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RECRUITINGPhase 3INTERVENTIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 Should NOT Join This Trial: - 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

DRUG

Etavopivat A

Participants will receive an oral dose of Etavopivat A.

DRUG

Etavopivat B

Participants will receive an oral dose of Etavopivat B.

DRUG

Etavopivat C

Participants will receive an oral dose of Etavopivat C.

Locations (20)

Univ of Alabama Birmingham
Birmingham, Alabama, United States
Phoenix Children's Hsptl
Phoenix, Arizona, United States
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
UCSF Oakland Benioff ChildHosp
Oakland, California, United States
UCSF Oakland Benioff ChildHosp
Oakland, California, United States
Children's Hosp Of Orange
Orange, California, United States
University Of California Irvine
Orange, California, United States
University of Connecticut
Farmington, Connecticut, United States
Children's National Medical Center
Washington D.C., District of Columbia, United States
Foundation for Sickle Cell Disease Research
Hollywood, Florida, United States
Univ of Miami/SCCC
Miami, Florida, United States
Emory University School of Medicine
Atlanta, Georgia, United States
Children's Healthcare Atlanta
Atlanta, Georgia, United States
Center for Blood Disorders Augusta University
Augusta, Georgia, United States
Univer Of Illinois at Chicago
Chicago, Illinois, United States
Children's Hosp-New Orleans
New Orleans, Louisiana, United States
Boston Medical Center
Boston, Massachusetts, United States
Washington University-St.Louis
St Louis, Missouri, United States
NYC Health+Hospitals
Brooklyn, New York, United States