RightFit
RecruitingPhase 3

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

Who can join

As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.

Age
2 Years and older
Sex
All sexes
Healthy volunteers
Not accepted
Compensation
Not stated

Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.

What to expect

No one can promise a trial will help you personally — that is what the research is trying to find out.

What participation involves

  • Time periods mentioned: 264 weeks

Only what the study's summary and detailed description on ClinicalTrials.gov say. Time periods may describe the whole study or a part of it — ask the study team what applies to you.

Show the study's own words
  • "The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country."
Compensation
Not stated
  • Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
  • Participants are monitored closely by the research team throughout the study.
  • Some participants may receive access to an investigational treatment, which is still being studied.
  • Your participation helps researchers learn whether this approach could help future patients.

About this study

In plain language

This study tests how safe etavopivat is and how well it works over a long time. It is for people with sickle cell disease or thalassaemia who have benefited from etavopivat in an earlier study. These inherited blood disorders affect haemoglobin, the protein that carries oxygen in the body.

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.

Compensation is read from the study's own description and may be missing — always confirm with the study team.

Eligibility

Who can join

  • You must still be in an earlier etavopivat study for sickle cell disease or thalassaemia and have finished at least one treatment period.
  • The study doctor must decide that etavopivat has helped you.
  • If your dose was lowered or treatment was paused, you must successfully return to the full dose before joining.
  • If you take hydroxyurea, crizanlizumab, or l-glutamine powder (Endari), the study doctor must consider your dose stable. Changes for age, weight, or medical reasons may be allowed.

Who can't join

  • You have another health problem, not related to sickle cell disease or thalassaemia, that the study doctor thinks could make the study unsafe or hard to follow.
  • You left an etavopivat study or stopped its treatment for good.
  • Your etavopivat dose has been lowered long-term (more than 28 days), or your treatment is still paused.
  • You used a medicine that blocks haemoglobin S from clumping during the earlier study, or expect to need one during this study.
  • You used an experimental selectin blocker, a medicine that blocks certain proteins involved in cells sticking together, during the earlier study, or expect to need one.
  • You used erythropoietin or other medicines that help make blood cells for more than 4 weeks in a row during the earlier study, or expect to need them as ongoing treatment.
  • You used medicines that strongly increase the activity of CYP3A4, a protein that breaks down medicines, within 2 weeks before transfer, or expect to need them during this study.
  • You are in a study other than an approved earlier etavopivat study, or plan to join another clinical study during this study.

Simplified for readability. The official criteria below are what the study team uses.

Official eligibility 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.

Study sites (39)

AL

AZ

CA

CT

DC

FL

GA

IL

LA

MA

MO

NC

NY

OH

SC

TN

TX

VA

WA

WI