A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
Recruiting · NCT06612268 · Interventional (participants receive a specific treatment) · Lead sponsor: Novo Nordisk A/S
View the official record on ClinicalTrials.gov →Interventions studied
EtavopivatPlacebo
What this trial is about
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.
Who can take part
Age range
12 Years and older
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 3
Study type
Interventional (participants receive a specific treatment)
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.
Where it is running
60 locations listed across 27 US states.
- Uni of Alabama at Birmingham - Birmingham, Alabama, United States
- Univer South Alabama Ped/Onc - Mobile, Alabama, United States
- Phoenix Children's Hsptl - Phoenix, Arizona, United States
- Arkansas Children's Hospital - Little Rock, Arkansas, United States
- Children's Hospital Los Angeles - Endocrinology - Los Angeles, California, United States
- UCLA Health - Los Angeles, California, United States
- Valley Children's Hospital - Madera, California, United States
- University Of California Irvine - Orange, California, United States
- Stanford University_Palo Alto - Palo Alto, California, United States
- Harbor-UCLA Medical Center - Torrance, California, United States
- Clinical and Transl Res Center - Aurora, Colorado, United States
- Nemours/AI duPont Hosp-Chld - Wilmington, Delaware, United States
- Childrens National Medical Ctr - Washington D.C., District of Columbia, United States
- MedStar Hlth Res Institute - Washington D.C., District of Columbia, United States
- Memorial Healthcare - Hollywood, Florida, United States
- Children's Healthcare Atlanta - Atlanta, Georgia, United States
- Childrens Hospital of Chicago - Chicago, Illinois, United States
- Univer Of Illinois at Chicago - Chicago, Illinois, United States
- Children's Hosp-New Orleans - New Orleans, Louisiana, United States
- Boston Children's Hospital - Boston, Massachusetts, United States
- Boston Medical Center - Boston, Massachusetts, United States
- Henry Ford Hospital_Detroit - Detroit, Michigan, United States
- University of Minnesota - Minneapolis, Minnesota, United States
- Washington University-St.Louis - St Louis, Missouri, United States
- Children's Nebraska - Omaha, Nebraska, United States
- Cure 4 the Kids Foundation - Las Vegas, Nevada, United States
- Newark Beth Israel MC - Newark, New Jersey, United States
- NYC Health+Hospitals - Brooklyn, New York, United States
- Interfaith Medical Center - Brooklyn, New York, United States
- Northwell Health - Mount Kisco, New York, United States
- Cohen Children's Medical Ctr - Queens, New York, United States
- Jacobi Medical Center - The Bronx, New York, United States
- Montefiore Medical Ctr - The Bronx, New York, United States
- Duke Comprehen Sickle Cell - Durham, North Carolina, United States
- Duke University Medical Center - Durham, North Carolina, United States
- East Carolina Univ-Greenville - Greenville, North Carolina, United States
- Atrium Health-Wake Forest Bapt - Winston-Salem, North Carolina, United States
- Univ Hosp Cleveland Med Ctr - Cleveland, Ohio, United States
- Ohio State Univ Wexner Med Ctr - Columbus, Ohio, United States
- Univ of OK Health Sciences Ctr - Oklahoma City, Oklahoma, United States
- Children's Hosptl Philadelphia - Philadelphia, Pennsylvania, United States
- St Christopher Hosp for Child - Philadelphia, Pennsylvania, United States
- University of Tennessee Health Science Center - Memphis, Tennessee, United States
- St. Jude Children's Res Hosp - Memphis, Tennessee, United States
- UT Health University of Texas - Houston, Texas, United States
- Inova Health System - Fairfax, Virginia, United States
- Children's Hsptl Of The Kings - Norfolk, Virginia, United States
- Virginia Comm Univ Medical Ctr - Richmond, Virginia, United States
- Mary Bridge Children's Health - Tacoma, Washington, United States
- Mary Bridge Children's Health - Tacoma, Washington, United States
- Acıbadem Adana Hastanesi-Hematoloji - Adana, Turkey (Türkiye)
- Başkent Üniversitesi Adana-Hematoloji - Adana, Turkey (Türkiye)
- Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji - Adana, Turkey (Türkiye)
- Agogo Presbyterian Hospital, Ghana - Agogo, Ghana
- Al-Moosa Health Group - Al Ahsa, Saudi Arabia
- King Abdulaziz Hospital-Al Ahsa-National Guard - Al Ahsa, Saudi Arabia
- Prince Mohammed bin Fahad Hospital for Blood Disorders - Al Qaţīf, Saudi Arabia
- AmsterdamUMC VUMC - Amsterdam, Netherlands
- Hacettepe University Hematology - Ankara, Turkey (Türkiye)
- Hacettepe Üniversitesi Hastanesi- Hematoloji - Ankara, Turkey (Türkiye)
Showing 60 of 175 listed sites. See the official record for the complete list.
Read the full protocol, contacts and eligibility on ClinicalTrials.gov →