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New treatment option for Sickle Cell Disease
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?
Etavopivat is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for sickle cell disease.
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
- 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.
- 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 1-15 episodes of documented vaso occlusive crises (VOC) within the 12 months prior to screening. Documentation must exist in the participant's...
- 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 g/L)...
You likely can't join if
- More than 15 VOCs within the past 12 months prior to screening documented in the participant's medical record. Events based solely on participant...
- 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 inducers of CYP3A4 (cytochrome p450 3a4) within 2 weeks of starting study treatment or...
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 1-15 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...
- 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 g/L) at screening.
- More than 15 VOCs within the past 12 months prior to screening documented in the participant's medical record. Events based solely on participant recall without supporting documentation should not be counted towards...
- 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 in the previous 12 months (i.e., an average...
- 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 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.
The study team makes the final eligibility decision.
Where it's taking place
- 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
- Orange, California, United States
- Palo Alto, California, United States
- Torrance, California, United States
- Aurora, Colorado, 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
- Detroit, Michigan, United States
- Minneapolis, Minnesota, United States
- St Louis, Missouri, United States
+ 114 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 - 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?
Study sites include 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 128 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.