An Open-label, Phase 1b Study of R289, an IRAK1/4 Inhibitor, in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS) Who Are Refractory/Resistant to Prior Therapies

  • STATUS
    Recruiting
  • End date
    May 31, 2024
  • sponsor
    Rigel Pharmaceutical
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Updated on 15 September 2022
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Summary

The study will be an open-label, Phase 1b study of R289 to determine tolerability and preliminary efficacy in patients with LR MDS who are relapsed, refractory/resistant, intolerant, or have inadequate response to prior therapies such as erythropoietin (EPO), thrombopoietin (TPO), luspatercept, or hypomethylating agents (HMAs) for MDS.

Description

An Open-label, Phase 1b Study of R289, an IRAK1/4 Inhibitor, in Patients With Lower-risk Myelodysplastic Syndromes (LR MDS) Who Are Refractory/Resistant to Prior Therapies

Details
Condition Low Risk Myelodysplastic Syndromes, Hematology, Myelodysplastic Syndromes (MDS)
Clinical Study IdentifierTX306123
SponsorRigel Pharmaceutical
Last Modified on15 September 2022

Eligibility

 Yes No Not Sure

Inclusion Criteria

Patient must be ≥ 18 years of age at the time of signing the informed consent
Must have definitive diagnosis of MDS with very low, low, or intermediate-1 risk (International Prognostic Scoring System (IPSS)-R ≤ 3.5) and ≤5% bone marrow myeloblasts
Must be relapsed, refractory/resistant, intolerant, or have inadequate response to therapies with known clinical benefits for MDS, such as TPOs, EPOs, luspatercept, and HMAs (i.e., azacytidine or decitabine). Patients with del (5q) must have failed prior lenalidomide therapy
Must meet at least one of the disease-related criteria for RBC transfusion, platelet count, or absolute neutrophil (ANC) within 8 weeks prior to initial administration of study treatment
Symptomatic anemia untransfused with hemoglobin < 9.0 g/dL within 8 weeks of registration or red blood cell (RBC) transfusion dependent defined as receiving > 3 units of packed red blood cells (PRBCs) in the preceding 16 weeks for a hemoglobin <9.0 g/dL
Clinically relevant thrombocytopenia (platelet counts of <100 × 109/L in at least 2 blood counts prior to study treatment and transfusion dependence)
Absolute neutrophil count (ANC) of < 1.0 × 109/L in at least 2 blood counts prior to randomization
Must have Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2 at screening
Must have Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2 at screening
Must have adequate organ function, defined as
Hepatic function
aspartate amino transferase (AST) or alanine aminotransferase (ALT) ≤ 1.5 × upper limit of normal (ULN)
total bilirubin ≤ 1.5 × ULN
Renal function defined as creatinine clearance > 60 mL/min (using Cockcroft-Gault), or blood creatine < 1.5 mg/dL

Exclusion Criteria

Prior treatment for MDS (i.e., TPOs, EPOs, luspatercept, HMAs) concluded < 4 weeks prior to study treatment
Clinically significant anemia resulting from iron, B12 or folate deficiencies, autoimmune or hereditary hemolysis, or GI bleeding
All subjects must have documented marrow iron stores. If marrow iron stain is not available, the transferrin saturation must be > 20% or a serum ferritin > 100 ng/100 mL
MDS secondary to treatment with radiotherapy, chemotherapy, and/or immunotherapy for malignant or autoimmune diseases
Diagnosis of chronic myelomonocytic leukemia
History of uncontrolled seizures
Uncontrolled bacterial or viral infection (i.e., documented HIV, hepatitis B or hepatitis C)
History of an active malignancy within the past 2 years prior to study entry, with the exception of
Adequately treated in situ carcinoma of the cervix uteri
Adequately treated basal cell carcinoma or localized squamous cell carcinoma of the skin, or
Any other malignancy with a life expectancy of more than 2 years
History of or active, clinically significant, cardiovascular, respiratory, GI, renal, hepatic, neurological, psychiatric, musculoskeletal, genitourinary, dermatological, or other disorder that, in the Investigator's opinion, could affect the conduct of the study or the absorption, metabolism or excretion of the study treatment
Prior history of bone marrow transplantation
Marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval > 480 milliseconds [msec]) (Common Terminology Criteria for Adverse Events [CTCAE] Grade 1) using Fridericia's QT correction formula
History of additional risk factors for TdP (e.g., heart failure, hypokalemia, family history of Long QT Syndrome)
Treatment with cytotoxic chemotherapeutic agents or experimental agents for the treatment of MDS within 8 weeks of study treatment
Receiving any other concurrent chemotherapy, radiotherapy, or immunotherapy (within 8 weeks of initiating study treatment)
Use of concomitant medications that prolong the QT/QTc interval during study treatment
Use of concomitant medications that are strong CYP3A or CYP2B6 inhibitors or inducers during study treatment
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