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Long-term Etavopivat Treatment for Sickle Cell Disease or Thalassemia

This research study is looking at the long-term safety and effectiveness of Etavopivat A, Etavopivat B, or Etavopivat C in people with sickle cell disease or thalassemia. These are inherited blood disorders that affect hemoglobin (the protein that carries oxygen in your body). You can join this study if you are already participating in another Etavopivat study for sickle cell disease or thalassemia, have completed a treatment period in that study, and your doctor believes you have benefited from Etavopivat. The study aims to see how many side effects occur and how well the treatment works over a long period, up to 316 weeks. The study is currently unclear about its recruitment status and plans to enroll 480 participants.

Study design
This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 480 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for treatment-emergent adverse events and adverse reactions from baseline (week 0) up to the end of the study (up to week 316).

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06609226

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

Recruiting
PHASE3Ages 2+InterventionalTreatment
Novo Nordisk A/S
~480 participants
Updated 2026-08-13 on ClinicalTrials.gov
What's tested:Etavopivat AEtavopivat BEtavopivat C

At a glance

Recruiting sites
44 of 106 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately
Measured over Baseline (week 0 of FLORAL) up to end of study (up to week 316)
+1 more outcome measured

Conditions

Sickle Cell Disease
Thalassemia

Explore related trials

Sickle Cell DiseaseThalassemia

Where it's being run

106 sites across 47 states
United Kingdom8
Turkey (Türkiye)7
California6
New York6
North Carolina5
Egypt5
Kenya5
France4

Study leadership

  • Clinical Transparency (dept. 2834) · STUDY_DIRECTOR · Novo Nordisk A/S

Who to contact

Novo Nordisk
[email protected]
(+1) 866-867-7178
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

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.

Exclusion

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.

What this trial measures

  • Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separatelyBaseline (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 separatelyBaseline (week 0 of FLORAL) up to end of study (up to week 316)

    Measured as number of adverse reactions.