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NCT06612268PHASE3Recruiting

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

Novo Nordisk A/S

Start Date

2/17/2025

Completion Date

3/12/2029

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.

Eligibility Criteria

Age Range: 12 years to No maximum

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.

Interventions

DRUG

Etavopivat

DRUG

Placebo

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Conditions

Sickle Cell Disease

Locations

Uni of Alabama at Birmingham

Birmingham, Alabama 35233

United States

Univer South Alabama Ped/Onc

Mobile, Alabama 36604

United States

Phoenix Children's Hsptl

Phoenix, Arizona 85016

United States

Arkansas Children's Hospital

Little Rock, Arkansas 72202

United States

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California 90027

United States

UCLA Health

Los Angeles, California 90095

United States

Valley Children's Hospital

Madera, California 93636

United States

University Of California Irvine

Orange, California 92868

United States

Stanford University_Palo Alto

Palo Alto, California 94304

United States

Harbor-UCLA Medical Center

Torrance, California 90502

United States

Clinical and Transl Res Center

Aurora, Colorado 80045

United States

Nemours/AI duPont Hosp-Chld

Wilmington, Delaware 19803

United States

Childrens National Medical Ctr

Washington D.C., District of Columbia 20010

United States

MedStar Hlth Res Institute

Washington D.C., District of Columbia 20010

United States

Memorial Healthcare

Hollywood, Florida 33021

United States

Children's Healthcare Atlanta

Atlanta, Georgia 30329

United States

Childrens Hospital of Chicago

Chicago, Illinois 60611

United States

Univer Of Illinois at Chicago

Chicago, Illinois 60612

United States

Children's Hosp-New Orleans

New Orleans, Louisiana 70118

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

Boston Medical Center

Boston, Massachusetts 02118-2393

United States

Henry Ford Hospital_Detroit

Detroit, Michigan 48202

United States

University of Minnesota

Minneapolis, Minnesota 55455

United States

Washington University-St.Louis

St Louis, Missouri 63110

United States

Children's Nebraska

Omaha, Nebraska 68114

United States

Cure 4 the Kids Foundation

Las Vegas, Nevada 89135

United States

Newark Beth Israel MC

Newark, New Jersey 07112-2027

United States

NYC Health+Hospitals

Brooklyn, New York 11203

United States

Interfaith Medical Center

Brooklyn, New York 11238

United States

Northwell Health

Mount Kisco, New York 10549

United States

Cohen Children's Medical Ctr

Queens, New York 11040

United States

Jacobi Medical Center

The Bronx, New York 10461

United States

Montefiore Medical Ctr

The Bronx, New York 10467

United States

Duke Comprehen Sickle Cell

Durham, North Carolina 27710

United States

Duke University Medical Center

Durham, North Carolina 27710

United States

East Carolina Univ-Greenville

Greenville, North Carolina 27834

United States

Atrium Health-Wake Forest Bapt

Winston-Salem, North Carolina 27157

United States

Univ Hosp Cleveland Med Ctr

Cleveland, Ohio 44106

United States

Ohio State Univ Wexner Med Ctr

Columbus, Ohio 43203

United States

Univ of OK Health Sciences Ctr

Oklahoma City, Oklahoma 73104

United States

Children's Hosptl Philadelphia

Philadelphia, Pennsylvania 19104

United States

St Christopher Hosp for Child

Philadelphia, Pennsylvania 19134

United States

University of Tennessee Health Science Center

Memphis, Tennessee 38104

United States

St. Jude Children's Res Hosp

Memphis, Tennessee 38105

United States

UT Health University of Texas

Houston, Texas 77030

United States

Inova Health System

Fairfax, Virginia 22031

United States

Children's Hsptl Of The Kings

Norfolk, Virginia 23507

United States

Virginia Comm Univ Medical Ctr

Richmond, Virginia 23298

United States

Mary Bridge Children's Health

Tacoma, Washington 98405

United States

Mary Bridge Children's Health

Tacoma, Washington 98405

United States

Royal Prince Alfred Hospital - Department of Haematology

Camperdown, New South Wales 2050

Australia

Prince of Wales Hospital - Department of Haematology

Randwick, New South Wales 2031

Australia

Monash Medical Centre - Haematology Department

Clayton, Victoria 3168

Australia

Fiona Stanley Hospital - Haemophilia and Haemostasis Centre

Murdoch, Western Australia 6150

Australia

CHU Saint-Pierre - UMC Sint-Pieter

Brussels, 1000

Belgium

CHU - UVC Brugmann

Brussels, 1020

Belgium

HUB - Hôpital Erasme

Brussels, 1070

Belgium

UZ Antwerpen - UZA - Haematology

Edegem, 2650

Belgium

UZ Antwerpen - UZA - Kinderhemato-Oncologie

Edegem, 2650

Belgium

UZ Leuven - Kindergeneeskunde

Leuven, 3000

Belgium

MultiHemo - Grupo Oncoclínicas

Recife, Pernambuco 50070-460

Brazil

MultiHemo - Grupo Oncoclínicas

Recife, Pernambuco 52070-460

Brazil

Hemocentro UNICAMP

Distrito de Barão Geraldo - Campinas, São Paulo 13083-878

Brazil

HC da FMUSP Hospital das Clínicas São Paulo

São Paulo, São Paulo 05403-010

Brazil

HC da FMUSP Hospital das Clínicas São Paulo

São Paulo, São Paulo 05403-010

Brazil

Fundação Hemocentro de Ribeirão Preto

Ribeirão Preto, São 14051-140

Brazil

Hemorio-Fundarj

Rio de Janeiro, 20211-030

Brazil

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

São Paulo, 01229-001

Brazil

Foothills Med Ctr-Univ Calgary

Calgary, Alberta T2N 2T9

Canada

Stollery Children's Hospital

Edmonton, Alberta T6G 2B7

Canada

University of Alberta_Edmonton

Edmonton, Alberta T6G 2B7

Canada

BC Children's Hospital

Vancouver, British Columbia V6H 3V4

Canada

St Pauls Hospital

Vancouver, British Columbia V6Z 2A5

Canada

LHSC - Victoria Hospital

London, Ontario N6A5W9

Canada

Scarborough Health Network Research Institute (SHNRI)

Scarborough Village, Ontario M1E 4B9

Canada

UHN-Toronto General Hospital

Toronto, Ontario M5G 2C4

Canada

University of Toronto

Toronto, Ontario M5G1X8

Canada

CHUM-Hosp de Univ Montreal

Montreal, Quebec H2X 0C1

Canada

Montreal Children's Hospital

Montreal, Quebec H4A 3J1

Canada

Hospital Pablo Tobon Uribe

Medellín, Antioquia 50034

Colombia

Clinica de la Costa

Barranquilla, Atlántico 80020

Colombia

Sociedad de Oncología y Hematología del Cesar SAS (SOHEC)

Valledupar, Cesar Department 200001

Colombia

Clínica IMAT Oncomedica Auna S.A.S.

Montería, Departamento de Córdoba 230002

Colombia

Fundacion Valle del Lili

Cali, Valle del Cauca Department 760032

Colombia

Sociedad de Oncología y Hematología del Cesar SAS (SOHEC)

Antioquia, 4006

Colombia

Centro de investigaciones clínicas, fudación valle del lili

Cali, 760032

Colombia

Clínica IMAT Oncomedica Auna S.A.S.

Montería, 230002

Colombia

Ap-Hp-Hopital Avicenne

Bobigny, 93000

France

Centre Hospitalier Universitaire de Bordeaux-Hopital Pellegrin

Bordeaux, 33000

France

Ap-Hp-Hopital Henri Mondor

Créteil, 94000

France

Centre Hospitalier Universitaire Grenoble Alpes-Site Nord Michallon

Grenoble, 38043

France

Hospices Civils de Lyon-Hopital Edouard Herriot

Lyon, 69437

France

AP-HP-HOPITAL NECKER_Service d'hématologie

Paris, 75015

France

Ap-Hp-Hopital Robert Debre

Paris, 75019

France

Centre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle

Rouen, 76031

France

Kintampo Health Research Centre (KHRC)

Kintampo, Bono East 27WF+98M

Ghana

Navrongo Health Research Centre

Navrongo, Upper East Region UB-03821

Ghana

Agogo Presbyterian Hospital, Ghana

Agogo, 0000

Ghana

"Laiko" General Hospital of Athens

Athens, 115 26

Greece

Childrens' Hospital of Athens "Agia Sofia"

Athens, 115 27

Greece

Hippokration Hospital

Athens, 11527

Greece

Hippokration Hospital

Athens, 11527

Greece

University General Hospital Of Ioannina

Ioannina, 45500

Greece

University General Hospital Of Ioannina

Ioannina, 45500

Greece

"AHEPA" University General Hospital of Thessaloniki

Thessaloniki, 546 36

Greece

"AHEPA" University General Hospital of Thessaloniki

Thessaloniki, 546 36

Greece

Gauhati Medical College and Hospital

Guwahati, Assam 781032

India

All India Institute of Medical Sciences (AIIMS), Raipur

Raipur, Chhattisgarh 492099

India

Zydus Medical College & Hospital

Dohad, Gujarat 389151

India

Nirmal Hospital Pvt. Ltd.

Surat, Gujarat 395002

India

SSG Hospital, Baroda

Vadodara, Gujarat 390001

India

HOCC Haemato Oncology Care Centre

Vadodara, Gujarat 390020

India

JSS Hospital, Mysore

Mysuru, Karnataka 570004

India

Government Medical College, Kozhikode

Kozhikode, Kerala 673008

India

Government Medical College, Kozhikode

Kozhikode, Kerala 673008

India

Arihant Multispeciality Hospital

Nagpur, Maharashtra 440026

India

IMS and SUM Hospital

Bhubaneswar, Odisha 751003

India

RNT Medical College

Udaipur, Rajasthan 313001

India

NIZAM'S Institute of Medical Sciences

Hyderabad, Telangana 500082

India

Post Graduate Institute of Child Health

Noida, Uttar Pradesh 201303

India

All India Institute of Medical Sciences_New Dehli

New Delhi, 110029

India

Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche

Orbassano, Torino 10043

Italy

Azienda Ospedaliera Universitaria San Luigi Gonzaga

Orbassano, Torino 10043

Italy

Azienda Ospedaliera Universitaria Integrata Verona Policlinico GB Rossi - Borgo Roma - Centro Ricerche Cliniche Di Verona S.r.l.

Verona, Veneto 37134

Italy

ASST Civili di Brescia

Brescia, 25123

Italy

ARNAS Garibaldi Catania - Presidio Ospedaliero Garibaldi-Centro

Catania, 95123

Italy

Azienda Ospedaliero Universitaria Di Modena - UOC Pediatria ad indirizzo Oncoematologico

Modena, 41100

Italy

Fondazione IRCCS San Gerardo dei Tintori

Monza, 20900

Italy

A.O.U. Università Studi della Campania "Luigi Vanvitelli"

Naples, 80138

Italy

Azienda Ospedale Universita Padova

Padova, 35128

Italy

Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello

Palermo, 90146

Italy

Gertrude's Children's Hospital

Nairobi, Nairobi County 00509

Kenya

KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital

Siaya, Siaya County 40600

Kenya

International Cancer Institute (ICI)

Eldoret, Uasin Gishu County 8088-30100

Kenya

Ahero Clinical Trials Unit

Kisumu, 40101

Kenya

American University of Beirut Medical Centre

Hamra, 11-0236

Lebanon

Hospital Nini

Tripoli, 1434

Lebanon

AmsterdamUMC VUMC - Hematologie

Amsterdam, 1081 HV

Netherlands

Erasmus MC

Rotterdam, 3015 GD

Netherlands

HagaZiekenhuis

The Hague, 2545AA

Netherlands

UMC Utrecht, Van Creveldkliniek

Utrecht, 3584 CX

Netherlands

University of Nigeria Teaching Hospital (UNTH)

Ituku-Ozalla, Enugu State 400001

Nigeria

Centre of Excellence for Sickle Cell Disease Research and Training, University of Abuja (CESRTA)

Abuja, Federal Capital Territory (fct) 90410

Nigeria

University College Hospital Paediatric Haematology and Oncology Unit, Ibadan

Ibadan, Oyo State 0000

Nigeria

Lagos University Teaching Hospital, Lagos

Lagos, 102215

Nigeria

Sultan Qaboos University Hospital

Muscat, Sultanet of Oman/Muscat/Al Khoud 123

Oman

The Royal Hospital

Muscat, 111

Oman

King Abdulaziz Hospital-Al Ahsa-National Guard

Al Ahsa, 36428

Saudi Arabia

Prince Mohammed bin Fahad Hospital for Blood Disorders

Al Qaţīf, 32643

Saudi Arabia

National Guard Hospital Dammam

Dhahran, 34232

Saudi Arabia

King Khalid University Hospital

Riyadh, 12372

Saudi Arabia

King Faisal Specialist Hospital & Research Centre, Riyadh

Riyadh, 12713

Saudi Arabia

Hospital Vall d'Hebron

Barcelona, 08035

Spain

Hospital Vall d'Hebron

Barcelona, 08035

Spain

Hospital Gregorio Marañón

Madrid, 28009

Spain

Hospital Universitario La Paz

Madrid, 28046

Spain

Hospital Universitario Virgen del Rocío

Seville, 41013

Spain

Acıbadem Adana Hastanesi-Hematoloji

Adana, 01130

Turkey (Türkiye)

Başkent Üniversitesi Adana-Hematoloji

Adana, 01250

Turkey (Türkiye)

Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji

Adana, 01250

Turkey (Türkiye)

Hacettepe University Hematology

Ankara, 06230

Turkey (Türkiye)

Hacettepe Üniversitesi Hastanesi- Hematoloji

Ankara, 06230

Turkey (Türkiye)

Mersin University Medical Faculty Pediatric Hematology

Mersin, 33110

Turkey (Türkiye)

Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji

Mersin, 33110

Turkey (Türkiye)

VM Medical Park Mersin Hastanesi- Hematoloji

Mersin, 33200

Turkey (Türkiye)

Makerere University College of Health Sciences (MakCHS)

Kampala, Central Region 00000

Uganda

Joint Clinical Research Centre (JCRC)

Kampala, Central Region (Wakiso District) 00000

Uganda

Jinja Regional Referral Hospital

Jinja, Eastern Region 0000

Uganda

Addenbrooke's Hospital - Haemophilia and Thrombophilia

Cambridge, CB2 0QQ

United Kingdom

Whittington Hospital

London, N19 5NF

United Kingdom

Guy's Hospital - Haematology

London, SE1 9RT

United Kingdom

King's College Hospital - Paediatric Research

London, SE5 9RS

United Kingdom

Kings College Hospital - Haematology

London, SE5 9RS

United Kingdom

Imperial College London

London, W12 0NN

United Kingdom