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)

Phase 2

Recruiting

• Conditions: Myelodysplastic Syndromes; Blood - Anaemia; Blood - Haematological diseases; Blood - Normal development and function of platelets and erythrocytes

• Registration Number: ACTRN12620000696998
• Lead Sponsor: Keros Therapeutics Australia Pty Ltd

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-06-22
• Last Update Posted: 5 July 2021

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 54

Inclusion Criteria

1.Male or female > or = 18 years of age, at the time of signing informed consent.
2.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.
3.< 5% blasts in bone marrow.
4.Peripheral blood white blood cell (WBC) count < 13,000/µL.
5.Anemia defined as:
-In LTB participants (defined as having received < 4 units of RBCs within 8 weeks), mean hemoglobin concentration < 10.0 g/dL of two measurements (one performed within 1 day prior to Cycle 1 Day 1 and the other performed 7-28 days prior, not influenced by RBC transfusion within 7 days of measurement) OR
-In HTB participants (defined as requiring > or = 4 units of RBCs for hemoglobin < or = 9.0 g/dL within 8 weeks)
6.Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia).
7.Females of child-bearing potential and sexually active males must agree to use effective methods of contraception.

Exclusion Criteria

1. 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.
2. Diagnosis of secondary MDS (ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases).
3. Vitamin B12 deficiency.
4. Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
5. Treatment within 28 days prior to Cycle 1 Day 1 with:
a. Erythropoiesis stimulating agent (ESA) OR
b. Granulocyte colony-stimulating factor (G-CSF) OR
c. Granulocyte-macrophage colony-stimulating factor (GM-CSF)
6. Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day 1.
7. Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
8. 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.
9. Platelet count > 450 x 10*9/L or < 30 x 10*9/L.
10. Transferrin saturation < 15%.
11. Ferritin < 50 µg/L.
12. Folate < 4.5 nmol/L (< 2.0 ng/mL).
13. Vitamin B12 < 148 pmol/L (< 200 pg/mL).
14. Estimated glomerular filtration rate (GFR) < 40 mL/min/1.73 m2 (as determined by the Chronic Kidney Disease Epidemiology Collaboration [CKD-EPI].
15. Pregnant or lactating females.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Evaluate safety and tolerability of KER-050 in participants with very low, low, or intermediate risk MDS.<br><br>To be assessed by monitoring<br>• Incidence and severity of AE;<br>• Incidence of serious adverse events (SAE) and suspected unexpected serious adverse reactions;<br>• Clinically significant changes from baseline in:<br>- Laboratory evaluations (hematology, chemistry, urinalysis, urine collection, coagulation, lipid profile tests);<br>- Electrocardiograms (ECG);<br>- Vital signs;<br>- Physical examinations;<br>- Eastern Cooperative Oncology Group (ECOG) Performance Status; <br>- Anti-drug antibody.[Monitored from time of signing informed consent throughout the course of the study through Day 183.];Progression to higher-risk MDS/AML. Assessed based on progression to AML as per WHO classification, further confirmatory histology or cytology results, etc. may be requested. [Assessed at screening, Day 99 and Day 183.]