NCT06609226 · Novo Nordisk A/S
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
(FLORAL)
What this study is about
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.
View original scientific description
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.
Interventions
DRUG
Etavopivat A
Participants will receive an oral dose of Etavopivat A.
DRUG
Etavopivat B
Participants will receive an oral dose of Etavopivat B.
DRUG
Etavopivat C
Participants will receive an oral dose of Etavopivat C.
Primary outcome measures
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)
Measured as number of events.
Number of adverse reactions, reported for each indication and age group separately
Time frame: Baseline (week 0 of FLORAL) up to end of study (up to week 316)
Measured as number of adverse reactions.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 rechallenged 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 during participation in the parent study (i.e., no changes to the dose except for changes to weight or age reasons).
- Have been compliant with the treatment regimen at the discretion of the investigator during participation of the parent study.
Exclusion criteria
- Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigator'
Where
- Birmingham, Alabama
- Phoenix, Arizona
- Los Angeles, California
- Oakland, California
- Orange, California
- Farmington, Connecticut
- Washington D.C., District of Columbia
- Hollywood, Florida
- Miami, Florida
- Atlanta, Georgia
- Augusta, Georgia
- Chicago, Illinois
And 18 more locations — see the full list below.
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 10, 2026 · Source of record for eligibility and locations