Etavopivat: Etavopivat will be administered orally.
Placebo: Placebo matching Etavopivat will be administered orally.
Study summary
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.
Eligibility
Sex
ALL
Min age
12 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Male or female.
* Age 12 years or above at the time of signing the informed consent.
* Confirmed diagnosis of sickle cell disease: Documentation of sickle cell disease (SCD) genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing or screening test results from central laboratory. Molecular genotyping is not required. SCD genotype may be determined from the results of haemoglobin (Hb) electrophoresis, high-performance liquid chromatography (HPLC) or similar testing. Note that Hb electrophoresis is performed by the central laboratory at screening.
* Have 2-15 episodes of documented vaso occlusive crises (VOC) within the 12 months prior to screening. Documentation must exist in the participant's medical record prior to randomisation. Events based solely on participant recall without supporting documentation should not be counted towards eligibility.
* Hb greater than or equal to (≥) 5.0 and less than or equal to (≤) 10.0 g/dL (greater than or equal to (≥) 50 and less than or equal to (≤) 100 grams per litre \[g/L\]) at screening.
Exclusion Criteria:
* Use of voxelotor or similar agent within 28 days prior to starting study treatment or anticipated need for this agent during the study.
* Use of a selectin antagonist (e.g., crizanlizumab, monoclonal antibody or small molecule) within 28 days or 5 half-lives (whichever is longer) prior to starting study treatment or anticipated need for such agents during the study.
* Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or greater than or equal to 6 transfusion events within the previous 12 months prior to screening (i.e., an average of 1 transfusion event every 60 days).
* Participants who have received an RBC transfusion for any reason within 60 days of the screening period or 60 days of the randomisation day are only eligible if HbA (adult haemoglobin) less than 10% by Hb electrophoresis is documented prior to starting study treatment.
* Receiving or use of concomitant medications that are strong or moderate inducers of CYP3A4 (cytochrome p450 3a4) within 2 weeks of starting study treatment or anticipated need for such agents during the study.
* Use of erythropoietin or other haematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study.
* Receipt of prior cellular-based therapy (e.g., haematopoietic cell transplant, gene modification therapy).
* Hepatic dysfunction characterized by:
* Alanine aminotransferase (ALT) greater than 4.0 × upper limit of normal (ULN) or
* Direct bilirubin greater than 3.0 × ULN.
* Participants who are not taking or are unable to take antimalarial prophylaxis at the time of consent and during the study if they live in areas of endemic malaria where prophylaxis is recommended.
* Severe renal dysfunction (estimated glomerular filtration rate \[eGFR\] at screening, calculated by the central laboratory greater than 30 mL/min/1.73 m\^ 2) or on chronic dialysis.
* Travelled distance on standardized 6MWT below 100m at screening.
Primary outcome measure(s)
Number of adjudicated Vaso-occlusive crisis (VOC) events with a medical contact — Baseline (week 0) to week 52 Measured as Count of events.
Trial sites (175)
Facility
City
Region
Status
Uni of Alabama at Birmingham
Birmingham
Alabama
Recruiting
Univer South Alabama Ped/Onc
Mobile
Alabama
Recruiting
Phoenix Children's Hsptl
Phoenix
Arizona
Recruiting
Arkansas Children's Hospital
Little Rock
Arkansas
Recruiting
Children's Hospital Los Angeles - Endocrinology
Los Angeles
California
Recruiting
UCLA Health
Los Angeles
California
Completed
Valley Children's Hospital
Madera
California
Recruiting
University Of California Irvine
Orange
California
Completed
Stanford University_Palo Alto
Palo Alto
California
Recruiting
Harbor-UCLA Medical Center
Torrance
California
Recruiting
Clinical and Transl Res Center
Aurora
Colorado
Completed
Nemours/AI duPont Hosp-Chld
Wilmington
Delaware
Recruiting
Childrens National Medical Ctr
Washington D.C.
District of Columbia
Recruiting
MedStar Hlth Res Institute
Washington D.C.
District of Columbia
Recruiting
Memorial Healthcare
Hollywood
Florida
Recruiting
Children's Healthcare Atlanta
Atlanta
Georgia
Recruiting
Childrens Hospital of Chicago
Chicago
Illinois
Recruiting
Univer Of Illinois at Chicago
Chicago
Illinois
Recruiting
Children's Hosp-New Orleans
New Orleans
Louisiana
Recruiting
Boston Children's Hospital
Boston
Massachusetts
Recruiting
Boston Medical Center
Boston
Massachusetts
Recruiting
Henry Ford Hospital_Detroit
Detroit
Michigan
Completed
University of Minnesota
Minneapolis
Minnesota
Recruiting
Washington University-St.Louis
St Louis
Missouri
Recruiting
Children's Nebraska
Omaha
Nebraska
Recruiting
Cure 4 the Kids Foundation
Las Vegas
Nevada
Completed
Newark Beth Israel MC
Newark
New Jersey
Recruiting
NYC Health+Hospitals
Brooklyn
New York
Recruiting
Interfaith Medical Center
Brooklyn
New York
Recruiting
Northwell Health
Mount Kisco
New York
Completed
Cohen Children's Medical Ctr
Queens
New York
Recruiting
Jacobi Medical Center
The Bronx
New York
Recruiting
Montefiore Medical Ctr
The Bronx
New York
Recruiting
Duke Comprehen Sickle Cell
Durham
North Carolina
Recruiting
Duke University Medical Center
Durham
North Carolina
Recruiting
East Carolina Univ-Greenville
Greenville
North Carolina
Recruiting
Atrium Health-Wake Forest Bapt
Winston-Salem
North Carolina
Recruiting
Univ Hosp Cleveland Med Ctr
Cleveland
Ohio
Recruiting
Ohio State Univ Wexner Med Ctr
Columbus
Ohio
Recruiting
Univ of OK Health Sciences Ctr
Oklahoma City
Oklahoma
Recruiting
+ 135 more sites — see the full list on the official registry below.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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