Assessing Combination Hydroxyurea and Exogenous Erythropoietin in Sickle Cell Disease
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- 入组人数
- 17
- 试验地点
- 2
- 主要终点
- Percentage of Participants With Hemoglobin (Hb) Response
研究概览
简要总结
The proposed study is a Phase 1/2 multi-center study evaluating the safety and efficacy of erythropoietin (EPO) in combination with hydroxyurea in the treatment of chronic anemia in patients with sickle cell disease (SCD).
详细描述
Sickle cell disease (SCD) is a devastating inherited hemoglobin disorder characterized by recurrent episodes of pain and chronic hemolytic anemia. Chronic anemia contributes to multi-organ damage and decreased life expectancy in SCD. However, there are limited treatment options for anemia in SCD. Erythropoietin (EPO) is the standard of care for treatment of anemia related to chronic kidney disease (CKD) and is also used ad hoc in patients with SCD. However, there is limited data on the safety and efficacy of EPO in patients with SCD, especially in combination with hydroxyurea. Therefore, this study aims to treat patients on stable hydroxyurea therapy with subcutaneous EPO, with the goal of assessing the safety of EPO therapy and its effect on chronic anemia in SCD.
(Note: Outcome measure changes were in place prior to study initiation.)
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
- •Aged ≥ 18 years
- •Confirmed diagnosis of SCD (HbSS or HbS/β0-thalassemia genotypes)
- •Screening Hb ≤ 9.0 g/dL
- •Screening transferrin saturation ≥ 20% and ferritin ≥ 50 ng/mL
- •Must be on stable-dose hydroxyurea treatment (i.e., no changes in dose within 60 days prior to start of study drug) and plan to continue taking hydroxyurea at the same dose and schedule during the study
- •If receiving L-glutamine or crizanlizumab, must have been receiving the drug at a stable dose for at least 60 days prior to screening and plan to continue taking the drug at the same dose and schedule during the study
排除标准
- •Participating in a chronic transfusion program (pre-planned series of transfusions for prophylactic purposes) and/or planning on undergoing an exchange transfusion during the duration of the study; episodic transfusion in response to worsened anemia or VOC is permitted, but participant should not have received a blood transfusion within 60 days of start of study drug
- •Received voxelotor or EPO within 30 days of start of study drug
- •Untreated iron deficiency, or had initiation or change in dose of supplemental iron within 30 days of start of study drug
- •Ongoing acute illness, infection, or VOC within 2 weeks of start of study drug
- •Arterial or venous thrombosis within 180 days of start of study drug
- •Grade 3 hypertension (defined as systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg; medical intervention indicated; more than one drug or more intensive therapy than previously used indicated) on two consecutive measurements
- •Unstable angina, uncontrolled seizure disorder, or active malignancy
- •End-stage renal disease requiring hemodialysis
- •Current pregnancy or breastfeeding
- •Received active treatment on another investigational trial within 30 days (or 5 half-lives of that agent, whichever is greater) prior to start of study drug or plans to participate in another investigational drug trial
研究组 & 干预措施
Erythropoietin
Subjects on a stable dose of hydroxyurea will be treated with increasing doses of subcutaneous erythropoietin (EPO) as tolerated for an initial 12 weeks, during which the main safety and efficacy endpoints (including the primary endpoint of hemoglobin response) will be assessed. Subjects may continue on treatment for an additional 12 weeks as clinically indicated, with assessment of additional endpoints at the end of the 24-week study period.
干预措施: Hydroxyurea (Drug)
Erythropoietin
Subjects on a stable dose of hydroxyurea will be treated with increasing doses of subcutaneous erythropoietin (EPO) as tolerated for an initial 12 weeks, during which the main safety and efficacy endpoints (including the primary endpoint of hemoglobin response) will be assessed. Subjects may continue on treatment for an additional 12 weeks as clinically indicated, with assessment of additional endpoints at the end of the 24-week study period.
干预措施: Epoetin Alfa-BioSimilar (Drug)
结局指标
主要结局
Percentage of Participants With Hemoglobin (Hb) Response
时间窗: Baseline to 12 weeks
Hb response is defined as a Hb increase of ≥ 1.0 g/dL at 12 weeks compared to baseline
次要结局
- Change in Number of Blood Transfusions Per Year(Baseline to 12 weeks)
研究者
Julia Xu
Assistant Professor
University of Pittsburgh