Sickle Cell Disease study: Etavopivat
Official title A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
ClinicalTrials.gov ID: NCT06612268
What this study is testing
What is Etavopivat?
An investigational oral medicine being studied as a potential treatment for sickle cell disease. It is designed to activate the red‑blood‑cell enzyme pyruvate kinase‑R, which may improve the cells' energy balance.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study aims to find out if etavopivat reduces vaso‑occlusive pain crises, organ damage, fatigue, and improves exercise ability in people with sickle cell disease.
- Who it's for
- Adults and adolescents who have sickle cell disease.
- What's involved
- Participants are randomly given either etavopivat or a placebo and will have regular check‑ups and tests to monitor pain episodes, organ health, fatigue, and exercise capacity.
- Time commitment
- The trial lasts about two years with multiple study visits over that period.
- Phase 3: a large, late-stage study
- Time commitment: about 2 years
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- 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...
- Have 2-15 episodes of documented vaso occlusive crises (VOC) within the 12 months prior to screening.
- 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...
You likely can't join if
- 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...
- Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) or greater than or equal...
- 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...
- Receiving or use of concomitant medications that are strong or moderate inducers of CYP3A4 (cytochrome p450 3a4) within 2 weeks of starting study...
- Use of erythropoietin or other haematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents...
See the full eligibility 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...
- 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.
- 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.
- 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...
- 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...
- 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...
- 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).
- 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.
The study team makes the final eligibility decision.
Where it is enrolling
- Birmingham, Alabama, United States
- Mobile, Alabama, United States
- Phoenix, Arizona, United States
- Little Rock, Arkansas, United States
- Los Angeles, California, United States
- Madera, California, United States
- Palo Alto, California, United States
- Torrance, California, United States
- Wilmington, Delaware, United States
- Washington D.C., District of Columbia, United States
- Hollywood, Florida, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- New Orleans, Louisiana, United States
- Boston, Massachusetts, United States
- Minneapolis, Minnesota, United States
- St Louis, Missouri, United States
- Omaha, Nebraska, United States
- Newark, New Jersey, United States
- Brooklyn, New York, United States
+ 85 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The study runs about 2 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Birmingham, Alabama, United States; Mobile, Alabama, United States; Phoenix, Arizona, United States; Little Rock, Arkansas, United States; Los Angeles, California, United States; Madera, California, United States and 99 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.