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 1

• Conditions: Anemia in Patients with Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS).; MedDRA version: 27.0Level: PTClassification code 10028533Term: Myelodysplastic syndromeSystem Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); MedDRA version: 20.0Level: LLTClassification code 10002272Term: AnemiaSystem Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2021-001838-19-DE
• Lead Sponsor: Keros Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2021-09-29
• Last Update Posted: 8 July 2024

Recruitment & Eligibility

• Status: A
• Sex: All
• Target Recruitment: 141

Inclusion Criteria

1. Diagnosis of MDS (Parts 1 and 2) according to WHO classification that meets International Prognostic Scoring System-Revised (IPSS-R)
classification of very low, low, or intermediate risk disease.
2. < 5% blasts in bone marrow during the Pretreatment Period.
3. Peripheral blood white blood cell (WBC) count < 13,000/µL during the
Pretreatment Period.
4. Anemia defined as:
a. In LTB participants, having received 1 to 3 units of RBCs for Hgb = 9.0
g/dL within 8 weeks of the Pretreatment Period OR
b. In HTB participants (defined as requiring > or = 4 units of RBCs for hemoglobin < or = 9.0 g/dL within 8 weeks)
5. Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 (if related to anemia.
6. Females of child-bearing potential and sexually active males must agree to use effective methods of contraception.

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 34
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 76

Exclusion Criteria

1. Active infection requiring parenteral antibiotic therapy within 28 days
prior to C1D1 or oral antibiotics within 14 days of C1D1. Prophylactic
antibiotics and/or antifungals for neutropenia are allowed.
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 with ESA within 56 days prior to C5D1.
6. Prior or concurrent chronic treatment with G-CSF or GM-CSF.
7. Iron chelation therapy if initiated within 8 weeks prior to Cycle 1 Day
1.
8. Vitamin B12 therapy within 8 weeks prior to Cycle 1 Day 1.
9. 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 halflife
prior to Cycle 1 Day 1, whichever is longer.
10. Platelet count > 450 x 10*9/L or < 30 x 10*9/L.
11. Transferrin saturation < 15%.
12. Ferritin < 50 µg/L.
13. Folate < 4.5 nmol/L (< 2.0 ng/mL).
14. Vitamin B12 < 148 pmol/L (< 200 pg/mL).
15. Estimated glomerular filtration rate (GFR) < 40 mL/min/1.73 m2 (as
determined by the Chronic Kidney Disease Epidemiology Collaboration
[CKD-EPI].
16. Pregnant or lactating females.

Medical History
•Diagnosis of MDS with deletion of chromosome 5q (Del5q).
•Presence of uncontrolled heart disease or New York Heart Association Class III or IV heart failure.
•Presence of uncontrolled hypertension (Grade = 2 high blood
pressure).
•History of stroke, deep venous thrombosis, or arterial embolism within 6 months prior to C1D1(or C5D1 for the Part 1 Extension).
•Any malignancy other than MDS that has not been in remission and/or has required systemic therapy 1 year prior to C1D1 (or C5D1 for the Part 1 Extension).
•Diagnosis of secondary MDS.
•History of solid organ or hematological transplantation
•Body mass index = 40 kg/m2.

Treatment History
•Prior treatment with azacitidine, decitabine, lenalidomide, luspatercept, or sotatercept.
•Treatment with ESA within 8 weeks prior to C1D1 (or C5D1 for the Part 1 Extension).
•Prior or concurrent chronic treatment with granulocyte colony
stimulating factor (G-CSF) orgranulocyte-macrophage colony
stimulating factor (GM-CSF), for reasons other than thetreatment of
MDS.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified