2023-509175-16-00招募中3 期
A global phase 3, randomised, double-blind and placebo-controlled study evaluating the efficacy and safety of etavopivat in adolescents and adults with sickle cell disease
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 75
- 试验地点
- 33
- 主要终点
- Number of adjudicated VOC events with a medical contact.
研究概览
简要总结
To demonstrate superiority of treatment with etavopivat versus placebo in adolescents and adults with sickle cell disease
入排标准
- 年龄范围
- 0 years 至 65+ years（65+ Years, 18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Male or female
- •Age 12 years or above at the time of signing the informed consent
- •Confirmed diagnosis of sickle cell disease (SCD): Documentation of SCD genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing or screening test results from central laboratory.
- •Have 2−15 episodes of documented vaso-occlusive crises (VOCs) within the 12 months prior to screening. Documentation must exist in the participant’s medical record prior to randomisation. Events based solely on participant recall without supporting documentation should not be counted towards eligibility.
- •Hb ≥5.0 and ≤10.0 g/dL (≥50 and ≤100 g/L) at screening.
排除标准
- •Use of voxelotor or similar agent within 28 days prior to starting study treatment or anticipated need for this agent during the study
- •Severe renal dysfunction (estimated glomerular filtration rate [eGFR] at screening, calculated by the central laboratory <30 mL/min/1.73 m2) or on chronic dialysis.
- •Travelled distance on standardized 6-minute walking test below 100m at screening.
- •Use of a selectin antagonist (e.g., crizanlizumab, monoclonal antibody or small molecule) within 28 days or 5 half-lives (whichever is longer) prior to starting study treatment or anticipated need for such agents during the study.
- •Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or ≥6 transfusion events in the previous 12 months (i.e., an average of 1 transfusion event every 60 days).
- •Participants who have received an RBC transfusion for any reason within 60 days of the screening period or 60 days of the randomisation day are only eligible if HbA (adult Hb) <10% by Hb electrophoresis is documented prior to starting study treatment.
- •Receiving or use of concomitant medications that are strong inducers of CYP3A4 within 2 weeks of starting study treatment or anticipated need for such agents during the study.
- •Use of erythropoietin or other haematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study.
- •Receipt of prior cellular-based therapy (e.g., haematopoietic cell transplant, gene modification therapy).
- •Hepatic dysfunction characterized by: Alanine aminotransferase (ALT) >4.0 × upper limit of normal (ULN) or Direct bilirubin >3.0 × ULN.
- •Participants who are not taking or are unable to take antimalarial prophylaxis at the time of consent and during the study if they live in areas of endemic malaria where prophylaxis is recommended.
结局指标
主要结局
Number of adjudicated VOC events with a medical contact.
Number of adjudicated VOC events with a medical contact.
次要结局
- Change in Hb >1 g/dL (yes/no)
- Time to onset of first adjudicated VOC.
- Change in standardised T-score on the PROMIS Fatigue 7a Scale.
- Change in haemoglobin (Hb)
- Change in lactate dehydrogenase (LDH)
- Change in absolute reticulocyte count
- Change in indirect bilirubin
- Change in distance travelled during the 6-minute walking test (6MWT)
- Participants achieving the threshold for clinically meaningful change (yes/no) in PROMIS fatigue scale 7a
- Participants achieving the threshold for clinically meaningful change (yes/no) in 6MWT
研究者
EU Submission Hub
Scientific
Novo Nordisk A/S
研究点 (33)
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