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Long-term treatment with Etavopivat for sickle cell disease and thalassemia

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

Phase 3 Interventional Novo Nordisk A/S · NCT06609226

This study is testing how safe and effective a new medication called Etavopivat is for people with sickle cell disease and thalassemia who have already seen benefits from it.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment480 (estimated)
Ages2 Years and up
SexAll
SponsorNovo Nordisk A/S Industry-sponsored
Drugs / interventionscrizanlizumab
Locations103 sites (Birmingham, Alabama and 102 other locations)
Trial IDNCT06609226 on ClinicalTrials.gov

What this trial studies

This study evaluates the long-term safety and efficacy of Etavopivat, a new medication for treating blood disorders such as sickle cell disease and thalassemia. Participants must have previously benefited from Etavopivat in a parent study and will be monitored over a period of up to 264 weeks. The study aims to determine how well the treatment works and its safety profile in individuals with these inherited blood disorders.

Who should consider this trial

Good fit: Ideal candidates are individuals who have previously participated in an Etavopivat parent study and have shown clinical benefit from the treatment.

Not a fit: Patients who have not participated in the parent study or have not derived clinical benefit from Etavopivat will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of sickle cell disease and thalassemia, enhancing patients' quality of life.

How similar studies have performed: Other studies involving similar treatments for sickle cell disease and thalassemia have shown promise, but this specific approach with Etavopivat is relatively novel.

Eligibility criteria

Show full inclusion / exclusion criteria 
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 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 Criteria:

* 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.

Where this trial is running

Birmingham, Alabama and 102 other locations

+53 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Sickle Cell DiseaseThalassemia
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.