A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease (Hibiscus 2)

September 16, 2026 updated by: Novo Nordisk A/S

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

510

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
    • New South Wales
      • Camperdown, New South Wales, Australia, 2050
        • Recruiting
        • Royal Prince Alfred Hospital - Department of Haematology
      • Randwick, New South Wales, Australia, 2031
        • Recruiting
        • Prince of Wales Hospital - Department of Haematology
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Recruiting
        • Monash Medical Centre - Haematology Department
    • Western Australia
      • Murdoch, Western Australia, Australia, 6150
        • Recruiting
        • Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
  • Belgium
      • Brussels, Belgium, 1070
        • Recruiting
        • HUB - Hôpital Erasme
      • Brussels, Belgium, 1020
        • Not yet recruiting
        • CHU - UVC Brugmann
      • Brussels, Belgium, 1000
        • Recruiting
        • CHU Saint-Pierre - UMC Sint-Pieter
      • Edegem, Belgium, 2650
        • Recruiting
        • UZ Antwerpen - UZA - Kinderhemato-Oncologie
      • Edegem, Belgium, 2650
        • Recruiting
        • UZ Antwerpen - UZA - Haematology
      • Leuven, Belgium, 3000
        • Recruiting
        • UZ Leuven - Kindergeneeskunde
  • Brazil
      • Rio de Janeiro, Brazil, 20211-030
        • Recruiting
        • Hemorio-Fundarj
      • São Paulo, Brazil, 01229-001
        • Recruiting
        • Hospital Samaritano Higienópolis - Instituto de Conhecimento Ensino e Pesquisa (ICEP)
    • Pernambuco
      • Recife, Pernambuco, Brazil, 52070-460
        • Not yet recruiting
        • MultiHemo - Grupo Oncoclínicas
      • Recife, Pernambuco, Brazil, 50070-460
        • Recruiting
        • MultiHemo - Grupo Oncoclínicas
    • São
      • Ribeirão Preto, São, Brazil, 14051-140
        • Recruiting
        • Fundação Hemocentro de Ribeirão Preto
    • São Paulo
      • Distrito de Barão Geraldo - Campinas, São Paulo, Brazil, 13083-878
        • Recruiting
        • Hemocentro UNICAMP
      • São Paulo, São Paulo, Brazil, 05403-010
        • Not yet recruiting
        • HC da FMUSP Hospital das Clínicas São Paulo
      • São Paulo, São Paulo, Brazil, 05403-010
        • Recruiting
        • HC da FMUSP Hospital das Clínicas São Paulo
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 2T9
        • Recruiting
        • Foothills Med Ctr-Univ Calgary
      • Edmonton, Alberta, Canada, T6G 2B7
        • Recruiting
        • Stollery Children's Hospital
      • Edmonton, Alberta, Canada, T6G 2B7
        • Recruiting
        • University of Alberta_Edmonton
    • British Columbia
      • Vancouver, British Columbia, Canada, V6H 3V4
        • Recruiting
        • BC Children's Hospital
      • Vancouver, British Columbia, Canada, V6Z 2A5
        • Not yet recruiting
        • St Pauls Hospital
    • Ontario
      • London, Ontario, Canada, N6A5W9
        • Recruiting
        • LHSC - Victoria Hospital
      • Scarborough Village, Ontario, Canada, M1E 4B9
        • Not yet recruiting
        • Scarborough Health Network Research Institute (SHNRI)
      • Toronto, Ontario, Canada, M5G 2C4
        • Completed
        • UHN-Toronto General Hospital
      • Toronto, Ontario, Canada, M5G1X8
        • Recruiting
        • University of Toronto
    • Quebec
      • Montreal, Quebec, Canada, H4A 3J1
        • Recruiting
        • Montreal Children's Hospital
      • Montreal, Quebec, Canada, H2X 0C1
        • Recruiting
        • CHUM-Hosp de Univ Montreal
  • Colombia
      • Antioquia, Colombia, 4006
        • Not yet recruiting
        • Sociedad de Oncología y Hematología del Cesar SAS (SOHEC)
      • Cali, Colombia, 760032
        • Not yet recruiting
        • Centro de investigaciones clínicas, fudación valle del lili
      • Montería, Colombia, 230002
        • Not yet recruiting
        • Clínica IMAT Oncomedica Auna S.A.S.
    • Antioquia
      • Medellín, Antioquia, Colombia, 50034
        • Not yet recruiting
        • Hospital Pablo Tobon Uribe
    • Atlántico
      • Barranquilla, Atlántico, Colombia, 80020
        • Recruiting
        • Clinica de la Costa
    • Cesar Department
      • Valledupar, Cesar Department, Colombia, 200001
        • Recruiting
        • Sociedad de Oncología y Hematología del Cesar SAS (SOHEC)
    • Departamento de Córdoba
      • Montería, Departamento de Córdoba, Colombia, 230002
        • Recruiting
        • Clínica IMAT Oncomedica Auna S.A.S.
    • Valle del Cauca Department
      • Cali, Valle del Cauca Department, Colombia, 760032
        • Not yet recruiting
        • Fundacion Valle del Lili
  • France
      • Bobigny, France, 93000
        • Not yet recruiting
        • Ap-Hp-Hopital Avicenne
      • Bordeaux, France, 33000
        • Recruiting
        • Centre Hospitalier Universitaire de Bordeaux-Hopital Pellegrin
      • Créteil, France, 94000
        • Recruiting
        • Ap-Hp-Hopital Henri Mondor
      • Grenoble, France, 38043
        • Recruiting
        • Centre Hospitalier Universitaire Grenoble Alpes-Site Nord Michallon
      • Lyon, France, 69437
        • Recruiting
        • Hospices Civils de Lyon-Hopital Edouard Herriot
      • Paris, France, 75019
        • Recruiting
        • Ap-Hp-Hopital Robert Debre
      • Paris, France, 75015
        • Not yet recruiting
        • AP-HP-HOPITAL NECKER_Service d'hématologie
      • Rouen, France, 76031
        • Not yet recruiting
        • Centre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle
  • Ghana
      • Agogo, Ghana, 0000
        • Completed
        • Agogo Presbyterian Hospital, Ghana
    • Bono East
      • Kintampo, Bono East, Ghana, 27WF+98M
        • Not yet recruiting
        • Kintampo Health Research Centre (KHRC)
    • Upper East Region
      • Navrongo, Upper East Region, Ghana, UB-03821
        • Not yet recruiting
        • Navrongo Health Research Centre
  • Greece
      • Athens, Greece, 11527
        • Not yet recruiting
        • Hippokration Hospital
      • Athens, Greece, 11527
        • Recruiting
        • Hippokration Hospital
      • Athens, Greece, 115 26
        • Recruiting
        • "Laiko" General Hospital of Athens
      • Athens, Greece, 115 27
        • Recruiting
        • Childrens' Hospital of Athens "Agia Sofia"
      • Ioannina, Greece, 45500
        • Recruiting
        • University General Hospital Of Ioannina
      • Ioannina, Greece, 45500
        • Not yet recruiting
        • University General Hospital Of Ioannina
      • Thessaloniki, Greece, 546 36
        • Not yet recruiting
        • "AHEPA" University General Hospital of Thessaloniki
      • Thessaloniki, Greece, 546 36
        • Recruiting
        • "AHEPA" University General Hospital of Thessaloniki
  • India
      • New Delhi, India, 110029
        • Recruiting
        • All India Institute of Medical Sciences_New Dehli
    • Assam
      • Guwahati, Assam, India, 781032
        • Recruiting
        • Gauhati Medical College and Hospital
    • Chhattisgarh
      • Raipur, Chhattisgarh, India, 492099
        • Recruiting
        • All India Institute of Medical Sciences (AIIMS), Raipur
    • Gujarat
      • Dohad, Gujarat, India, 389151
        • Recruiting
        • Zydus Medical College & Hospital
      • Surat, Gujarat, India, 395002
        • Recruiting
        • Nirmal Hospital Pvt. Ltd.
      • Vadodara, Gujarat, India, 390001
        • Recruiting
        • SSG Hospital, Baroda
      • Vadodara, Gujarat, India, 390020
        • Recruiting
        • HOCC Haemato Oncology Care Centre
    • Karnataka
      • Mysuru, Karnataka, India, 570004
        • Recruiting
        • JSS Hospital, Mysore
    • Kerala
      • Kozhikode, Kerala, India, 673008
        • Recruiting
        • Government Medical College, Kozhikode
      • Kozhikode, Kerala, India, 673008
        • Not yet recruiting
        • Government Medical College, Kozhikode
    • Maharashtra
      • Nagpur, Maharashtra, India, 440026
        • Recruiting
        • Arihant Multispeciality Hospital
    • Odisha
      • Bhubaneswar, Odisha, India, 751003
        • Recruiting
        • IMS and SUM Hospital
    • Rajasthan
      • Udaipur, Rajasthan, India, 313001
        • Recruiting
        • RNT Medical College
    • Telangana
      • Hyderabad, Telangana, India, 500082
        • Recruiting
        • Nizam's Institute of Medical Sciences
    • Uttar Pradesh
      • Noida, Uttar Pradesh, India, 201303
        • Recruiting
        • Post Graduate Institute of Child Health
  • Italy
      • Brescia, Italy, 25123
        • Recruiting
        • ASST Civili di Brescia
      • Catania, Italy, 95123
        • Recruiting
        • ARNAS Garibaldi Catania - Presidio Ospedaliero Garibaldi-Centro
      • Modena, Italy, 41100
        • Not yet recruiting
        • Azienda Ospedaliero Universitaria Di Modena - UOC Pediatria ad indirizzo Oncoematologico
      • Monza, Italy, 20900
        • Recruiting
        • Fondazione IRCCS San Gerardo dei Tintori
      • Naples, Italy, 80138
        • Recruiting
        • A.O.U. Università Studi della Campania "Luigi Vanvitelli"
      • Padova, Italy, 35128
        • Recruiting
        • Azienda Ospedale Universita Padova
      • Palermo, Italy, 90146
        • Recruiting
        • Azienda Ospedaliera Ospedali Riuniti Villa Sofia - Cervello
    • Torino
      • Orbassano, Torino, Italy, 10043
        • Not yet recruiting
        • Azienda Ospedaliera Universitaria San Luigi Gonzaga
      • Orbassano, Torino, Italy, 10043
        • Recruiting
        • Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche
    • Veneto
      • Verona, Veneto, Italy, 37134
        • Recruiting
        • Azienda Ospedaliera Universitaria Integrata Verona Policlinico GB Rossi - Borgo Roma - Centro Ricerche Cliniche Di Verona S.r.l.
  • Kenya
      • Kisumu, Kenya, 40101
        • Not yet recruiting
        • Ahero Clinical Trials Unit
    • Nairobi County
      • Nairobi, Nairobi County, Kenya, 00509
        • Not yet recruiting
        • Gertrude's Children's Hospital
    • Siaya County
      • Siaya, Siaya County, Kenya, 40600
        • Not yet recruiting
        • KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital
    • Uasin Gishu County
      • Eldoret, Uasin Gishu County, Kenya, 8088-30100
        • Not yet recruiting
        • International Cancer Institute (ICI)
  • Lebanon
      • Hamra, Lebanon, 11-0236
        • Recruiting
        • American University of Beirut Medical Centre
      • Tripoli, Lebanon, 1434
        • Recruiting
        • Hospital Nini
  • Netherlands
      • Amsterdam, Netherlands, 1081 HV
        • Recruiting
        • AmsterdamUMC VUMC
      • Rotterdam, Netherlands, 3015 GD
        • Recruiting
        • Erasmus MC
      • The Hague, Netherlands, 2545AA
        • Recruiting
        • HagaZiekenhuis
      • Utrecht, Netherlands, 3584 CX
        • Recruiting
        • UMC Utrecht, Van Creveldkliniek
  • Nigeria
      • Lagos, Nigeria, 102215
        • Not yet recruiting
        • Lagos University Teaching Hospital, Lagos
    • Enugu State
      • Ituku-Ozalla, Enugu State, Nigeria, 400001
        • Not yet recruiting
        • University of Nigeria Teaching Hospital (UNTH)
    • Federal Capital Territory (fct)
      • Abuja, Federal Capital Territory (fct), Nigeria, 90410
        • Not yet recruiting
        • Centre of Excellence for Sickle Cell Disease Research and Training, University of Abuja (CESRTA)
    • Oyo State
      • Ibadan, Oyo State, Nigeria, 0000
        • Not yet recruiting
        • University College Hospital Paediatric Haematology and Oncology Unit, Ibadan
  • Oman
      • Muscat, Oman, 111
        • Completed
        • The Royal Hospital
    • Sultanet of Oman/Muscat/Al Khoud
      • Muscat, Sultanet of Oman/Muscat/Al Khoud, Oman, 123
        • Recruiting
        • Sultan Qaboos University Hospital
  • Saudi Arabia
      • Al Ahsa, Saudi Arabia, 36428
        • Recruiting
        • King Abdulaziz Hospital-Al Ahsa-National Guard
      • Al Ahsa, Saudi Arabia, 36342
        • Not yet recruiting
        • Al-Moosa Health Group
      • Al Qaţīf, Saudi Arabia, 32643
        • Recruiting
        • Prince Mohammed bin Fahad Hospital for Blood Disorders
      • Dhahran, Saudi Arabia, 34232
        • Recruiting
        • National Guard Hospital Dammam
      • Riyadh, Saudi Arabia, 12713
        • Recruiting
        • King Faisal Specialist Hospital & Research Centre, Riyadh
      • Riyadh, Saudi Arabia, 12372
        • Recruiting
        • King Khalid University Hospital
  • Spain
      • Barcelona, Spain, 08035
        • Recruiting
        • Hospital Vall d'hebrón
      • Barcelona, Spain, 08035
        • Not yet recruiting
        • Hospital Vall d'hebrón
      • Madrid, Spain, 28046
        • Recruiting
        • Hospital Universitario La Paz
      • Madrid, Spain, 28009
        • Recruiting
        • Hospital Gregorio Marañón
      • Seville, Spain, 41013
        • Recruiting
        • Hospital Universitario Virgen del Rocío
  • Turkey (Türkiye)
      • Adana, Turkey (Türkiye), 01130
        • Recruiting
        • Acıbadem Adana Hastanesi-Hematoloji
      • Adana, Turkey (Türkiye), 01250
        • Recruiting
        • Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji
      • Adana, Turkey (Türkiye), 01250
        • Recruiting
        • Başkent Üniversitesi Adana-Hematoloji
      • Ankara, Turkey (Türkiye), 06230
        • Recruiting
        • Hacettepe University Hematology
      • Ankara, Turkey (Türkiye), 06230
        • Recruiting
        • Hacettepe Üniversitesi Hastanesi- Hematoloji
      • Mersin, Turkey (Türkiye), 33110
        • Not yet recruiting
        • Mersin University Medical Faculty Pediatric Hematology
      • Mersin, Turkey (Türkiye), 33110
        • Recruiting
        • Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji
      • Mersin, Turkey (Türkiye), 33200
        • Recruiting
        • VM Medical Park Mersin Hastanesi- Hematoloji
  • Uganda
    • Central Region
      • Kampala, Central Region, Uganda, 00000
        • Not yet recruiting
        • Makerere University College of Health Sciences (MakCHS)
    • Central Region (Wakiso District)
      • Kampala, Central Region (Wakiso District), Uganda, 00000
        • Not yet recruiting
        • Joint Clinical Research Centre (JCRC)
    • Eastern Region
      • Jinja, Eastern Region, Uganda, 0000
        • Not yet recruiting
        • Jinja Regional Referral Hospital
  • United Kingdom
      • Cambridge, United Kingdom, CB2 0QQ
        • Recruiting
        • Addenbrooke's Hospital - Haemophilia and Thrombophilia
      • London, United Kingdom, N19 5NF
        • Recruiting
        • Whittington Hospital
      • London, United Kingdom, W12 0NN
        • Recruiting
        • Imperial College London
      • London, United Kingdom, SE5 9RS
        • Recruiting
        • Kings College Hospital - Haematology
      • London, United Kingdom, SE5 9RS
        • Recruiting
        • King's College Hospital - Paediatric Research
      • London, United Kingdom, SE1 9RT
        • Recruiting
        • Guy's Hospital - Haematology
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35233
        • Recruiting
        • Uni of Alabama at Birmingham
      • Mobile, Alabama, United States, 36604
        • Recruiting
        • Univer South Alabama Ped/Onc
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Recruiting
        • Phoenix Children's Hsptl
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
        • Recruiting
        • Arkansas Children's Hospital
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital Los Angeles - Endocrinology
      • Los Angeles, California, United States, 90095
        • Completed
        • UCLA Health
      • Madera, California, United States, 93636
        • Recruiting
        • Valley Children's Hospital
      • Orange, California, United States, 92868
        • Completed
        • University of California Irvine
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Stanford University_Palo Alto
      • Torrance, California, United States, 90502
        • Recruiting
        • Harbor-UCLA Medical Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Completed
        • Clinical and Transl Res Center
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Recruiting
        • Nemours/AI duPont Hosp-Chld
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • MedStar Hlth Res Institute
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • Childrens National Medical Ctr
    • Florida
      • Hollywood, Florida, United States, 33021
        • Recruiting
        • Memorial Healthcare
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Recruiting
        • Children's Healthcare Atlanta
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Childrens Hospital of Chicago
      • Chicago, Illinois, United States, 60612
        • Recruiting
        • Univer Of Illinois at Chicago
    • Louisiana
      • New Orleans, Louisiana, United States, 70118
        • Recruiting
        • Children's Hosp-New Orleans
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Boston Children's Hospital
      • Boston, Massachusetts, United States, 02118-2393
        • Recruiting
        • Boston Medical Center
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Completed
        • Henry Ford Hospital_Detroit
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • University of Minnesota
    • Missouri
      • St Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University-St.Louis
    • Nebraska
      • Omaha, Nebraska, United States, 68114
        • Recruiting
        • Children's Nebraska
    • Nevada
      • Las Vegas, Nevada, United States, 89135
        • Completed
        • Cure 4 the Kids Foundation
    • New Jersey
      • Newark, New Jersey, United States, 07112-2027
        • Recruiting
        • Newark Beth Israel MC
    • New York
      • Brooklyn, New York, United States, 11203
        • Recruiting
        • NYC Health+Hospitals
      • Brooklyn, New York, United States, 11238
        • Recruiting
        • Interfaith Medical Center
      • Mount Kisco, New York, United States, 10549
        • Completed
        • Northwell Health
      • Queens, New York, United States, 11040
        • Recruiting
        • Cohen Children's Medical Ctr
      • The Bronx, New York, United States, 10461
        • Recruiting
        • Jacobi Medical Center
      • The Bronx, New York, United States, 10467
        • Recruiting
        • Montefiore Medical Ctr
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical Center
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke Comprehen Sickle Cell
      • Greenville, North Carolina, United States, 27834
        • Recruiting
        • East Carolina Univ-Greenville
      • Winston-Salem, North Carolina, United States, 27157
        • Recruiting
        • Atrium Health-Wake Forest Bapt
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • Univ Hosp Cleveland Med Ctr
      • Columbus, Ohio, United States, 43203
        • Recruiting
        • Ohio State Univ Wexner Med Ctr
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73104
        • Recruiting
        • Univ of OK Health Sciences Ctr
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19134
        • Recruiting
        • St Christopher Hosp for Child
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hosptl Philadelphia
    • Tennessee
      • Memphis, Tennessee, United States, 38105
        • Recruiting
        • St. Jude Children's Res Hosp
      • Memphis, Tennessee, United States, 38104
        • Recruiting
        • University of Tennessee Health Science Center
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • UT Health University of Texas
    • Virginia
      • Fairfax, Virginia, United States, 22031
        • Recruiting
        • Inova Health System
      • Norfolk, Virginia, United States, 23507
        • Recruiting
        • Children's Hsptl Of The Kings
      • Richmond, Virginia, United States, 23298
        • Recruiting
        • Virginia Comm Univ Medical Ctr
    • Washington
      • Tacoma, Washington, United States, 98405
        • Recruiting
        • Mary Bridge Children's Health
      • Tacoma, Washington, United States, 98405
        • Not yet recruiting
        • Mary Bridge Children's Health

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Etavopivat
Participants will be randomised to receive oral dose of Etavopivat.
Drug: Etavopivat
Etavopivat will be administered orally.
Placebo Comparator: Placebo
Participants will be randomised to receive oral dose of placebo.
Drug: Placebo
Placebo matching Etavopivat will be administered orally.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact
Time Frame: Baseline (week 0) to week 52
Measured as Count of events.
Baseline (week 0) to week 52

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to onset of first adjudicated Vaso-occlusive crisis (VOC)
Time Frame: Baseline (week 0) to week 52
Measured as time in days.
Baseline (week 0) to week 52
Change in distance travelled during the 6-minute walking test (6MWT)
Time Frame: Baseline (week 0) to week 52
Measured in Meters.
Baseline (week 0) to week 52
Change in standardised T-score on the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 7a Scale
Time Frame: Baseline (week 0) to week 52
Measured in T-score.
Baseline (week 0) to week 52
Change in haemoglobin (Hb)
Time Frame: Baseline (week 0) to week 52
Measured in grams per decilitre (g/dL).
Baseline (week 0) to week 52
Change in lactate dehydrogenase (LDH)
Time Frame: Baseline (week 0) to week 52
Measured in Units per litre (U/L).
Baseline (week 0) to week 52
Change in absolute reticulocyte count
Time Frame: Baseline (week 0) to week 52
Measured as Cells x10^9/L.
Baseline (week 0) to week 52
Change in indirect bilirubin
Time Frame: Baseline (week 0) to week 52
Measured in milligrams per decilitre (mg/dL).
Baseline (week 0) to week 52
Change in Haemoglobin (Hb) greater than 1 grams per decilitre (g/dL)
Time Frame: Baseline (week 0) to week 24
Measured in Count of participants.
Baseline (week 0) to week 24
Participants achieving the threshold for clinically meaningful change (yes/no) in PROMIS fatigue scale 7a
Time Frame: Baseline (week 0) to week 52
Measured as Count of participants.
Baseline (week 0) to week 52
Participants achieving the threshold for clinically meaningful change (yes/no) in 6MWT
Time Frame: Baseline (week 0) to week 52
Measured as Count of participants.
Baseline (week 0) to week 52

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novo Nordisk A/S

Investigators

  • Study Director: Clinical Transparency (dept. 2834), Novo Nordisk A/S

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 17, 2025

Primary Completion (Estimated)

August 27, 2027

Study Completion (Estimated)

August 12, 2029

Study Registration Dates

First Submitted

September 23, 2024

First Submitted That Met QC Criteria

September 23, 2024

First Posted (Actual)

September 25, 2024

Study Record Updates

Last Update Posted (Actual)

September 17, 2026

Last Update Submitted That Met QC Criteria

September 16, 2026

Last Verified

September 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NN7535-7807
  • 2023 (U.S. NIH Grant/Contract: GRAMMY Museum Foundation)
  • U1111-1298-3431 (Other Identifier: World Health Organisation (WHO))
  • 2023-509175-16 (Other Identifier: European Medical Agency (EMA))

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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