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

Novo Nordisk A/S316 个研究点分布在 3 个国家目标入组 408 人2025年2月17日

适应症Sickle Cell Disease

干预措施Etavopivat Placebo

概览

阶段: 3 期
干预措施: Etavopivat
疾病 / 适应症: Sickle Cell Disease
发起方: Novo Nordisk A/S
入组人数: 408
试验地点: 316
主要终点: Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact
状态: 招募中
最后更新: 8天前

概览研究者入排标准研究组 & 干预措施结局指标研究点相似试验

概览

简要总结

This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.

注册库

clinicaltrials.gov

开始日期

2025年2月17日

结束日期

2029年3月12日

最后更新

8天前

研究类型

Interventional

研究设计

Parallel

性别

All

研究者

发起方

Novo Nordisk A/S

责任方

Sponsor

入排标准

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: Documentation of sickle cell disease (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. Molecular genotyping is not required. SCD genotype may be determined from the results of haemoglobin (Hb) electrophoresis, high-performance liquid chromatography (HPLC) or similar testing. Note that Hb electrophoresis is performed by the central laboratory at screening.
•Have 1-15 episodes of documented vaso occlusive crises (VOC) 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 greater than or equal to (≥) 5.0 and less than or equal to (≤) 10.0 g/dL (greater than or equal to (≥) 50 and less than or equal to (≤) 100 g/L) at screening.

排除标准

•More than 15 VOCs within the past 12 months prior to screening documented in the participant's medical record. Events based solely on participant recall without supporting documentation should not be counted towards eligibility.
•Use of voxelotor or similar agent within 28 days prior to starting study treatment or anticipated need for this agent during the study.
•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 greater than or equal to 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 haemoglobin) less than 10% by Hb electrophoresis is documented prior to starting study treatment.
•Receiving or use of concomitant medications that are strong inducers of CYP3A4 (cytochrome p450 3a4) 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) greater than 4.0 × upper limit of normal (ULN) or

研究组 & 干预措施

Etavopivat

Participants will be randomised to receive oral dose of Etavopivat.

干预措施: Etavopivat

Placebo

Participants will be randomised to receive oral dose of placebo.

干预措施: Placebo

结局指标

主要结局

Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact

时间窗: Baseline (week 0) to week 52

Measured as Count of events.

次要结局

Time to onset of first adjudicated Vaso-occlusive crisis (VOC)(Baseline (week 0) to week 52)
Change in distance travelled during the 6-minute walking test (6MWT)(Baseline (week 0) to week 52)
Change in standardised T-score on the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 7a Scale(Baseline (week 0) to week 52)
Change in haemoglobin (Hb)(Baseline (week 0) to week 52)
Change in lactate dehydrogenase (LDH)(Baseline (week 0) to week 52)
Change in absolute reticulocyte count(Baseline (week 0) to week 52)
Change in indirect bilirubin(Baseline (week 0) to week 52)
Change in Haemoglobin (Hb) greater than 1 grams per decilitre (g/dL)(Baseline (week 0) to week 24)
Time to onset of first adjudicated Vaso-occlusive crisis (VOC)(Baseline (week 0) to week 52)
Change in standardised T-score on the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 7a Scale(Baseline (week 0) to week 52)
Change in haemoglobin (Hb)(Baseline (week 0) to week 52)
Change in lactate dehydrogenase (LDH)(Baseline (week 0) to week 52)
Change in absolute reticulocyte count(Baseline (week 0) to week 52)
Change in indirect bilirubin(Baseline (week 0) to week 52)
Change in distance travelled during the 6-minute walking test (6MWT)(Baseline (week 0) to week 52)
Participants achieving the threshold for clinically meaningful change (yes/no) in PROMIS fatigue scale 7a(Baseline (week 0) to week 52)
Participants achieving the threshold for clinically meaningful change (yes/no) in 6MWT(Baseline (week 0) to week 52)