IRIDA Study of KER-047

Phase 1

• Conditions: Iron-Refractory Iron-Deficiency Anemia (IRIDA); MedDRA version: 20.0Level: PTClassification code 10022972Term: Iron deficiency anaemiaSystem Organ Class: 10005329 - Blood and lymphatic system disorders; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2021-000348-22-ES
• Lead Sponsor: Keros Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-08-10
• Last Update Posted: 4 March 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Participants are eligible for the study if all of the following criteria apply:
1. Male or female > or = 18 years of age, at the time of signing informed consent.
2. Diagnosis of IRIDA based on the following:
- Documented homozygous or compound heterozygous TMPRSS6 gene variant(s) of Class 3 or greater (variant of uncertain significance (VUS), likely pathogenic, pathogenic) per the Association for Clinical Genomic Science (ACGS).
3. Serum TSAT at screening <15%.
4. Participants receiving oral iron supplementation must be on a stable dose for > or = 4 weeks prior to Day 1, with a maximum of 60 mg/day of oral elemental iron. Intravenous (IV) iron is not permitted during the study.
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 10
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:
Medical History
1. Body mass index (BMI) >35 kg/m squared
2. Any active infection requiring parenteral antibiotic therapy within 28 days prior to Day 1 or oral antibiotics within 14 days of Day 1. Any infection with >5 days of fever (>38.5 degrees C) within 28 days prior to Day 1.
3. Presence of uncontrolled heart disease or New York Heart Association (NYHA) Class 3 or 4 heart failure.
4. History of uncontrolled hyper- or hypothyroidism.
5. History of drug or alcohol abuse, as defined by the investigator, within the past 2 years.
6. History of stroke, arterial embolism, or unresolved deep venous thrombosis (DVT) within 6 months prior to Day 1.
7. Major surgery within 28 days prior to Day 1. Participants who had surgery more than 28 days prior to Day 1 must have recovered satisfactorily to participate in the study, in the opinion of the Investigator.
8. Known positive for human immunodeficiency virus (HIV), active infectious hepatitis B (HBV), or active infectious hepatitis C (HCV).
9. Any malignancy that has not been in remission and/or has required systemic therapy including radiation, chemotherapy, hormonal therapy, or surgery within the last year prior to Day 1.
10. History of solid organ or hematological transplantation.
11. History of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the investigational drug.
12. History of corneal dysplasia.
13. History of any known inflammatory or immunodeficiency disorder (e.g., lupus, common variable immunodeficiency [CVID]).

Treatment History
14. Treatment with IV iron within 28 days prior to study entry
15. Receiving treatment with proton pump inhibitors (PPIs). Participants receiving PPIs who discontinue use at least 7 days prior to Day 1 are permitted to enroll.
16. Receiving and plan to continue any disallowed medications listed in the protocol.
17. Treatment with another investigational drug or device, or approved therapy for investigational use = 28 days prior to Day 1, or, if the half-life of the previous product is known, within 5 times the half-life prior to Day 1, whichever is longer.

Laboratory Exclusions
18. Hemoglobin level =13.8 g/dL (8.56 mmol/L) (males) or > or = 12.1 g/dL (7.51 mmol/L) (females)
19. Serum ferritin <50 or >700 µg/L
20. Absolute lymphocyte count <1.00 x 10^9/L
21. Absolute neutrophil count <1.50 x 10^9/L
22. Estimated glomerular filtration rate (eGFR) by Chronic Kidney Disease-Epidemiology Collaboration (CKD-EPI) creatinine equation < 45 mL/min/1.73 m2
23. Alanine transaminase (ALT) or aspartate transaminase (AST) >2x upper limit of normal (ULN)
24. C-reactive protein (CRP) > or =10 mg/L

Study & Design

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