 months of canakinumab treatment. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Anemia, Myelodysplastic Syndromes
What is being tested?
Canakinumab Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
3 sitesGermany
Study dates
26 Apr 2022to 29 Feb 2024
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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).
  • Symptomatic anemia (all NTD, LTB, or HTB): has to be documented in the 16 weeksbaseline period ending on the day of inclusion. Patients should be registered only if it is expected at time of registration that
  • a valid and complete hemoglobin and transfusion history will be available at inclusion AND
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  • 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

Who cannot join

  • Patients meeting any of the following exclusion criteria are not to be enrolled in the trial.
  • 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
Show all 50Show fewer
  • Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
  • 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
  • Total bilirubin ≥ 2.0 x ULN
  • higher levels are acceptable if these can be attributed to active red blood cell precursor destruction within the bone marrow (ie, ineffective erythropoiesis).
  • subjects are excluded if there is evidence of autoimmune hemolytic anemia manifested as a corrected reticulocyte count of > 2% with either a positive Coombs' test or over 50% indirect bilirubin
  • Active second malignancy at time of study entry
  • Prior history of malignancies, other than MDS, unless the subject has been free of the disease for ≥ 5 years. However, subjects with the following history/concurrent conditions are allowed:
  • Basal or squamous cell carcinoma of the skin
  • Carcinoma in situ of the cervix
  • Carcinoma in situ of the breast
  • Incidental histologic finding of prostate cancer (T1a or T1b using the tumor, nodes, metastasis [TNM] clinical staging system)
  • Major surgery within 8 weeks prior to screening (NOTE: Subjects must have completely recovered from any previous surgery prior to inclusion)
  • Myocardial infarction, uncontrolled angina, uncontrolled heart failure, or uncontrolled cardiac arrhythmia within 6 months prior to screening
  • Positive test result as an indicator for active or latent tuberculosis (evaluation performed per local treatment guidelines or clinical practice)
  • Subjects with suspected or proven immunocompromised state or infections, including:
  • Known history of testing positive for Human Immunodeficiency Virus (HIV) infections.
  • Known active or recurrent, hepatitis B and C (positive or indeterminate laboratory results).
  • Those with any other medical condition such as active infection, treated or untreated, which in the opinion of the investigator places the subject at an unacceptable risk for participation in immunomodulatory therapy.
  • Those requiring systemic or local treatment with any immune modulating agent in doses with systemic effects e.g.: Prednisone >20 mg (or equivalent) oral or intravenous daily for >14 days; Prednisone > 5 mg and ≤ 20 mg (or equivalent) daily for > 30 days; Equivalent dose of methotrexate >15 mg weekly.
  • History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational product (see Investigator´s Brochure).
  • Excluded treatments before and during study treatment
  • Anticancer cytotoxic chemotherapeutic agent or treatment for at least 14 days prior to registration and during study treatment
  • Corticosteroids or other immunosuppressive therapies (TNF-Blocker, other IL-1 Blocking Agent, Disease-modifying anti-rheumatic drugs (DMARDs) including ciclosporin, cyclophosphamide, hydroxychloroquine, leflunomide, methotrexate, mycophenolate, sulfasalazine) for at least 14 days prior to registration and during study treatment
  • Treatment with ESAs, luspatercept, granulocyte colony-stimulating factor (G-CSF), granulocyte-macrophage colony-stimulating factor (GM-CSF) for at least 14 days prior to registration and during study treatment
  • Treatment with disease modifying agents (eg, immune-modulatory drugs [IMiDs such as lenalidomide, hypomethylating agents] or experimental agents for underlying MDS disease for at least 14 days prior to registration and during study treatment
  • Prior treatment with canakinumab
  • Live vaccination during study treatment
  • Special considerations for females
  • Pregnant or breastfeeding females
  • Positive pregnancy test in women of childbearing potential NOTE: Urine or serum pregnancy test should be repeated within 3 days prior to receiving study treatment. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test is required additionally
  • Female subjects of childbearing potential unwilling to use an adequate method of contraception for the course of the study through 90 days after the last dose of study medication NOTE: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject
  • Special considerations for males
  • Male subjects with procreative capacity not willing to use an adequate method of contraception, starting with the first dose of study therapy through 90 days after the last dose of study therapy NOTE: Abstinence is acceptable if this is the usual lifestyle and preferred contraception for the subject
  • Regulatory requirements
  • Participation in other interventional trials
  • Patients under legal supervision or guardianship

Trial locations and contacts

3 sites in 1 country.

Germany3 sites

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

    Berlin, 14195

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

    Dresden, 01307

  • Klinik und Poliklinik für Hämatologie und Zelltherapie, Hämostaseologie

    Leipzig, 04318

Other trials in Germany

About this study

Official title: 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-R Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes or MDS/MPN

Hematologic improvement of erythrocytes after 6 months of canakinumab treatment.

Read the detailed description

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 after 6 months of treatment.

Being tested
Canakinumab Injection
Keywords
anemia, low risk MDS, inflammation, IL-1beta blockade, NLRP3 activation
Medical subject headings
Anemia, Myelodysplastic Syndromes, Inflammation

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Canakinumab InjectionDrug

    Also called Ilaris(R)

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

Study groups in NCT05237713
Study groupRoleWhat they receive
Canakinumab treatmentOther200 mg canakinumab subcutaneously every three weeks
Receives: Drug: Canakinumab Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. HI-E after 6 months of treatment

    Erythroid response rate (HI-E) of canakinumab

    Measured: 6 months

Secondary outcomes (5)

  1. HI-E response duration

    Duration of erythroid response rate will be measured up to loss of response or reaching end of study (after max. 3 years of treatment)

    Measured: up to three years

  2. Number of (serious) adverse events

    The safety profile of canakinumab will be described by collecting AE (adverse event) and SAE (serious adverse event) information up to the start of new treatment or reaching end of study (after max. 3 years of treatment). Special consideration will be laid on events that lead to treatment discontinuation.

    Measured: up to three years

  3. Disease progression

    Proportion of disease progression (after reaching PD at any time during the trial after primary end-point visit)

    Measured: after 24 weeks

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

    QoL assessment using the QUALMS questionnaire up to end of treatment:

    38-item assessment tool for patients with Myelodysplastic Syndromes (MDS) QUALMS scores ranged from 24 to 99, with higher scores for better outcome

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

  5. 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)

    QoL assessment using the EORTC-C30 questionnaire up to end of treatment:

    To assess patient-reported quality of life during canakinumab treatment: 30 questions assessing the quality of life of oncology patients across 10 subscales will be analyzed. All subscales have a score range from 0 to 100 points.

    Function subscales: a higher score represents a higher quality of life. Symptoms subscales: higher score represents higher level of symptoms/problems, i.e., represents lower quality of life

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

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Feb 2022
  2. Study start26 Apr 2022
  3. Primary completion29 Feb 2024
  4. Study completion29 Feb 2024

Why it stopped: Slow recruitment. It is not considered likely that the CANFIRE trial will be able to reach the target number of patients. Clinical responders will be further treated within the trial (expected duration: 3 years after stop of enrollment).

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05237713
Sponsor's protocol ID
CANFIRE
Status last verified
May 2024
First submitted
21 Jan 2022
Last updated
6 May 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Platzbecker AS, Zoldan K, Brauer D, Kiewe P, Sasca D, Sockel K, Illmer T, Giagounidis A, Nowak D, Rieger K, Wortmann F, Gotze K, Schmidt-Hieber M, Mayer K, Vehling-Kaiser U, Weide R, Mahlmann S, Przybilla J, Melzer S, Cross M, Schneider M, Hasenclever D, Metzeler KH, Platzbecker U. Canakinumab or emavusertib for anemia in lower-risk MDS: results of the CANFIRE and LUCAS phase 2, open-label, multicenter trials. Commun Med (Lond). 2026 Sep 16;6(1):486. doi: 10.1038/s43856-026-01885-z.

    PubMed 42749863

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Canakinumab Injection in people with Anemia and Myelodysplastic Syndromes, run by University of Leipzig at 3 sites in Germany.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 50 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 6 May 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Slow recruitment. It is not considered likely that the CANFIRE trial will be able to reach the target number of patients. Clinical responders will be further treated within the trial (expected duration: 3 years after stop of enrollment).). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

HI-E after 6 months of treatment (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05237713, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 May 2024; AzTrials last refreshed its copy on 8 Nov 2026.

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