Recruiting

Phase 3

EtavopivatA Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

Sponsor:

Novo Nordisk A/S

Code:

NCT06612268

Conditions

Sickle Cell Disease

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

Interventions

Etavopivat

Placebo

Study Details

Brief summary:

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.

Conditions

Sickle Cell Disease

Study ID

NCT06612268

Start date

Feb 17, 2025

Status verified date

Aug, 2026

Completion date

Aug 12, 2029

Anticipated

Primary completion date

Aug 27, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 12+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

408 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Etavopivat

Participants will be randomised to receive oral dose of Etavopivat.

placebo comparator: Placebo

Participants will be randomised to receive oral dose of placebo.

Interventions

Etavopivat

Etavopivat will be administered orally.

Placebo

Placebo matching Etavopivat will be administered orally.

Primary outcome measure

Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact [ Time Frame: Baseline (week 0) to week 52 ]

Central Contacts and Locations

Central contacts

Novo Nordisk

(+1) 866-867-7178 clinicaltrials@novonordisk.com

Locations

Uni of Alabama at Birmingham

Recruiting

Birmingham, Alabama, United States, 35233

Univer South Alabama Ped/Onc

Recruiting

Mobile, Alabama, United States, 36604

Phoenix Children's Hsptl

Recruiting

Phoenix, Arizona, United States, 85016

Arkansas Children's Hospital

Recruiting

Little Rock, Arkansas, United States, 72202

Children's Hospital Los Angeles - Endocrinology

Recruiting

Los Angeles, California, United States, 90027

Valley Children's Hospital

Recruiting

Madera, California, United States, 93636

Stanford University_Palo Alto

Recruiting

Palo Alto, California, United States, 94304

Harbor-UCLA Medical Center

Recruiting

Torrance, California, United States, 90502

Nemours/AI duPont Hosp-Chld

Recruiting

Wilmington, Delaware, United States, 19803

Childrens National Medical Ctr

Recruiting

Washington D.C., District of Columbia, United States, 20010

MedStar Hlth Res Institute

Recruiting

Washington D.C., District of Columbia, United States, 20010

Memorial Healthcare

Recruiting

Hollywood, Florida, United States, 33021

Children's Healthcare Atlanta

Recruiting

Atlanta, Georgia, United States, 30329

Childrens Hospital of Chicago

Recruiting

Chicago, Illinois, United States, 60611

Univer Of Illinois at Chicago

Recruiting

Chicago, Illinois, United States, 60612

Children's Hosp-New Orleans

Recruiting

New Orleans, Louisiana, United States, 70118

Boston Children's Hospital

Recruiting

Boston, Massachusetts, United States, 02115

Boston Medical Center

Recruiting

Boston, Massachusetts, United States, 02118-2393

University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

Washington University-St.Louis

Recruiting

St Louis, Missouri, United States, 63110

Children's Nebraska

Recruiting

Omaha, Nebraska, United States, 68114

Newark Beth Israel MC

Recruiting

Newark, New Jersey, United States, 07112-2027

NYC Health+Hospitals

Recruiting

Brooklyn, New York, United States, 11203

Interfaith Medical Center

Recruiting

Brooklyn, New York, United States, 11238

Cohen Children's Medical Ctr

Recruiting

Queens, New York, United States, 11040

Jacobi Medical Center

Recruiting

The Bronx, New York, United States, 10461

Montefiore Medical Ctr

Recruiting

The Bronx, New York, United States, 10467

Duke Comprehen Sickle Cell

Recruiting

Durham, North Carolina, United States, 27710

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

East Carolina Univ-Greenville

Recruiting

Greenville, North Carolina, United States, 27834

Atrium Health-Wake Forest Bapt

Recruiting

Winston-Salem, North Carolina, United States, 27157

Univ Hosp Cleveland Med Ctr

Recruiting

Cleveland, Ohio, United States, 44106

Ohio State Univ Wexner Med Ctr

Recruiting

Columbus, Ohio, United States, 43203

Univ of OK Health Sciences Ctr

Recruiting

Oklahoma City, Oklahoma, United States, 73104

Children's Hosptl Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

St Christopher Hosp for Child

Recruiting

Philadelphia, Pennsylvania, United States, 19134

University of Tennessee Health Science Center

Recruiting

Memphis, Tennessee, United States, 38104

St. Jude Children's Res Hosp

Recruiting

Memphis, Tennessee, United States, 38105

UT Health University of Texas

Recruiting

Houston, Texas, United States, 77030

Inova Health System

Recruiting

Fairfax, Virginia, United States, 22031

Children's Hsptl Of The Kings

Recruiting

Norfolk, Virginia, United States, 23507

Virginia Comm Univ Medical Ctr

Recruiting

Richmond, Virginia, United States, 23298

Mary Bridge Children's Health

Recruiting

Tacoma, Washington, United States, 98405

Foothills Med Ctr-Univ Calgary

Recruiting

Calgary, Alberta, Canada, T2N 2T9

Stollery Children's Hospital

Recruiting

Edmonton, Alberta, Canada, T6G 2B7

University of Alberta_Edmonton

Recruiting

Edmonton, Alberta, Canada, T6G 2B7

BC Children's Hospital

Recruiting

Vancouver, British Columbia, Canada, V6H 3V4

LHSC - Victoria Hospital

Recruiting

London, Ontario, Canada, N6A5W9

University of Toronto

Recruiting

Toronto, Ontario, Canada, M5G1X8

CHUM-Hosp de Univ Montreal

Recruiting

Montreal, Quebec, Canada, H2X 0C1

Montreal Children's Hospital

Recruiting

Montreal, Quebec, Canada, H4A 3J1

More Information

Sponsor

Novo Nordisk A/S

Last update posted

Aug 13, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Novo Nordisk A/S on 2026-08-13.