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
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
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.
Inclusion Criteria:
Exclusion Criteria:
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:
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.
Severe renal dysfunction (estimated glomerular filtration rate [eGFR] at screening, calculated by the central laboratory greater than 30 mL/min/1.73 m^ 2) or on chronic dialysis.
Travelled distance on standardized 6MWT below 100m at screening.
clinicaltrials@novonordisk.com(+1) 866-867-7178
Los Angeles, California 90027, United States
Camperdown, New South Wales 2050, Australia
Randwick, New South Wales 2031, Australia
Murdoch, Western Australia 6150, Australia
Distrito de Barão Geraldo - Campinas, São Paulo 13083-878, Brazil
São Paulo, 01229-001, Brazil
Scarborough Village, Ontario M1E 4B9, Canada
Valledupar, Cesar Department 200001, Colombia
Antioquia, 4006, Colombia
Grenoble, 38043, France
Rouen, 76031, France
Orbassano, Torino 10043, Italy
Verona, Veneto 37134, Italy
Modena, 41100, Italy
Siaya, Siaya County 40600, Kenya
Ituku-Ozalla, Enugu State 400001, Nigeria
Abuja, Federal Capital Territory (fct) 90410, Nigeria
Ibadan, Oyo State 0000, Nigeria
Al Ahsa, 36342, Saudi Arabia
Al Ahsa, 36428, Saudi Arabia
Al Qaţīf, 32643, Saudi Arabia
Adana, 01250, Turkey (Türkiye)
Mersin, 33110, Turkey (Türkiye)
Mersin, 33110, Turkey (Türkiye)
Kampala, Central Region 00000, Uganda
Kampala, Central Region (Wakiso District) 00000, Uganda