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Taking people in SpainPhase 3

Long-term etavopivat treatment for sickle cell disease or thalassaemia

Tests long-term safety of etavopivat in children and adults with blood disorders.

Key facts

Who runs it
Novo Nordisk A/S
Where
Barakaldo, Barcelona, and Madrid, SpainSee the 3 sitesAlso in 16 other countries, 98 sites
Trial code
Registry title
A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
Status
Recruiting
Phase
Phase 3
People taking part
480 people, planned
Started
10 Jan 2025
Expected to end
30 Dec 2030
Type of study
Interventional, 5 groups
Treatments tested
  • Etavopivat A
  • Etavopivat B
  • Etavopivat C
Record updated
18 September 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for people with sickle cell disease or thalassaemia who took part in an etavopivat study and benefited from the treatment.

Age
From 2 years old.
Sex
All sexes can join.
Disease stage
Sickle cell disease (SCD) or thalassaemia.
Earlier treatment
Must still be in an etavopivat parent study and have completed at least a treatment period of it. The investigator must judge that etavopivat helped them. Anyone whose dose was reduced or paused must be able to take the full dose again before joining. People on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) can join if their dose was stable in the parent study.

Main reasons for not taking part

  • Left an etavopivat study or stopped treatment for good
  • Permanent dose reduction (more than 28 days) or treatment still paused
  • Another disorder that the investigator thinks could risk safety or following the study plan
  • Use of haemoglobin S (HbS) polymerisation inhibitors or experimental selectin antagonists
  • Strong CYP3A4 inducers within 2 weeks of the transfer visit, or taking part in another clinical study

The registry criteria, word for word

Show all 13 criteriaShow less

Who can take part

  • 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 take part

  • Use of any of the following within the timeframes prior to the transfer visit as stated:
  • 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
  • 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
  • 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
  • Use of haemoglobin S (HbS) polymerisation inhibitors within participation of the parent study or anticipated need for this agent during this study
  • 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 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
  • 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

Where in Spain

Also in 16 other countries, 98 sites

  • Hospital Universitario de CrucesBarakaldo, SpainTaking people
  • Hospital Universitari Vall d'HebronBarcelona, SpainTaking people
  • Hospital Universitario La PazMadrid, SpainTaking people

About the trial

What the trial wants to find out

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.

The 5 groups in the trial

  • Participants greater than or equal to (≥) 12 years old with sickle cell diseaseTreatment being tested

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

    Treatment: Etavopivat A and Etavopivat C

  • Participants ≥ 12 years old with sickle cell disease transfusion dependentTreatment being tested

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

    Treatment: Etavopivat A and Etavopivat C

  • Participants ≥ 12 years old with transfusion-dependent thalassaemiaTreatment being tested

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

    Treatment: Etavopivat A and Etavopivat C

  • Participants ≥ 12 years old with non-transfusion dependent thalassaemiaTreatment being tested

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

    Treatment: Etavopivat A and Etavopivat C

  • Participants ≥ 2 years to less than (<) 12 years old with sickle cell diseaseTreatment being tested

    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.

    Treatment: Etavopivat A, Etavopivat B, and Etavopivat C

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record → Contacts and Locations

Print a page for my doctor

Data and sources

Registry record
Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
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
Plain-language title
Long-term etavopivat treatment for sickle cell disease or thalassaemiaGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 12 November 2026.
Record updated
18 September 2026