Phase IIa Study to Characterize the Effects of the Spiegelmer® NOX H94 on Anemia of Chronic Disease in Patients With Cancer
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 8
- 主要终点
- Response rate of anemia
研究概览
简要总结
This study is conducted to determine the safety, tolerability, and efficacy of NOX-H94 in patients with anemia of chronic disease (ACD). Furthermore, this study is intended to provide data needed to correlate plasma concentrations of NOX-H94 with its efficacy and to choose the appropriate dose and dose schedule of subsequent efficacy studies.
Some chronic diseases, e.g. tumors, inflammation, renal disease, are associated with high hepcidin concentrations in the blood. These hepcidin concentrations cause a reduction in iron concentrations in the blood and subsequently impair formation of red blood cells. Treatment with NOX-H94 is expected to inhibit this patho-mechanism by binding and inactivating hepcidin.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent
- •Female or male aged >18 years
- •Clinically significant anemia of chronic disease (ACD) attributed to histologically or cytologically proven malignancy, either hematological or solid tumor, of any grade or stage: Hemoglobin (Hb) 7.0 g/dL to 10 g/dL, Transferrin saturation (TSAT) <50%, Serum iron <50 µg/dL (SI: <9.0 µmol/L), AND Ferritin >30 ng/mL (SI: >30 µg/L)
- •Previous treatment with systemic anti-cancer therapy / regimen
- •Eastern Cooperative Oncology Group (ECOG) performance status of ≤2
- •Estimated life expectancy ≥12 weeks
- •Men must agree to follow effective contraception methods during treatment and for 3 months after completion of treatment. Women of childbearing potential must agree to use two forms of effective contraception during treatment and for 3 months after completion of treatment.
排除标准
- •Inability to personally provide written informed consent or to understand and collaborate throughout the study
- •History of pure red cell aplasia, thalassemia major or sickle cell disease History of anemia unrelated to cancer <10 g/dL within 6 months prior to screening
- •Uncorrected iron deficiency
- •Regular need for blood transfusions at intervals <6 weeks
- •Acute or myeloid leukemia
- •Known or suspected chronic bleeding
- •Tumor with gastro-intestinal involvement without negative test for fecal occult blood
- •Suspected or known history of hemochromatosis
- •Known infection with human immunodeficiency virus, hepatitis B, or hepatitis C
- •Impaired liver function with bilirubin ≥2.0 mg/dL (26 μmol/L), AST or ALT ≥2 times upper limit
- •History or risk of significant hepatic disease, e.g. chronic alcohol abuse, hepatic steatosis, hepatic cirrhosis, or organ transplantation
- •Severe renal impairment: estimated glomerular filtration rate (eGFR) <30 mL/min (Cockcroft-Gault)
- •Known central nervous system malignancy or metastasis
- •Significant cardiac disease (e.g. uncontrolled hypertension: systolic blood pressure [BP] >150 mmHg or diastolic BP >100 mmHg; myocardial infarction or unstable angina pectoris) within 6 months prior to screening
- •Positive pregnancy test (serum ß-hCG at screening, urine pregnancy test prior to first treatment) or lactation
- •Previous participation in this study or treatment with an investigational agent <21 days prior to treatment start
- •Hemolysis or bleeding >500 mL (measured or estimated) within 6 weeks prior to treatment start
- •Treatment with erythropoiesis-stimulating agents (ESAs) or red blood cell (RBC) transfusions <21 days prior to treatment start
- •Cytotoxic anti-tumor treatment <21 days prior to treatment start or planned during the anticipated study period (within 3 months from treatment start or randomization). Maintenance therapy is permitted throughout the study (e.g. lenalidomide, interferon)
研究组 & 干预措施
Open-label pilot group
Twice weekly administration of NOX-H94
干预措施: NOX-H94 (Drug)
Open-label pilot group
Twice weekly administration of NOX-H94
干预措施: Placebo solution (Drug)
结局指标
主要结局
Response rate of anemia
时间窗: treatment start to 1 week after treatment end
• Hb increase ≥1 g/dL OR reticulocyte index normalization (≥1%) at any time point until 1 week after the end of treatment AND absence of all of the following treatment failure criteria until 1 week after the end of treatment: * Erythrocyte transfusion, ESA or IV iron, * Hb drop by ≥1 g/dL * Treatment interruption due to adverse events (AEs)
次要结局
- Response(Treatment start to 8 weeks after end of treatment)
- Failure(Treatment start to 1 week after end of treatment)
- Safety and tolerability(Treatment start to 8 weeks after end of treatment)
- Pharmacokinetics(Treatment start to 8 weeks after end of treatment)
- Reticulocytes(Treatment start until 8 weeks after end of treatment)
- Red blood cells(Treatment start until 8 weeks after end of treatment)
- Transferrin(Treatment start to 8 weeks after end of treatment)
- Serum iron(Treatment start to 8 weeks after end of treatment)
- Ferritin(Treatment start to 8 weeks after end of treatment)
- Transferrin saturation(Treatment start to 8 weeks after end of treatment)
- Hemoglobin(Treatment start to 8 weeks after end of treatment)