A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
A Global Phase 3, Randomised, Double-blind and Placebo-controlled Study Evaluating the Efficacy and Safety of Etavopivat in Adolescents and Adults With Sickle Cell Disease
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 12 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: 2 years
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 about 2 years."
- 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 whether etavopivat can reduce sickle cell pain crises in people age 12 and older with sickle cell disease. It also checks whether the medicine can reduce organ damage and tiredness and help people exercise more. The study lasts about 2 years, and chance decides whether participants get etavopivat or a placebo (a treatment with no active medicine).
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.
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- Age 12 or older when signing the study consent form.
- Male or female.
- Sickle cell disease confirmed by blood tests.
- Have had 2–15 sickle cell pain crises in the past 12 months, recorded in medical records.
- Have a hemoglobin level of 5–10 g/dL at screening. Hemoglobin is the protein in red blood cells that carries oxygen.
Who can't join
- Used voxelotor or a similar drug within 28 days before study treatment, or expect to need it during the study. Also excluded: recent use of a selectin blocker, such as crizanlizumab, or plans to use one. Its waiting period is at least 28 days and may be longer based on how long the drug stays in the body.
- Get regular red blood cell transfusions, or have had 6 or more transfusions in the past 12 months.
- Had a red blood cell transfusion within 60 days of screening or treatment assignment, unless a blood test before treatment shows adult hemoglobin (HbA) below 10%.
- Used medicines that moderately or strongly speed up the liver enzyme CYP3A4 within 2 weeks before treatment, or medicines that help make blood cells, such as erythropoietin, within 28 days before treatment. Expected use during the study also excludes you.
- Previously had cell-based treatment, such as a blood-forming cell transplant or treatment that changes genes.
- Have certain abnormal liver tests: ALT above 4 times the normal limit or direct bilirubin above 3 times the normal limit. Regular dialysis also excludes you. The listing calls its kidney cutoff 'severe' but gives eGFR above 30; ask the study team to clarify this kidney test rule.
- Cannot or do not take medicine to prevent malaria when recommended in the area where you live, both at consent and during the study.
- Walk less than 100 meters during the 6-minute walk test at screening.
Simplified for readability. The official criteria below are what the study team uses.
Official eligibility criteria
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.
Study sites (50)
AL
- Uni of Alabama at Birmingham · Birmingham, AL 35233More trials in Birmingham
- Univer South Alabama Ped/Onc · Mobile, AL 36604More trials in Mobile
AR
- Arkansas Children's Hospital · Little Rock, AR 72202More trials in Little Rock
AZ
- Phoenix Children's Hsptl · Phoenix, AZ 85016More trials in Phoenix
CA
- Children's Hospital Los Angeles - Endocrinology · Los Angeles, CA 90027More trials in Los Angeles
- UCLA Health · Los Angeles, CA 90095More trials in Los Angeles
- Valley Children's Hospital · Madera, CA 93636More trials in Madera
- University Of California Irvine · Orange, CA 92868More trials in Orange
- Stanford University_Palo Alto · Palo Alto, CA 94304More trials in Palo Alto
- Harbor-UCLA Medical Center · Torrance, CA 90502More trials in Torrance
CO
- Clinical and Transl Res Center · Aurora, CO 80045More trials in Aurora
DC
- Childrens National Medical Ctr · Washington D.C., DC 20010More trials in Washington D.C.
- MedStar Hlth Res Institute · Washington D.C., DC 20010More trials in Washington D.C.
DE
- Nemours/AI duPont Hosp-Chld · Wilmington, DE 19803More trials in Wilmington
FL
- Memorial Healthcare · Hollywood, FL 33021More trials in Hollywood
GA
- Children's Healthcare Atlanta · Atlanta, GA 30329More trials in Atlanta
IL
- Childrens Hospital of Chicago · Chicago, IL 60611More trials in Chicago
- Univer Of Illinois at Chicago · Chicago, IL 60612More trials in Chicago
LA
- Children's Hosp-New Orleans · New Orleans, LA 70118More trials in New Orleans
MA
- Boston Children's Hospital · Boston, MA 02115More trials in Boston
- Boston Medical Center · Boston, MA 02118-2393More trials in Boston
MI
- Henry Ford Hospital_Detroit · Detroit, MI 48202More trials in Detroit
MN
- University of Minnesota · Minneapolis, MN 55455More trials in Minneapolis
MO
- Washington University-St.Louis · St Louis, MO 63110More trials in St Louis
NC
- Duke Comprehen Sickle Cell · Durham, NC 27710More trials in Durham
- Duke University Medical Center · Durham, NC 27710More trials in Durham
- East Carolina Univ-Greenville · Greenville, NC 27834More trials in Greenville
- Atrium Health-Wake Forest Bapt · Winston-Salem, NC 27157More trials in Winston-Salem
NE
- Children's Nebraska · Omaha, NE 68114More trials in Omaha
NJ
- Newark Beth Israel MC · Newark, NJ 07112-2027More trials in Newark
NV
- Cure 4 the Kids Foundation · Las Vegas, NV 89135More trials in Las Vegas
NY
- NYC Health+Hospitals · Brooklyn, NY 11203More trials in Brooklyn
- Interfaith Medical Center · Brooklyn, NY 11238More trials in Brooklyn
- Northwell Health · Mount Kisco, NY 10549More trials in Mount Kisco
- Cohen Children's Medical Ctr · Queens, NY 11040More trials in Queens
- Jacobi Medical Center · The Bronx, NY 10461More trials in The Bronx
- Montefiore Medical Ctr · The Bronx, NY 10467More trials in The Bronx
OH
- Univ Hosp Cleveland Med Ctr · Cleveland, OH 44106More trials in Cleveland
- Ohio State Univ Wexner Med Ctr · Columbus, OH 43203More trials in Columbus
OK
- Univ of OK Health Sciences Ctr · Oklahoma City, OK 73104More trials in Oklahoma City
PA
- Children's Hosptl Philadelphia · Philadelphia, PA 19104More trials in Philadelphia
- St Christopher Hosp for Child · Philadelphia, PA 19134More trials in Philadelphia
TN
- University of Tennessee Health Science Center · Memphis, TN 38104More trials in Memphis
- St. Jude Children's Res Hosp · Memphis, TN 38105More trials in Memphis
TX
- UT Health University of Texas · Houston, TX 77030More trials in Houston
VA
- Inova Health System · Fairfax, VA 22031More trials in Fairfax
- Children's Hsptl Of The Kings · Norfolk, VA 23507More trials in Norfolk
- Virginia Comm Univ Medical Ctr · Richmond, VA 23298More trials in Richmond
WA
- Mary Bridge Children's Health · Tacoma, WA 98405More trials in Tacoma
- Mary Bridge Children's Health · Tacoma, WA 98405More trials in Tacoma