EUCTR2021-000348-22-ES进行中（未招募）1 期
A Phase 2, Open-label, Dose Escalation and Dose Expansion Study of KER-047 for the Treatment of IRIDA
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •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
排除标准
- •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
研究者
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