This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
408
Etavopivat will be administered orally.
Placebo matching Etavopivat will be administered orally.
Uni of Alabama at Birmingham
Birmingham, Alabama, United States
RECRUITINGUniver South Alabama Ped/Onc
Mobile, Alabama, United States
RECRUITINGPhoenix Children's Hsptl
Phoenix, Arizona, United States
RECRUITINGArkansas Children's Hospital
Little Rock, Arkansas, United States
Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact
Measured as Count of events.
Time frame: Baseline (week 0) to week 52
Change in Haemoglobin (Hb) greater than 1 grams per decilitre (g/dL)
Measured in Count of participants.
Time frame: Baseline (week 0) to week 24
Time to onset of first adjudicated Vaso-occlusive crisis (VOC)
Measured as time in days.
Time frame: Baseline (week 0) to week 52
Change in standardised T-score on the Patient-Reported Outcomes Measurement Information System (PROMIS) Fatigue 7a Scale
Measured in T-score.
Time frame: Baseline (week 0) to week 52
Change in haemoglobin (Hb)
Measured in grams per decilitre (g/dL).
Time frame: Baseline (week 0) to week 52
Change in lactate dehydrogenase (LDH)
Measured in Units per litre (U/L).
Time frame: Baseline (week 0) to week 52
Change in absolute reticulocyte count
Measured as Cells x10\^9/L.
Time frame: Baseline (week 0) to week 52
Change in indirect bilirubin
Measured in milligrams per decilitre (mg/dL).
Time frame: Baseline (week 0) to week 52
Change in distance travelled during the 6-minute walking test (6MWT)
Measured in Meters.
Time frame: Baseline (week 0) to week 52
Participants achieving the threshold for clinically meaningful change (yes/no) in PROMIS fatigue scale 7a
Measured as Count of participants.
Time frame: Baseline (week 0) to week 52
Participants achieving the threshold for clinically meaningful change (yes/no) in 6MWT
Measured as Count of participants.
Time frame: Baseline (week 0) to week 52
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Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
RECRUITINGUCLA Health
Los Angeles, California, United States
RECRUITINGValley Children's Hospital
Madera, California, United States
RECRUITINGUniversity Of California Irvine
Orange, California, United States
COMPLETEDStanford University_Palo Alto
Palo Alto, California, United States
RECRUITINGHarbor-UCLA Medical Center
Torrance, California, United States
RECRUITING...and 160 more locations