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No longer recruiting (closed or complete)Last updated: 7 February 2024

This phase I study is evaluating how safe and tolerable different targeted therapies (MBG453, NIS793 and canakinumab) are alone, or combined, in people with lower risk myelodysplastic syndromes.A Phase Ib, Multicenter, Open-label Platform Study of Select Drug Combinations in Adult Patients With Lower Risk (Very Low, Low, or Intermediate Risk) Myelodysplastic Syndrome

Trial purpose

Medical clipboardCancer treatment

Tumor type

Blood Cancers Haematological

Age

People18+

Clinical summary

Summary

Eligible participants will be assigned to one of five experimental arms. In Experimental Arm 1, participants will receive MBG453 alone, every 4 weeks, to confirm safety and tolerability of RD. In Experimental Arm 2, participants will receive NIS793 alone, every 3 weeks, to establish RD in this indication and confirm safety and tolerability. In Experimental Arm 3, participants will receive canakinumab alone, every 4 weeks, to confirm safety and tolerability of RD. In Experimental Arm 4, participants will receive MBG453 + NIS793 combined treatment every 3 weeks to confirm safety and tolerability of combination RD. In Experimental Arm 5 MBG453 + canakinumab combined treatment every 4 weeks to confirm safety and tolerability of combination RD.

Conditions

This trial is treating people with very low, low and intermediate risk myelodisplastic syndrome

Eligibility

Inclusion

  1. Signed informed consent must be obtained prior to participation in the study.
  2. Patients must be ≥ 18 years of age at the time of signing the informed consent form (ICF).
  3. Patients must have a diagnosis prior to participation in the study of IPSS-R very low, low, or intermediate risk MDS with ≤10% bone marrow blasts and one or more of the following:

    1. Symptomatic anemia with hemoglobin <10 g/dL that has relapsed after or is refractory to ESAs (or the patient is intolerant to ESAs)
    2. Symptomatic anemia with hemoglobin <10 g/dL) that is ESA-naive with EPO level ≥ 500 /uL
    3. Thrombocytopenia with platelets <30,000/uL or with clinically significant bleeding or bruising and platelets <50,000/uL
    4. Neutropenia with an absolute neutrophil count (ANC) <500/ µL or with recurrent and/or severe infections and an ANC that is <1000/ µL and amenable to response assessments by International Working Group (IWG) response criteria in myelodysplasia (Cheson et al 2006)
  4. Patients who are refractory to, intolerant of, or ineligible/unable to receive SOC therapeutic options including lenalidomide
  5. Eastern Cooperative Oncology Group (ECOG) performance status (PS) ≤2
  6. Patient must be a candidate for serial bone marrow aspirate and/or biopsy according to the institutions' guidelines and be willing to undergo a bone marrow aspirate and/or biopsy at screening, during and at the end of therapy on this study -

Exclusion

  1. Systemic antineoplastic therapy (including cytotoxic chemotherapy, alpha-interferon, kinase inhibitors or other targeted small molecules, and toxin-immunoconjugates) or any experimental therapy within 14 days or 5 half-lives, whichever is longer, before the first dose of study treatment.
  2. History of hypersensitivity to any of the study treatments or its excipients or to drugs of similar chemical classes.
  3. Patients with chronic myelomonocytic leukemia (CMML) or myelodysplastic/myeloproliferative neoplasms (MDS/MPN)
  4. Use of hematopoietic colony-stimulating growth factors (e.g. G-CSF, GM-CSF, M-CSF), thrombopoietin mimetics or ESAs anytime ≤ 2 weeks (or 5 half-lives, whichever is longer) prior to start of study treatment.
  5. Systemic chronic corticosteroid therapy (>10 mg/day prednisone or equivalent) or any immunosuppressive therapy within 7 days of first dose of study treatment. Topical, inhaled, nasal and ophthalmic steroids are allowed.
  6. For arms containing canakinumab: Patients with ANC < 500 /µL

Inclusion

  • You have been diagnosed with cancer, but have not received any treatment.
  • You have had treatment but your cancer has gotten worse or has not responded to the treatment you have been given.

Exclusion

  • You have certain types of non-cancer medical conditions.
  • You have had certain treatments, surgical procedures or drugs.
Message

Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.

Participating hospitals

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More information

Trial Identifiers

Information on this page is partially produced from ClinicalTrials.gov *. View further details about this trial on the registry via the links below:

Trial sponsor

Novartis Pharmaceuticals

Scientific Title

A Phase Ib, Multicenter, Open-label Platform Study of Select Drug Combinations in Adult Patients With Lower Risk (Very Low, Low, or Intermediate Risk) Myelodysplastic Syndrome

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