RECRUITING Phase 3 ClinicalTrials.gov

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

Sponsor: Novo Nordisk A/S • ClinicalTrials.gov ID: NCT06612268

Registry updated: 2026-09-17

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Description

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.

Interventions

Etavopivat (Drug)
Etavopivat will be administered orally.
Placebo (Drug)
Placebo matching Etavopivat will be administered orally.

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.

Conditions

Sickle Cell Disease

Locations

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
Hippokration Hospital — Athens, Greece
Hippokration Hospital — Athens, Greece
University General Hospital Of Ioannina — Ioannina, Greece
University General Hospital Of Ioannina — Ioannina, Greece
"AHEPA" University General Hospital of Thessaloniki — Thessaloniki, Greece
"AHEPA" University General Hospital of Thessaloniki — Thessaloniki, Greece
Gauhati Medical College and Hospital — Guwahati, Assam, India
All India Institute of Medical Sciences (AIIMS), Raipur — Raipur, Chhattisgarh, India
Zydus Medical College & Hospital — Dohad, Gujarat, India
Nirmal Hospital Pvt. Ltd. — Surat, Gujarat, India
SSG Hospital, Baroda — Vadodara, Gujarat, India
HOCC Haemato Oncology Care Centre — Vadodara, Gujarat, India
JSS Hospital, Mysore — Mysuru, Karnataka, India
Government Medical College, Kozhikode — Kozhikode, Kerala, India
Government Medical College, Kozhikode — Kozhikode, Kerala, India
Arihant Multispeciality Hospital — Nagpur, Maharashtra, India
IMS and SUM Hospital — Bhubaneswar, Odisha, India
RNT Medical College — Udaipur, Rajasthan, India
NIZAM'S Institute of Medical Sciences — Hyderabad, Telangana, India
Post Graduate Institute of Child Health — Noida, Uttar Pradesh, India
All India Institute of Medical Sciences_New Dehli — New Delhi, India
Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche — Orbassano, Torino, Italy
Azienda Ospedaliera Universitaria San Luigi Gonzaga — Orbassano, Torino, Italy
Azienda Ospedaliera Universitaria Integrata Verona Policlinico GB Rossi - Borgo Roma - Centro Ricerche Cliniche Di Verona S.r.l. — Verona, Veneto, Italy
ASST Civili di Brescia — Brescia, Italy
ARNAS Garibaldi Catania - Presidio Ospedaliero Garibaldi-Centro — Catania, Italy
Azienda Ospedaliero Universitaria Di Modena - UOC Pediatria ad indirizzo Oncoematologico — Modena, Italy
Fondazione IRCCS San Gerardo dei Tintori — Monza, Italy
A.O.U. Università Studi della Campania "Luigi Vanvitelli" — Naples, Italy
Azienda Ospedale Universita Padova — Padova, Italy
Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello — Palermo, Italy
Gertrude's Children's Hospital — Nairobi, Nairobi County, Kenya
KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital — Siaya, Siaya County, Kenya
International Cancer Institute (ICI) — Eldoret, Uasin Gishu County, Kenya
Ahero Clinical Trials Unit — Kisumu, Kenya
American University of Beirut Medical Centre — Hamra, Lebanon
Hospital Nini — Tripoli, Lebanon
AmsterdamUMC VUMC — Amsterdam, Netherlands
Erasmus MC — Rotterdam, Netherlands
HagaZiekenhuis — The Hague, Netherlands
UMC Utrecht, Van Creveldkliniek — Utrecht, Netherlands
University of Nigeria Teaching Hospital (UNTH) — Ituku-Ozalla, Enugu State, Nigeria
Centre of Excellence for Sickle Cell Disease Research and Training, University of Abuja (CESRTA) — Abuja, Federal Capital Territory (fct), Nigeria
University College Hospital Paediatric Haematology and Oncology Unit, Ibadan — Ibadan, Oyo State, Nigeria
Lagos University Teaching Hospital, Lagos — Lagos, Nigeria
Sultan Qaboos University Hospital — Muscat, Sultanet of Oman/Muscat/Al Khoud, Oman
The Royal Hospital — Muscat, Oman
Al-Moosa Health Group — Al Ahsa, Saudi Arabia
King Abdulaziz Hospital-Al Ahsa-National Guard — Al Ahsa, Saudi Arabia
Prince Mohammed bin Fahad Hospital for Blood Disorders — Al Qaţīf, Saudi Arabia
National Guard Hospital Dammam — Dhahran, Saudi Arabia
King Khalid University Hospital — Riyadh, Saudi Arabia
King Faisal Specialist Hospital & Research Centre, Riyadh — Riyadh, Saudi Arabia
Hospital Vall d'Hebron — Barcelona, Spain
Hospital Vall d'Hebron — Barcelona, Spain
Hospital Gregorio Marañón — Madrid, Spain
Hospital Universitario La Paz — Madrid, Spain
Hospital Universitario Virgen del Rocío — Seville, Spain
Acıbadem Adana Hastanesi-Hematoloji — Adana, Turkey (Türkiye)
Başkent Üniversitesi Adana-Hematoloji — Adana, Turkey (Türkiye)
Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji — Adana, Turkey (Türkiye)
Hacettepe University Hematology — Ankara, Turkey (Türkiye)
Hacettepe Üniversitesi Hastanesi- Hematoloji — Ankara, Turkey (Türkiye)
Mersin University Medical Faculty Pediatric Hematology — Mersin, Turkey (Türkiye)
Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji — Mersin, Turkey (Türkiye)
VM Medical Park Mersin Hastanesi- Hematoloji — Mersin, Turkey (Türkiye)
Makerere University College of Health Sciences (MakCHS) — Kampala, Central Region, Uganda
Joint Clinical Research Centre (JCRC) — Kampala, Central Region (Wakiso District), Uganda
Jinja Regional Referral Hospital — Jinja, Eastern Region, Uganda
Addenbrooke's Hospital - Haemophilia and Thrombophilia — Cambridge, United Kingdom
Whittington Hospital — London, United Kingdom
Guy's Hospital - Haematology — London, United Kingdom
King's College Hospital - Paediatric Research — London, United Kingdom
Kings College Hospital - Haematology — London, United Kingdom
Imperial College London — London, United Kingdom

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Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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