A Phase 2, Open-Label, Ascending Dose Study of KER-050 for the Treatment of Anemia in Patients with Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)

Cancer Categories

Karmanos Trial ID

NCT ID

Age Group

Scope

Phase

Principal Investigator

Primary Objectives:

Part 1 Dose Escalation:

Safety: To evaluate the safety and tolerability of ascending doses of KER-050 in participants with very low, low, or intermediate risk MDS in order to determine the dose(s) that will be evaluated in Part 2 of
the study

Part 2 Dose Confirmation:

Safety: To confirm the safety and tolerability of the dose(s) selected in Part 1

Long-term Extension:

Safety: To evaluate the long-term safety and tolerability of KER-050 in participants with very low, low, or intermediate risk MDS in the LTE

Secondary Objectives (all parts):

Safety: To evaluate the progression to higher risk MDS or AML
Efficacy: To evaluate the efficacy of KER-050 on anemia in participants with very low, low, or intermediate risk MDS, separately for RS-positive and non-RS populations
Pharmacodynamic: To evaluate the PD effects of KER-050 on erythropoiesis in participants with very low, low, or intermediate risk MDS, separately for RS-positive and non-RS populations

Eligibility

Inclusion Criteria:

Diagnosis of MDS according to World Health Organization (WHO)/French American British (FAB) classification that meets Revised International Prognostic Scoring System (IPSS-R) classification of very low, low, or intermediate risk disease.
< 5% blasts in bone marrow.
Peripheral blood white blood cell count <13,000/µL.
Anemia defined as:

In non-transfused participants, having received no red blood cell (RBC) transfusions within 8 weeks Hgb concentration ≤ 10.0 g/dL OR
In LTB participants, having received 1 to 3 units RBCs for Hgb ≤ 9.0 g/dL within 8 weeks OR
In HTB participants, having received ≥ 4 units of RBCs for Hgb ≤ 9.0 g/dL within 8 weeks

Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia.
Females of child-bearing potential and sexually active males must agree to use effective methods of contraception.

Exclusion Criteria:

Any active infection requiring parenteral antibiotic therapy within 28 days prior to Cycle 1 Day 1 or oral antibiotics within 14 days of Cycle 1 Day 1.
Diagnosis of secondary MDS (i.e., MDS known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
Vitamin B12 deficiency.
Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
Treatment within 28 days prior to Cycle 1 Day 1 with:

Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.
Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
Treatment with another investigational drug or device or approved therapy for investigational use < or = 28 days prior to Cycle 1 Day 1, or if the half-life of the previous product is known, within 5 times the half-life prior to Cycle 1 Day 1, whichever is longer.
Platelet count > 450 x 10*9/L or < 30 x 10*9/L.
Transferrin saturation < 15%.
Ferritin < 50 µg/L.
Folate < 4.5 nmol/L (< 2.0 ng/mL).
Vitamin B12 < 148 pmol/L (< 200 pg/mL).
Estimated glomerular filtration rate (GFR) < 40 mL/min/1.73 m2 (as determined by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI].

Locations

3520 Forest Rd.
Lansing, MI 48910
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Phone: 517-975-9500

Applicable Disease Site

Therapies, Drugs, Devices