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Evaluating Etavopivat for 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

Phase 3 Interventional Novo Nordisk A/S · NCT06612268

This study is testing if a new drug called etavopivat can help reduce painful crises and improve overall health in adults and teens with sickle cell disease.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment408 (estimated)
Ages12 Years and up
SexAll
SponsorNovo Nordisk A/S Industry-sponsored
Drugs / interventionscrizanlizumab
Locations172 sites (Birmingham, Alabama and 171 other locations)
Trial IDNCT06612268 on ClinicalTrials.gov

What this trial studies

This study aims to determine the effectiveness of etavopivat in reducing the frequency of vaso-occlusive crises (pain crises) in adults and adolescents with sickle cell disease. Participants will be randomly assigned to receive either etavopivat or a placebo over a period of approximately two years. The study will also assess the drug's ability to minimize organ damage, enhance exercise tolerance, and alleviate fatigue associated with the condition. The results will help establish the therapeutic potential of etavopivat for managing sickle cell disease.

Who should consider this trial

Good fit: Ideal candidates include males and females aged 12 years or older with a confirmed diagnosis of sickle cell disease and a history of 1-15 vaso-occlusive crises in the past year.

Not a fit: Patients who do not have sickle cell disease or those with fewer than one documented vaso-occlusive crisis in the past year may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly reduce pain crises and improve the quality of life for patients with sickle cell disease.

How similar studies have performed: Other studies have shown promise in treating sickle cell disease with similar approaches, but the specific efficacy of etavopivat is still being evaluated.

Eligibility criteria

Show full inclusion / exclusion 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 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.

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:

  * 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.

Where this trial is running

Birmingham, Alabama and 171 other locations

+122 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Sickle Cell Disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.