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RECRUITING

NCT06612268

PHASE3

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

Sponsor: Novo Nordisk A/S

View on ClinicalTrials.gov

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.

Official title: 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

Key Details

Gender

All

Age Range

12 Years - Any

Study Type

INTERVENTIONAL

Enrollment

510

Start Date

2025-02-17

Completion Date

2029-08-12

Last Updated

2026-09-17

Healthy Volunteers

Conditions

Sickle Cell Disease

Interventions

DRUG

Etavopivat

Etavopivat will be administered orally.

DRUG

Placebo

Placebo matching Etavopivat will be administered orally.

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.

Locations (175)

Uni of Alabama at Birmingham

Birmingham, Alabama, United States

Univer South Alabama Ped/Onc

Mobile, Alabama, United States

Phoenix Children's Hsptl

Phoenix, Arizona, United States

Arkansas Children's Hospital

Little Rock, Arkansas, United States

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, United States

UCLA Health

Los Angeles, California, United States

Valley Children's Hospital

Madera, California, United States

University Of California Irvine

Orange, California, United States

Stanford University_Palo Alto

Palo Alto, California, United States

Harbor-UCLA Medical Center

Torrance, California, United States

Clinical and Transl Res Center

Aurora, Colorado, United States

Nemours/AI duPont Hosp-Chld

Wilmington, Delaware, United States

Childrens National Medical Ctr

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

MedStar Hlth Res Institute

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

Memorial Healthcare

Hollywood, Florida, United States

Children's Healthcare Atlanta

Atlanta, Georgia, United States

Childrens Hospital of Chicago

Chicago, Illinois, United States

Univer Of Illinois at Chicago

Chicago, Illinois, United States

Children's Hosp-New Orleans

New Orleans, Louisiana, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Boston Medical Center

Boston, Massachusetts, United States

Henry Ford Hospital_Detroit

Detroit, Michigan, United States

University of Minnesota

Minneapolis, Minnesota, United States

Washington University-St.Louis

St Louis, Missouri, United States

Children's Nebraska

Omaha, Nebraska, United States

Cure 4 the Kids Foundation

Las Vegas, Nevada, United States

Newark Beth Israel MC

Newark, New Jersey, United States

NYC Health+Hospitals

Brooklyn, New York, United States

Interfaith Medical Center

Brooklyn, New York, United States

Northwell Health

Mount Kisco, New York, United States

Cohen Children's Medical Ctr

Queens, New York, United States

Jacobi Medical Center

The Bronx, New York, United States

Montefiore Medical Ctr

The Bronx, New York, United States

Duke Comprehen Sickle Cell

Durham, North Carolina, United States

Duke University Medical Center

Durham, North Carolina, United States

East Carolina Univ-Greenville

Greenville, North Carolina, United States

Atrium Health-Wake Forest Bapt

Winston-Salem, North Carolina, United States

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio, United States

Ohio State Univ Wexner Med Ctr

Columbus, Ohio, United States

Univ of OK Health Sciences Ctr

Oklahoma City, Oklahoma, United States

Children's Hosptl Philadelphia

Philadelphia, Pennsylvania, United States

St Christopher Hosp for Child

Philadelphia, Pennsylvania, United States

University of Tennessee Health Science Center

Memphis, Tennessee, United States

St. Jude Children's Res Hosp

Memphis, Tennessee, United States

UT Health University of Texas

Houston, Texas, United States

Inova Health System

Fairfax, Virginia, United States

Children's Hsptl Of The Kings

Norfolk, Virginia, United States

Virginia Comm Univ Medical Ctr

Richmond, Virginia, United States

Mary Bridge Children's Health

Tacoma, Washington, United States

Mary Bridge Children's Health

Tacoma, Washington, United States

Royal Prince Alfred Hospital - Department of Haematology

Camperdown, New South Wales, Australia

Prince of Wales Hospital - Department of Haematology

Randwick, New South Wales, Australia

Monash Medical Centre - Haematology Department

Clayton, Victoria, Australia

Fiona Stanley Hospital - Haemophilia and Haemostasis Centre

Murdoch, Western Australia, Australia

CHU Saint-Pierre - UMC Sint-Pieter

Brussels, Belgium

CHU - UVC Brugmann

Brussels, Belgium

HUB - Hôpital Erasme

Brussels, Belgium

UZ Antwerpen - UZA - Haematology

Edegem, Belgium

UZ Antwerpen - UZA - Kinderhemato-Oncologie

Edegem, Belgium

UZ Leuven - Kindergeneeskunde

Leuven, Belgium

MultiHemo - Grupo Oncoclínicas

Recife, Pernambuco, Brazil

MultiHemo - Grupo Oncoclínicas

Recife, Pernambuco, Brazil

Hemocentro UNICAMP

Distrito de Barão Geraldo - Campinas, São Paulo, Brazil

HC da FMUSP Hospital das Clínicas São Paulo

São Paulo, São Paulo, Brazil

HC da FMUSP Hospital das Clínicas São Paulo

São Paulo, São Paulo, Brazil

Fundação Hemocentro de Ribeirão Preto

Ribeirão Preto, São, Brazil

Hemorio-Fundarj

Rio de Janeiro, Brazil

Hospital Samaritano Higienópolis - Instituto de Conhecimento Ensino e Pesquisa (ICEP)

São Paulo, Brazil

Foothills Med Ctr-Univ Calgary

Calgary, Alberta, Canada

Stollery Children's Hospital

Edmonton, Alberta, Canada

University of Alberta_Edmonton

Edmonton, Alberta, Canada

BC Children's Hospital

Vancouver, British Columbia, Canada

St Pauls Hospital

Vancouver, British Columbia, Canada

LHSC - Victoria Hospital

London, Ontario, Canada

Scarborough Health Network Research Institute (SHNRI)

Scarborough Village, Ontario, Canada

UHN-Toronto General Hospital

Toronto, Ontario, Canada

University of Toronto

Toronto, Ontario, Canada

CHUM-Hosp de Univ Montreal

Montreal, Quebec, Canada

Montreal Children's Hospital

Montreal, Quebec, Canada

Hospital Pablo Tobon Uribe

Medellín, Antioquia, Colombia

Clinica de la Costa

Barranquilla, Atlántico, Colombia

Sociedad de Oncología y Hematología del Cesar SAS (SOHEC)

Valledupar, Cesar Department, Colombia

Clínica IMAT Oncomedica Auna S.A.S.

Montería, Departamento de Córdoba, Colombia

Fundacion Valle del Lili

Cali, Valle del Cauca Department, Colombia

Sociedad de Oncología y Hematología del Cesar SAS (SOHEC)

Antioquia, Colombia

Centro de investigaciones clínicas, fudación valle del lili

Cali, Colombia

Clínica IMAT Oncomedica Auna S.A.S.

Montería, Colombia

Ap-Hp-Hopital Avicenne

Bobigny, France

Centre Hospitalier Universitaire de Bordeaux-Hopital Pellegrin

Bordeaux, France

Ap-Hp-Hopital Henri Mondor

Créteil, France

Centre Hospitalier Universitaire Grenoble Alpes-Site Nord Michallon

Grenoble, France

Hospices Civils de Lyon-Hopital Edouard Herriot

Lyon, France

AP-HP-HOPITAL NECKER_Service d'hématologie

Paris, France

Ap-Hp-Hopital Robert Debre

Paris, France

Centre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle

Rouen, France

Kintampo Health Research Centre (KHRC)

Kintampo, Bono East, Ghana

Navrongo Health Research Centre

Navrongo, Upper East Region, Ghana

Agogo Presbyterian Hospital, Ghana

Agogo, Ghana

"Laiko" General Hospital of Athens

Athens, Greece

Childrens' Hospital of Athens "Agia Sofia"

Athens, Greece

And 75 more locations on ClinicalTrials.gov

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (175)