A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

NCT06609226 | Recruiting Phase 3 FDA Drug

• 3 other identifiers: NN7535-7822U1111-1301-81302024-510805-27
• Study Type: interventional
• Target: 480
• Locations: 17 countries
• Sites: 103

Brief Summary

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

Conditions

Sickle Cell Disease; Thalassemia

Outcome Measures

Primary Outcomes (2)

• Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately

  Measured as number of events.

  Baseline (week 0 of FLORAL) up to end of study (up to week 316)

• Number of adverse reactions, reported for each indication and age group separately

  Measured as number of adverse reactions.

  Baseline (week 0 of FLORAL) up to end of study (up to week 316)

Secondary Outcomes (8)

• Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately

  Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)

• Change in VOCs, reported for each age group separately

  Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)

• Change in hemoglobin (Hb) concentration, reported for each age group separately

  Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)

• Annualised number of hospitalisations, reported for each age group separately

  Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)

• Average length of stay of hospitalisations, reported for each age group separately

  Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)

Study Arms (5)

Participants greater than or equal to (≥) 12 years old with sickle cell disease

EXPERIMENTAL

Participants will receive an oral dose of Etavopivat A or C.

Drug: Etavopivat A; Drug: Etavopivat C

Participants ≥ 12 years old with sickle cell disease transfusion dependent

EXPERIMENTAL

Participants will receive an oral dose of Etavopivat A or C.

Drug: Etavopivat A; Drug: Etavopivat C

Participants ≥ 12 years old with transfusion-dependent thalassaemia

EXPERIMENTAL

Participants will receive an oral dose of Etavopivat A or C.

Drug: Etavopivat A; Drug: Etavopivat C

Participants ≥ 12 years old with non-transfusion dependent thalassaemia

EXPERIMENTAL

Participants will receive an oral dose of Etavopivat A or C.

Drug: Etavopivat A; Drug: Etavopivat C

Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease

EXPERIMENTAL

Participants ≥ 12 years of age will receive an oral dose of Etavopivat A or C and participants \< 12 years of age will receive an oral dose of Etavopivat B.

Drug: Etavopivat A; Drug: Etavopivat B; Drug: Etavopivat C

Interventions

Etavopivat A DRUG

Participants will receive an oral dose of Etavopivat A.

Participants greater than or equal to (≥) 12 years old with sickle cell disease; Participants ≥ 12 years old with non-transfusion dependent thalassaemia; Participants ≥ 12 years old with sickle cell disease transfusion dependent; Participants ≥ 12 years old with transfusion-dependent thalassaemia; Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease

Etavopivat B DRUG

Participants will receive an oral dose of Etavopivat B.

Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease

Etavopivat C DRUG

Participants will receive an oral dose of Etavopivat C.

Participants greater than or equal to (≥) 12 years old with sickle cell disease; Participants ≥ 12 years old with non-transfusion dependent thalassaemia; Participants ≥ 12 years old with sickle cell disease transfusion dependent; Participants ≥ 12 years old with transfusion-dependent thalassaemia; Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study.
• Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator.
• Any participant with dose reduction or temporary discontinuation will need to be successfully rechallenged to the full dose of etavopivat before transferring.
• Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they have been on a stable dose in the parent study as defined at the investigator's discretion. Necessary adjustments related to weight or age are accepted. Participants with temporary dose reductions or pauses due to medical reasons may still be considered to have a stable dose, as determined by the investigator, who will assess the impact of these adjustments based on clinical context and the participant's overall health status.

You may not qualify if:

• Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
• Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study.
• Participants on permanent dose reduction (greater than \[\>\] 28 days or more) or ongoing temporary treatment discontinuation.
• Use of any of the following within the timeframes prior to the transfer visit as stated:
• Use of haemoglobin S (HbS) polymerisation inhibitors within participation of the parent study or anticipated need for this agent during this study.
• Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within the parent study or anticipated need for such agents during this study.
• Use of erythropoietin or other haematopoietic growth factor treatment for more than 4 consecutive weeks during the parent study or anticipated need of such agents for a maintenance treatment during this study.
• Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4 within 2 weeks of the transfer visit or anticipated need for such agents during the study.
• Current participation in a study that is not a designated parent study, or planned participation in any other clinical study, for the duration of FLORAL.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (103)

Univ of Alabama Birmingham

Birmingham, Alabama, 35233, United States

ACTIVE NOT RECRUITING

Phoenix Children's Hsptl

Phoenix, Arizona, 85016, United States

RECRUITING

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States

ACTIVE NOT RECRUITING

Children's Hospital Los Angeles - Endocrinology

Los Angeles, California, 90027, United States

RECRUITING

UCSF Oakland Benioff ChildHosp

Oakland, California, 94609, United States

ACTIVE NOT RECRUITING

UCSF Oakland Benioff ChildHosp

Oakland, California, 94609, United States

RECRUITING

Children's Hosp Of Orange

Orange, California, 92868, United States

COMPLETED

University Of California Irvine

Orange, California, 92868, United States

RECRUITING

University of Connecticut

Farmington, Connecticut, 06030, United States

ACTIVE NOT RECRUITING

Children's National Medical Center

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

COMPLETED

Foundation for Sickle Cell Disease Research

Hollywood, Florida, 33023, United States

RECRUITING

Univ of Miami/SCCC

Miami, Florida, 33136, United States

COMPLETED

Emory University School of Medicine

Atlanta, Georgia, 30303, United States

ACTIVE NOT RECRUITING

Children's Healthcare Atlanta

Atlanta, Georgia, 30329, United States

RECRUITING

Center for Blood Disorders Augusta University

Augusta, Georgia, 30912, United States

RECRUITING

Univer Of Illinois at Chicago

Chicago, Illinois, 60612, United States

NOT YET RECRUITING

Children's Hosp-New Orleans

New Orleans, Louisiana, 70118, United States

COMPLETED

Boston Medical Center

Boston, Massachusetts, 02118, United States

ACTIVE NOT RECRUITING

Washington University-St.Louis

St Louis, Missouri, 63110, United States

RECRUITING

NYC Health+Hospitals

Brooklyn, New York, 11203, United States

RECRUITING

Columbia University Medical Center_New York_0

New York, New York, 10032, United States

RECRUITING

Columbia University Medical Center_New York_0

New York, New York, 10032, United States

NOT YET RECRUITING

Weill Cornell Med Coll-NYPH

New York, New York, 10065, United States

RECRUITING

Jacobi Medical Center

The Bronx, New York, 10461, United States

ACTIVE NOT RECRUITING

Montefiore Medical Center

The Bronx, New York, 10467, United States

NOT YET RECRUITING

Atrium Levine Children's/Atrium Health

Charlotte, North Carolina, 28204, United States

NOT YET RECRUITING

Duke University_Durham

Durham, North Carolina, 27705, United States

RECRUITING

East Carolina Univ-Greenville

Greenville, North Carolina, 27834, United States

ACTIVE NOT RECRUITING

East Carolina University_Greenville

Greenville, North Carolina, 27834, United States

COMPLETED

Atrium Health-Wake Forest Bapt

Winston-Salem, North Carolina, 27157, United States

RECRUITING

Cincinnati Child's Hsp Med Ctr

Cincinnati, Ohio, 45229, United States

ACTIVE NOT RECRUITING

Neuro-Behavioral Clinical Research

North Canton, Ohio, 44720, United States

ACTIVE NOT RECRUITING

Medical University Of South Carolina_Charleston

Charleston, South Carolina, 29425, United States

RECRUITING

Methodist University Hospital

Memphis, Tennessee, 38104, United States

RECRUITING

Texas Children's Hospital_Houston

Houston, Texas, 77030, United States

RECRUITING

UT Health University of Texas

Houston, Texas, 77030, United States

RECRUITING

Virginia Comm Univ Medical Ctr

Richmond, Virginia, 23298, United States

RECRUITING

Mary Bridge Children's Health

Tacoma, Washington, 98405, United States

ACTIVE NOT RECRUITING

Versiti, CCBD_Milwaukee

Milwaukee, Wisconsin, 53226, United States

NOT YET RECRUITING

St Pauls Hospital

Vancouver, British Columbia, V6Z1Y6, Canada

ACTIVE NOT RECRUITING

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

ACTIVE NOT RECRUITING

University Health Network - Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

ACTIVE NOT RECRUITING

CHU Ste-Justine

Montreal, Quebec, H3T 1C5, Canada

ACTIVE NOT RECRUITING

Alexandria University Hospital

Alexandria, Egypt, 21131, Egypt

NOT YET RECRUITING

Zagazig University Hospital

Alsharkia, Egypt, 44519, Egypt

NOT YET RECRUITING

Faculty of Medicine Ain Shams Medical Research Institute (MASRI)

Cairo, 1181, Egypt

NOT YET RECRUITING

Cairo University

Cairo, Egypt, 11956, Egypt

RECRUITING

Abu El-Reesh El-Mounira Children University Hospital

Cairo, Egypt, 4241317, Egypt

RECRUITING

Ap-Hp-Hopital Henri Mondor

Créteil, 94000, France

NOT YET RECRUITING

Hospices Civils de Lyon-Hopital Edouard Herriot

Lyon, 69437, France

NOT YET RECRUITING

Ap-Hp-Hopital Robert Debre

Paris, 75019, France

RECRUITING

Ap-Hp-Hopital Robert Debre

Paris, 75019, France

NOT YET RECRUITING

Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie

Berlin, 13353, Germany

NOT YET RECRUITING

Universitätsklinikum Freiburg - Kinder- und Jugendklinik

Freiburg im Breisgau, 79106, Germany

RECRUITING

Kintampo Health Research Centre (KHRC)

Kintampo, Bono East, 27WF+98M, Ghana

NOT YET RECRUITING

Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH)

Accra, 0, Ghana

NOT YET RECRUITING

General Hospital Of Larissa Koutlibaneio And Triantafylleio - Thalassemia and SCD Unit

Larissa, Thessaly, 41221, Greece

RECRUITING

Hippokration Hospital

Athens, 11527, Greece

RECRUITING

General University Hospital of Patras

Pátrai, 26504, Greece

RECRUITING

'Ippokrateio' General Hospital of Thessaloniki

Thessaloniki, 54642, Greece

RECRUITING

All India Institute of Medical Sciences (AIIMS), Raipur

Raipur, Chhattisgarh, 492099, India

NOT YET RECRUITING

K.J Somaiya Hospital and Research Centre

Mumbai, Maharashtra, 400022, India

NOT YET RECRUITING

Victoria Hospital, Bangalore

Bangalore, 560002, India

NOT YET RECRUITING

Nirmal Hospital Pvt. Ltd.

Gujarat, 395002, India

NOT YET RECRUITING

Suretech Hospital and Research Centre Ltd.

Maharashtra, 440012, India

NOT YET RECRUITING

All India Institute of Medical Sciences_Delhi

New Delhi, 110029, India

NOT YET RECRUITING

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

Orbassano, Torino, 10043, Italy

RECRUITING

Azienda Ospedale Universita Padova

Padua, 35128, Italy

RECRUITING

Fondazione IRCCS Policlinico San Matteo

Pavia, 27100, Italy

RECRUITING

Gertrude's Children's Hospital

Nairobi, Nairobi County, 00509, Kenya

NOT YET RECRUITING

KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital

Siaya, Siaya County, 40600, Kenya

NOT YET RECRUITING

KEMRI-Walter-Reed Kericho

Kericho, 20200, Kenya

NOT YET RECRUITING

Kombewa Clinical Research Centre

Kisumu, 1932, Kenya

NOT YET RECRUITING

KEMRI Kondele Children Hospital, Kisumu

Kisumu, 40100, Kenya

RECRUITING

Ahero Clinical Trials Unit

Kisumu, 40101, Kenya

RECRUITING

American University of Beirut Medical Centre

Hamra, 11-0236, Lebanon

RECRUITING

Chronic Care Center

Hazmiyeh, 21211, Lebanon

RECRUITING

Hospital Nini

Tripoli, 1434, Lebanon

RECRUITING

University of Nigeria Teaching Hospital (UNTH)

Ituku-Ozalla, Enugu State, 400001, Nigeria

NOT YET RECRUITING

University College Hospital Paediatric Haematology and Oncology Unit, Ibadan

Ibadan, Oyo State, 0000, Nigeria

NOT YET RECRUITING

University of Abuja Teaching Hospital, Gwagwalada, Abuja

Abuja, 228, Nigeria

NOT YET RECRUITING

Barau Dikko Teaching Hospital, Kaduna

Kaduna, 800212, Nigeria

NOT YET RECRUITING

Aminu Kano Teaching Hospital (AKTH)

Kano, 3452, Nigeria

NOT YET RECRUITING

Lagos University Teaching Hospital, Lagos

Lagos, 102215, Nigeria

NOT YET RECRUITING

Sultan Qaboos University Hospital

Muscat, Sultanet of Oman/Muscat/Al Khoud, 123, Oman

RECRUITING

Prince Mohammad Bin Naser Hospital

Jizan, 82943, Saudi Arabia

NOT YET RECRUITING

King Khalid University Hospital

Riyadh, 12372, Saudi Arabia

RECRUITING

Hospital Universitario de Cruces

Barakaldo, 48903, Spain

ACTIVE NOT RECRUITING

Hospital Vall d'Hebron

Barcelona, 08035, Spain

RECRUITING

Hospital Universitario La Paz

Madrid, 28046, Spain

RECRUITING

Baskent Universitesi Adana

Adana, 01250, Turkey (Türkiye)

NOT YET RECRUITING

Başkent Üniversitesi Adana-Hematoloji

Adana, 01250, Turkey (Türkiye)

RECRUITING

Hacettepe University Hematology

Ankara, 06230, Turkey (Türkiye)

NOT YET RECRUITING

Hacettepe Üniversitesi Hastanesi- Hematoloji

Ankara, 06230, Turkey (Türkiye)

NOT YET RECRUITING

Mersin University Medical Faculty Pediatric Hematology

Mersin, 33110, Turkey (Türkiye)

NOT YET RECRUITING

Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji

Mersin, 33110, Turkey (Türkiye)

RECRUITING

Guy's Hospital - Haematology

London, SE1 9RT, United Kingdom

RECRUITING

Guy's Hospital

London, SE1 9RT, United Kingdom

NOT YET RECRUITING

King's College Hospital - Paediatric Research

London, SE5 9RS, United Kingdom

RECRUITING

Kings College Hospital - Haematology

London, SE5 9RS, United Kingdom

RECRUITING

Imperial College London

London, W12 0NN, United Kingdom

RECRUITING

Manchester Royal Infirmary

Manchester, M13 9WL, United Kingdom

RECRUITING

Royal Hallamshire Hospital

Sheffield, S10 2JF, United Kingdom

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell; Thalassemia

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Transparency (dept. 2834)

  Novo Nordisk A/S

  STUDY DIRECTOR

Central Study Contacts

Novo Nordisk

CONTACT

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

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 20, 2024
• First Posted: September 24, 2024
• Study Start: January 10, 2025
• Primary Completion (Estimated): December 30, 2030
• Study Completion (Estimated): December 30, 2030
• Last Updated: March 24, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

GB (7); FR (4); NG (6); US (39); TU (6); LE (3); IN (6); OM (1); IT (3); KE (6); CA (4); GH (2); DE (2); SA (2); GR (4); EG (5); ES (3)