Comparing 5 approaches for sickle cell disease
Official title: A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia
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
- 5 groups
- Sites in Vancouver, Toronto and 1 more city
- Recruiting
Interventions
- Medication
Etavopivat A
Participants will receive an oral dose of Etavopivat A.
- Medication
Etavopivat B
Participants will receive an oral dose of Etavopivat B.
- Medication
Etavopivat C
Participants will receive an oral dose of Etavopivat C.
Canadian Sites (4)
4 listed, none recruiting
- Not currently recruiting
St Pauls Hospital
Vancouver, British Columbia
- Not currently recruiting
The Hospital for Sick Children
Toronto, Ontario
- Not currently recruiting
University Health Network - Toronto General Hospital
Toronto, Ontario
- Not currently recruiting
CHU Ste-Justine
Montreal, Quebec
Eligibility Criteria
See who this study is looking for13 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
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 investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →.
- +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 consentInformed consentThe process of being told what taking part involves, then choosing freely.Read more → 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 investigatorPrincipal investigatorThe doctor or researcher responsible for running the study at a site.Read more →'s opinion might jeopardise participant's safety or compliance with the protocolProtocolThe detailed plan a study must follow.Read more →.
- −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.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 3Phase 3A large study comparing a treatment against the current standard.Read more → — a large study comparing this against the current standard, usually across many hospitals.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 5 groups in this study.
Groups A, B, C and D receive one or more of: Etavopivat A and Etavopivat C.
Registry label: A: Participants greater than or equal to (≥) 12 years old with sickle cell disease · B: Participants ≥ 12 years old with sickle cell disease transfusion dependent · C: Participants ≥ 12 years old with transfusion-dependent thalassaemia · D: Participants ≥ 12 years old with non-transfusion dependent thalassaemia
Group E receives one or more of: Etavopivat A, Etavopivat B and Etavopivat C.
Registry label: E: Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Groups are assigned by the study team using set rules, rather than by chance.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists a minimum age of 2 years, with no upper limit given.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 480 people.
The study is currently expected to finish around December 2030.
The main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more → (week 0 of FLORAL) up to end of study (up to week 316).
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Number of treatment emergent adverse eventsAdverse eventAny medical problem that happens during a study, whether or not the treatment caused it.Read more → (TEAEs), reported for each indication and age group separately — measured over BaselineBaselineYour starting measurements, taken before treatment begins.Read more → (week 0 of FLORAL) up to end of study (up to week 316).
Number of adverse reactions, reported for each indication and age group separately — measured over Baseline (week 0 of FLORAL) up to end of study (up to week 316).
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06609226 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About Etavopivat ADrug
Participants will receive an oral dose of Etavopivat A.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Etavopivat BDrug
Participants will receive an oral dose of Etavopivat B.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Etavopivat CDrug
Participants will receive an oral dose of Etavopivat C.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 2 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
Groups are assigned by the study team using set rules, rather than by chance.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 2 years, with no upper limit given.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: BaselineBaselineYour starting measurements, taken before treatment begins.Read more → (week 0 of FLORAL) up to end of study (up to week 316).
The study as a whole is currently expected to finish around 2030-12-30.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 480 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 7 locations, including: Vancouver, British Columbia, Canada; Toronto, Ontario, Canada; Montreal, Quebec, Canada; Brooklyn, New York, United States; New York, New York, United States; The Bronx, New York, United States, and 1 more.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
Other Sites (7)
NYC Health+Hospitals
Brooklyn, New York, United States
Columbia University Medical Center_New York_0
New York, New York, United States
Columbia University Medical Center_New York_0
New York, New York, United States
Weill Cornell Med Coll-NYPH
New York, New York, United States
Jacobi Medical Center
The Bronx, New York, United States
Montefiore Medical Center
The Bronx, New York, United States
Mary Bridge Children's Health
Tacoma, Washington, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.