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Sickle Cell DiseasePhase 3FLORALNCT06609226

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

A Phase 3 study testing Etavopivat A, Etavopivat B and Etavopivat C in people with Sickle Cell Disease and Thalassemia, run by Novo Nordisk A/S at 107 sites in 17 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Sickle Cell Disease, Thalassemia
What is being tested?
Etavopivat A, Etavopivat B and Etavopivat C
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
480 participants
Where
107 sitesin 17 countries
Study dates
10 Jan 2025to 30 Dec 2030
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 9Show fewer
  • 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.

Trial locations and contacts

107 sites in 17 countries. Contact the study team or a site near you to ask about taking part.

Novo Nordisk contact

(+1) 866-867-7178

clinicaltrials@novonordisk.com

107 sites in 17 countries

United States39 sites, 11 recruiting

  • Emory University School of Medicine

    Atlanta, Georgia, 30303

    Active, not recruiting

  • Children's Healthcare Atlanta

    Atlanta, Georgia, 30329

    Recruiting

  • Center for Blood Disorders Augusta University

    Augusta, Georgia, 30912

    Active, not recruiting

  • Univ of Alabama Birmingham

    Birmingham, Alabama, 35233

    Active, not recruiting

  • Boston Medical Center

    Boston, Massachusetts, 02118

    Active, not recruiting

  • NYC Health+Hospitals

    Brooklyn, New York, 11203

    Active, not recruiting

  • Medical University Of South Carolina_Charleston

    Charleston, South Carolina, 29425

    Active, not recruiting

  • Atrium Levine Children's/Atrium Health

    Charlotte, North Carolina, 28204

    Not yet recruiting

  • Univer Of Illinois at Chicago

    Chicago, Illinois, 60612

    Recruiting

  • Cincinnati Child's Hsp Med Ctr

    Cincinnati, Ohio, 45229

    Active, not recruiting

  • Duke University_Durham

    Durham, North Carolina, 27710

    Recruiting

  • University of Connecticut

    Farmington, Connecticut, 06030

    Active, not recruiting

  • East Carolina Univ-Greenville

    Greenville, North Carolina, 27834

    Active, not recruiting

  • East Carolina University_Greenville

    Greenville, North Carolina, 27834

    Completed

  • Foundation for Sickle Cell Disease Research

    Hollywood, Florida, 33023

    Active, not recruiting

  • Texas Children's Hospital_Houston

    Houston, Texas, 77030

    Recruiting

  • UT Health University of Texas

    Houston, Texas, 77030

    Recruiting

  • Children's Hospital Los Angeles - Endocrinology

    Los Angeles, California, 90027

    Active, not recruiting

  • Children's Hospital Los Angeles - Endocrinology

    Los Angeles, California, 90027

    Recruiting

  • Methodist University Hospital

    Memphis, Tennessee, 38104

    Active, not recruiting

  • Univ of Miami/SCCC

    Miami, Florida, 33136

    Completed

  • Versiti, CCBD_Milwaukee

    Milwaukee, Wisconsin, 53226

    Recruiting

  • Children's Hosp-New Orleans

    New Orleans, Louisiana, 70118

    Completed

  • Columbia University Medical Center_New York_0

    New York, New York, 10032

    Recruiting

  • Columbia University Medical Center_New York_0

    New York, New York, 10032

    Not yet recruiting

  • Weill Cornell Med Coll-NYPH

    New York, New York, 10065

    Active, not recruiting

  • Neuro-Behavioral Clinical Research

    North Canton, Ohio, 44720

    Active, not recruiting

  • UCSF Oakland Benioff ChildHosp

    Oakland, California, 94609

    Active, not recruiting

  • UCSF Oakland Benioff ChildHosp

    Oakland, California, 94609

    Recruiting

  • Children's Hosp Of Orange

    Orange, California, 92868

    Completed

  • University Of California Irvine

    Orange, California, 92868

    Active, not recruiting

  • Phoenix Children's Hsptl

    Phoenix, Arizona, 85016

    Active, not recruiting

  • Virginia Comm Univ Medical Ctr

    Richmond, Virginia, 23298

    Completed

  • Washington University-St.Louis

    St Louis, Missouri, 63110

    Active, not recruiting

  • Mary Bridge Children's Health

    Tacoma, Washington, 98405

    Active, not recruiting

  • Jacobi Medical Center

    The Bronx, New York, 10461

    Active, not recruiting

  • Montefiore Medical Center

    The Bronx, New York, 10467

    Active, not recruiting

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010

    Completed

  • Atrium Health-Wake Forest Bapt

    Winston-Salem, North Carolina, 27157

    Active, not recruiting

Other trials in United States

United Kingdom8 sites, 8 recruiting

  • Evelina London Children's Hospital - Haemophilia

    London, SE1 7EH

    Not yet recruiting

  • Guy's Hospital - Haematology

    London, SE1 9RT

    Recruiting

  • Guy's Hospital

    London, SE1 9RT

    Not yet recruiting

  • King's College Hospital - Paediatric Research

    London, SE5 9RS

    Recruiting

  • Kings College Hospital - Haematology

    London, SE5 9RS

    Recruiting

  • Imperial College London

    London, W12 0NN

    Recruiting

  • Manchester Royal Infirmary

    Manchester, M13 9WL

    Recruiting

  • Royal Hallamshire Hospital

    Sheffield, S10 2JF

    Recruiting

Other trials in United Kingdom

Kenya7 sites, 7 recruiting

  • KEMRI-Walter-Reed Kericho

    Kericho, 20200

    Not yet recruiting

  • Kombewa Clinical Research Centre

    Kisumu, 1932

    Recruiting

  • Kombewa Clinical Research Centre

    Kisumu, 1932

    Not yet recruiting

  • KEMRI Kondele Children Hospital, Kisumu

    Kisumu, 40100

    Recruiting

  • Ahero Clinical Trials Unit

    Kisumu, 40101

    Recruiting

  • Gertrude's Children's Hospital

    Nairobi, Nairobi County, 00509

    Recruiting

  • KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital

    Siaya, Siaya County, 40600

    Recruiting

Other trials in Kenya

Turkey (Türkiye)7 sites, 7 recruiting

  • Baskent Universitesi Adana

    Adana, 01250

    Not yet recruiting

  • Başkent Üniversitesi Adana-Hematoloji

    Adana, 01250

    Recruiting

  • Hacettepe University Hematology

    Ankara, 06230

    Not yet recruiting

  • Hacettepe Üniversitesi Hastanesi- Hematoloji

    Ankara, 06230

    Not yet recruiting

  • Mersin University Medical Faculty Pediatric Hematology

    Mersin, 33110

    Not yet recruiting

  • Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji

    Mersin, 33110

    Recruiting

  • VM Medical Park Mersin Hastanesi- Hematoloji

    Mersin, 33200

    Not yet recruiting

Other trials in Turkey (Türkiye)

Egypt6 sites, 6 recruiting

  • Alexandria University Hospital

    Alexandria, Egypt, 21131

    Recruiting

  • Alexandria University Hospital

    Alexandria, Egypt, 21131

    Not yet recruiting

  • Zagazig University Hospital

    Alsharkia, Egypt, 44519

    Recruiting

  • Faculty of Medicine Ain Shams Medical Research Institute (MASRI)

    Cairo, 1181

    Recruiting

  • Cairo University

    Cairo, Egypt, 11956

    Recruiting

  • Abu El-Reesh El-Mounira Children University Hospital

    Cairo, Egypt, 4241317

    Recruiting

Other trials in Egypt

India6 sites, 6 recruiting

  • Victoria Hospital, Bangalore

    Bangalore, 560002

    Not yet recruiting

  • Nirmal Hospital Pvt. Ltd.

    Gujarat, 395002

    Not yet recruiting

  • Suretech Hospital and Research Centre Ltd.

    Maharashtra, 440012

    Not yet recruiting

  • K.J Somaiya Hospital and Research Centre

    Mumbai, Maharashtra, 400022

    Not yet recruiting

  • All India Institute of Medical Sciences_Delhi

    New Delhi, 110029

    Not yet recruiting

  • All India Institute of Medical Sciences (AIIMS), Raipur

    Raipur, Chhattisgarh, 492099

    Not yet recruiting

Other trials in India

Nigeria6 sites, 6 recruiting

  • University of Abuja Teaching Hospital, Gwagwalada, Abuja

    Abuja, 228

    Not yet recruiting

  • University College Hospital Paediatric Haematology and Oncology Unit, Ibadan

    Ibadan, Oyo State, 0000

    Not yet recruiting

  • University of Nigeria Teaching Hospital (UNTH)

    Ituku-Ozalla, Enugu State, 400001

    Not yet recruiting

  • Barau Dikko Teaching Hospital, Kaduna

    Kaduna, 800212

    Not yet recruiting

  • Aminu Kano Teaching Hospital (AKTH)

    Kano, 3452

    Not yet recruiting

  • Lagos University Teaching Hospital, Lagos

    Lagos, 102215

    Not yet recruiting

Other trials in Nigeria

Canada4 sites

  • CHU Ste-Justine

    Montreal, Quebec, H3T 1C5

    Completed

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1X8

    Active, not recruiting

  • University Health Network - Toronto General Hospital

    Toronto, Ontario, M5G 2C4

    Active, not recruiting

  • St Pauls Hospital

    Vancouver, British Columbia, V6Z1Y6

    Active, not recruiting

Other trials in Canada

France4 sites, 4 recruiting

  • Ap-Hp-Hopital Henri Mondor

    Créteil, 94000

    Recruiting

  • Hospices Civils de Lyon-Hopital Edouard Herriot

    Lyon, 69437

    Recruiting

  • Ap-Hp-Hopital Robert Debre

    Paris, 75019

    Recruiting

  • Ap-Hp-Hopital Robert Debre

    Paris, 75019

    Not yet recruiting

Other trials in France

Greece4 sites, 4 recruiting

  • Hippokration Hospital

    Athens, 11527

    Recruiting

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

    Larissa, Thessaly, 41221

    Recruiting

  • General University Hospital of Patras

    Pátrai, 26504

    Recruiting

  • 'Ippokrateio' General Hospital of Thessaloniki

    Thessaloniki, 54642

    Recruiting

Other trials in Greece

Italy3 sites, 2 recruiting

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

    Orbassano, Torino, 10043

    Recruiting

  • Azienda Ospedale Universita Padova

    Padova, 35128

    Recruiting

  • Fondazione IRCCS Policlinico San Matteo

    Pavia, 27100

    Completed

Other trials in Italy

Lebanon3 sites, 3 recruiting

  • American University of Beirut Medical Centre

    Hamra, 11-0236

    Recruiting

  • Chronic Care Center

    Hazmiyeh, 21211

    Recruiting

  • Hospital Nini

    Tripoli, 1434

    Recruiting

Other trials in Lebanon

Spain3 sites, 2 recruiting

  • Hospital Universitario de Cruces

    Barakaldo, 48903

    Active, not recruiting

  • Hospital Vall d'Hebron

    Barcelona, 08035

    Recruiting

  • Hospital Universitario La Paz

    Madrid, 28046

    Recruiting

Other trials in Spain

Germany2 sites, 1 recruiting

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

    Berlin, 13353

    Not yet recruiting

  • Universitätsklinikum Freiburg - Kinder- und Jugendklinik

    Freiburg im Breisgau, 79106

    Completed

Other trials in Germany

Ghana2 sites, 2 recruiting

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

    Accra, 0

    Recruiting

  • Kintampo Health Research Centre (KHRC)

    Kintampo, Bono East, 27WF+98M

    Recruiting

Other trials in Ghana

Saudi Arabia2 sites, 2 recruiting

  • Prince Mohammad Bin Naser Hospital

    Jizan, 82943

    Not yet recruiting

  • King Khalid University Hospital

    Riyadh, 12372

    Recruiting

Other trials in Saudi Arabia

Oman1 site, 1 recruiting

  • Sultan Qaboos University Hospital

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

    Recruiting

Other trials in Oman

About this study

Official title: An Open-label, Multi-centre, Rollover Study to Characterise Long-term Safety and Efficacy of Etavopivat in Adults, Adolescents and Children Who Have Sickle Cell Disease or Thalassaemia and Have Completed a Treatment Period in an Etavopivat Study

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.

Being tested
Etavopivat A, Etavopivat B, Etavopivat C
Medical subject headings
Anemia, Sickle Cell, Thalassemia

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Etavopivat ADrug

    Participants will receive an oral dose of Etavopivat A.

  • Etavopivat BDrug

    Participants will receive an oral dose of Etavopivat B.

  • Etavopivat CDrug

    Participants will receive an oral dose of Etavopivat C.

Study groups in NCT06609226
Study groupRoleWhat they receive
Participants greater than or equal to (≥) 12 years old with sickle cell diseaseExperimentalParticipants will receive an oral dose of Etavopivat A or C.
Receives: Drug: Etavopivat A; Drug: Etavopivat C
Participants ≥ 12 years old with sickle cell disease transfusion dependentExperimentalParticipants will receive an oral dose of Etavopivat A or C.
Receives: Drug: Etavopivat A; Drug: Etavopivat C
Participants ≥ 12 years old with transfusion-dependent thalassaemiaExperimentalParticipants will receive an oral dose of Etavopivat A or C.
Receives: Drug: Etavopivat A; Drug: Etavopivat C
Participants ≥ 12 years old with non-transfusion dependent thalassaemiaExperimentalParticipants will receive an oral dose of Etavopivat A or C.
Receives: Drug: Etavopivat A; Drug: Etavopivat C
Participants ≥ 2 years to less than (<) 12 years old with sickle cell diseaseExperimentalParticipants ≥ 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.
Receives: Drug: Etavopivat A; Drug: Etavopivat B; Drug: Etavopivat C

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    Measured as number of events.

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

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

    Measured as number of adverse reactions.

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

Secondary outcomes (8)

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

    Measured as count.

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

  2. Change in VOCs, reported for each age group separately

    Measured as count.

    Measured: 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)

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

    Measured as grams per deciliter (g/dL).

    Measured: 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)

  4. Annualised number of hospitalisations, reported for each age group separately

    Measured as count.

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

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

    Measured as days.

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

  6. Change in Hb concentration

    Measured as g/dL.

    Measured: 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)

  7. Number of red blood cell (RBC) units transfused, reported for each indication separately

    Measured as units.

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

  8. Change in RBC units transfused, reported for each indication separately

    Measured as units.

    Measured: 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)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted24 Sep 2024
  2. Study start10 Jan 2025
  3. Primary completion30 Dec 2030 (estimated)
  4. Study completion30 Dec 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
480 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06609226
Sponsor's protocol ID
NN7535-7822
Other IDs
U1111-1301-8130, 2024-510805-27
Acronym
FLORAL
Status last verified
Sep 2026
First submitted
20 Sep 2024
Last updated
18 Sep 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Etavopivat A, Etavopivat B and Etavopivat C in people with Sickle Cell Disease and Thalassemia, run by Novo Nordisk A/S at 107 sites in 17 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 107 sites in United States, United Kingdom, Kenya, Turkey (Türkiye), Egypt and 12 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Novo Nordisk ((+1) 866-867-7178, clinicaltrials@novonordisk.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately (time frame: Baseline (week 0 of FLORAL) up to end of study (up to week 316)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06609226.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2026.

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