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Clinical Trials in the UK / NCT06609226
Recruiting Phase 3

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

NCT06609226 · tracked via the Priya Life Science UK tracker
Sponsor
Novo Nordisk A/S
Phase
Phase 3
Started
2025-01-10
Last updated
2026-09-18

Condition(s) studied

Sickle Cell DiseaseThalassemia

Investigational drug(s) / intervention(s)

Etavopivat AEtavopivat BEtavopivat C

Etavopivat A: Participants will receive an oral dose of Etavopivat A.

Etavopivat B: Participants will receive an oral dose of Etavopivat B.

Etavopivat C: Participants will receive an oral dose of Etavopivat C.

Study 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. 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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (107)

FacilityCityRegionStatus
Univ of Alabama Birmingham Birmingham Alabama Active Not Recruiting
Phoenix Children's Hsptl Phoenix Arizona Active Not Recruiting
Children's Hospital Los Angeles - Endocrinology Los Angeles California Active Not Recruiting
Children's Hospital Los Angeles - Endocrinology Los Angeles California Recruiting
UCSF Oakland Benioff ChildHosp Oakland California Active Not Recruiting
UCSF Oakland Benioff ChildHosp Oakland California Recruiting
Children's Hosp Of Orange Orange California Completed
University Of California Irvine Orange California Active Not Recruiting
University of Connecticut Farmington Connecticut Active Not Recruiting
Children's National Medical Center Washington D.C. District of Columbia Completed
Foundation for Sickle Cell Disease Research Hollywood Florida Active Not Recruiting
Univ of Miami/SCCC Miami Florida Completed
Emory University School of Medicine Atlanta Georgia Active Not Recruiting
Children's Healthcare Atlanta Atlanta Georgia Recruiting
Center for Blood Disorders Augusta University Augusta Georgia Active Not Recruiting
Univer Of Illinois at Chicago Chicago Illinois Recruiting
Children's Hosp-New Orleans New Orleans Louisiana Completed
Boston Medical Center Boston Massachusetts Active Not Recruiting
Washington University-St.Louis St Louis Missouri Active Not Recruiting
NYC Health+Hospitals Brooklyn New York Active Not Recruiting
Columbia University Medical Center_New York_0 New York New York Recruiting
Columbia University Medical Center_New York_0 New York New York Not Yet Recruiting
Weill Cornell Med Coll-NYPH New York New York Active Not Recruiting
Jacobi Medical Center The Bronx New York Active Not Recruiting
Montefiore Medical Center The Bronx New York Active Not Recruiting
Atrium Levine Children's/Atrium Health Charlotte North Carolina Not Yet Recruiting
Duke University_Durham Durham North Carolina Recruiting
East Carolina Univ-Greenville Greenville North Carolina Active Not Recruiting
East Carolina University_Greenville Greenville North Carolina Completed
Atrium Health-Wake Forest Bapt Winston-Salem North Carolina Active Not Recruiting
Cincinnati Child's Hsp Med Ctr Cincinnati Ohio Active Not Recruiting
Neuro-Behavioral Clinical Research North Canton Ohio Active Not Recruiting
Medical University Of South Carolina_Charleston Charleston South Carolina Active Not Recruiting
Methodist University Hospital Memphis Tennessee Active Not Recruiting
Texas Children's Hospital_Houston Houston Texas Recruiting
UT Health University of Texas Houston Texas Recruiting
Virginia Comm Univ Medical Ctr Richmond Virginia Completed
Mary Bridge Children's Health Tacoma Washington Active Not Recruiting
Versiti, CCBD_Milwaukee Milwaukee Wisconsin Recruiting
St Pauls Hospital Vancouver British Columbia Active Not Recruiting

+ 67 more sites — see the full list on the official registry below.

More Novo Nordisk A/S trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06609226 on ClinicalTrials.gov ↗ ← All trials in the UK