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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
480
Participants will receive an oral dose of Etavopivat A.
Participants will receive an oral dose of Etavopivat B.
Participants will receive an oral dose of Etavopivat C.
Univ of Alabama Birmingham
Birmingham, Alabama, United States
ACTIVE_NOT_RECRUITINGPhoenix Children's Hsptl
Phoenix, Arizona, United States
RECRUITINGChildren's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
ACTIVE_NOT_RECRUITINGChildren's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately
Measured as number of events.
Time frame: Baseline (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 as number of adverse reactions.
Time frame: Baseline (week 0 of FLORAL) up to end of study (up to week 316)
Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately
Measured as count.
Time frame: Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)
Change in VOCs, reported for each age group separately
Measured as count.
Time frame: Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
Change in hemoglobin (Hb) concentration, reported for each age group separately
Measured as grams per deciliter (g/dL).
Time frame: Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
Annualised number of hospitalisations, reported for each age group separately
Measured as count.
Time frame: Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)
Average length of stay of hospitalisations, reported for each age group separately
Measured as days.
Time frame: Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)
Change in Hb concentration
Measured as g/dL.
Time frame: Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
Number of red blood cell (RBC) units transfused, reported for each indication separately
Measured as units.
Time frame: Baseline (week 0 of FLORAL) up to end of treatment (up to week 312)
Change in RBC units transfused, reported for each indication separately
Measured as units.
Time frame: Baseline (of parent study [i.e., the previous etavopivat study that a participant is rolling over from]) up to end of treatment (up to week 312)
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UCSF Oakland Benioff ChildHosp
Oakland, California, United States
ACTIVE_NOT_RECRUITINGUCSF Oakland Benioff ChildHosp
Oakland, California, United States
RECRUITINGChildren's Hosp Of Orange
Orange, California, United States
COMPLETEDUniversity Of California Irvine
Orange, California, United States
RECRUITINGUniversity of Connecticut
Farmington, Connecticut, United States
ACTIVE_NOT_RECRUITINGChildren's National Medical Center
Washington D.C., District of Columbia, United States
COMPLETED...and 93 more locations