A Phase 2, Open-label, Dose Escalation and Dose Expansion Study of KER-047 for the Treatment of IRIDA

Phase 2

Recruiting

• Conditions: iron-refractory iron deficiency anemia (IRIDA); IRIDA; iron-refractory iron deficiency anemia; 10038158; 10005330

• Registration Number: NL-OMON54150
• Lead Sponsor: Keros Therapeutics, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 2023-10-25
• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: Not specified
• Target Recruitment: 1

Inclusion Criteria

Participants are eligible for the study if all of the following criteria apply:
1. Male or female >= 18 years of age, at the time of signing informed consent.
2. Confirmed 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, likely pathogenic,
pathogenic) per the Association for Clinical Genomic Science.
3. Serum TSAT at screening less than 15%.
4. Participants receiving oral iron supplementation must be on a stable dose
for >=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.
5. Ability to understand the purpose and risks of the study and provide signed
and dated informed consent and authorization to use protected health
information in accordance with national and local study participant privacy
regulations.
6. Females of childbearing potential and sexually active males must agree to
use effective methods of contraception as outlined in the protocol.
7. In the opinion of the Investigator, the participant is able and willing to
comply with the requirements of the protocol (e.g., all study procedures,
return for follow-up visits).

Exclusion Criteria

Participants are excluded from the study if any of the following criteria apply:
Medical History
1. Body mass index >35 kg/m2
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* C) within 28 days prior to Day 1.
3. Presence of uncontrolled heart disease or New York Heart Association Class 3
or 4 heart failure.
4. History or presence at screening of an uncontrolled chronic disease.
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
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, active infectious hepatitis
B, or active infectious hepatitis C.
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. Have had a fracture within 4 weeks of D1.
12. History of severe allergic or anaphylactic reactions or hypersensitivity to
recombinant proteins or excipients in the IMP.

Treatment History
13. Treatment with IV iron within 28 days prior to study entry.
14. 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.
15. Receiving and plan to continue any disallowed medications.
16. 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
17. Hemoglobin level >=13.8 g/dL (8.56 mmol/L) (males) or >=12.1 g/dL (7.51
mmol/L) (females)
18. Serum ferritin < 50 or > 500 µg/L
19. Absolute lymphocyte count < 1.00 x 109/L
20. Absolute neutrophil count < 1.50 x 109/L
21. Estimated glomerular filtration rate by Chronic Kidney Disease-Epidemiology
Collaboration creatinine equation < 45 mL/min/1.73 m2
22. Alanine transaminase or aspartate transaminase > 2 x upper limit of normal
(ULN)

Miscellaneous
23. Pregnant or lactating females.
24. Any other condition not specifically noted above that, in the judgment of
the Investigator or Sponsor, would preclude the participant from participating
in the study.
25. Participants who are investigational site staff members directly involved
in the conduct of the trial and their immediate family members, site staff
members otherwise supervised by the Investigator, or participants who are Keros
or CRO employees directly involved in the conduct of the study. Immediate
family is defined as a spouse, parent, child, or sibling, whether biological or
legally adopted.

Study & Design

• Study Type: Interventional
• Study Design: Not specified