RecruitingPhase 3

A Study to Evaluate How Well Etavopivat Works in People 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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
12 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 2 years

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "The study will last for about 2 years."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests whether etavopivat can reduce sickle cell pain crises in people age 12 and older with sickle cell disease. It also checks whether the medicine can reduce organ damage and tiredness and help people exercise more. The study lasts about 2 years, and chance decides whether participants get etavopivat or a placebo (a treatment with no active medicine).

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • Age 12 or older when signing the study consent form.
  • Male or female.
  • Sickle cell disease confirmed by blood tests.
  • Have had 2–15 sickle cell pain crises in the past 12 months, recorded in medical records.
  • Have a hemoglobin level of 5–10 g/dL at screening. Hemoglobin is the protein in red blood cells that carries oxygen.

Who can't join

  • Used voxelotor or a similar drug within 28 days before study treatment, or expect to need it during the study. Also excluded: recent use of a selectin blocker, such as crizanlizumab, or plans to use one. Its waiting period is at least 28 days and may be longer based on how long the drug stays in the body.
  • Get regular red blood cell transfusions, or have had 6 or more transfusions in the past 12 months.
  • Had a red blood cell transfusion within 60 days of screening or treatment assignment, unless a blood test before treatment shows adult hemoglobin (HbA) below 10%.
  • Used medicines that moderately or strongly speed up the liver enzyme CYP3A4 within 2 weeks before treatment, or medicines that help make blood cells, such as erythropoietin, within 28 days before treatment. Expected use during the study also excludes you.
  • Previously had cell-based treatment, such as a blood-forming cell transplant or treatment that changes genes.
  • Have certain abnormal liver tests: ALT above 4 times the normal limit or direct bilirubin above 3 times the normal limit. Regular dialysis also excludes you. The listing calls its kidney cutoff 'severe' but gives eGFR above 30; ask the study team to clarify this kidney test rule.
  • Cannot or do not take medicine to prevent malaria when recommended in the area where you live, both at consent and during the study.
  • Walk less than 100 meters during the 6-minute walk test at screening.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility criteria

Inclusion Criteria: * 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 2-15 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 grams per litre \[g/L\]) at screening. Exclusion Criteria: * 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 within the previous 12 months prior to screening (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 or moderate 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 * Direct bilirubin greater than 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. * 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.

Study sites (50)

AL

AR

AZ

CA

CO

DC

DE

FL

GA

IL

LA

MA

MI

MN

MO

NC

NE

NJ

NV

NY

OH

OK

PA

TN

TX

VA

WA