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Etavopivat for Sickle Cell Disease
Phase 2
Recruiting
Led By Amy Tang, MD
Research Sponsored by Novo Nordisk A/S
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 weeks
Awards & highlights
No Placebo-Only Group
Summary
This trial tests etavopivat, a medication aimed at improving blood flow and oxygen use in the brain and muscles, in young patients with sickle cell disease. Etavopivat is being developed for the treatment of sickle cell disease.
Who is the study for?
This trial is for children aged 12-21 with Sickle Cell Disease, specifically those with hemoglobin SS or S/beta0 thalassemia and a hemoglobin level ≤ 9.0 g/dL. Stable hydroxyurea therapy is okay, but not if they've had recent severe complications like organ dysfunction, transfusions, surgery on stomach/small intestine, or unstable heart conditions.
What is being tested?
The study tests Etavopivat's effects on brain and muscle blood flow in young patients with Sickle Cell Disease. It's an open-label trial where all participants receive the drug to see how it influences their cerebral hemodynamics.
What are the potential side effects?
While specific side effects of Etavopivat are not listed here, common risks may include potential reactions at the infusion site, fatigue, headache or dizziness due to changes in blood flow; however individual experiences can vary.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 24 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 weeks
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Primary study objectives
Effect of etavopivat on cerebral blood flow (CBF)
Effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
Effect of etavopivat on oxygen ejection fraction (OEF)
Secondary study objectives
Adverse events in participants with SCD
Muscle hemodynamic effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
Muscle hemodynamic effect of etavopivat on muscle blood flow
+4 moreAwards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
1Treatment groups
Experimental Treatment
Group I: EtavopivatExperimental Treatment1 Intervention
Single-arm, open-label
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Etavopivat
2024
Completed Phase 1
~20
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Sickle Cell Disease (SCD) include hydroxyurea, red blood cell transfusions, and pyruvate kinase activators like Etavopivat. Hydroxyurea works by increasing fetal hemoglobin (Hb F) production, which reduces red blood cell sickling and vaso-occlusive events.
Red blood cell transfusions provide normal red blood cells, decreasing the proportion of sickled cells and reducing complications such as stroke and acute chest syndrome. Pyruvate kinase activators enhance red blood cell energy metabolism, increasing ATP levels and reducing oxidative stress, thereby improving red blood cell function and lifespan.
These treatments are essential for SCD patients as they help manage pain, prevent complications, and improve overall quality of life.
Mitapivat increases ATP and decreases oxidative stress and erythrocyte mitochondria retention in a SCD mouse model.
Mitapivat increases ATP and decreases oxidative stress and erythrocyte mitochondria retention in a SCD mouse model.
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Who is running the clinical trial?
Novo Nordisk A/SLead Sponsor
1,552 Previous Clinical Trials
2,445,023 Total Patients Enrolled
Forma Therapeutics, Inc.Lead Sponsor
12 Previous Clinical Trials
1,209 Total Patients Enrolled
Emory UniversityOTHER
1,700 Previous Clinical Trials
2,604,586 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I need regular blood transfusions to manage my condition.I have not had a spleen disorder in the last 14 days.I have had swollen fingers or toes in the last 14 days.I have severe kidney problems or am on long-term dialysis.I have not had chemotherapy or radiation in the last 2 years.I have had a stroke or brain bleed in the last 2 years.I have sickle cell disease with either HbSS or HbS/β0 thalassemia.I have had major surgery on my stomach or small intestine.I am currently pregnant or breastfeeding.I am currently being treated for a serious infection.I have received a red blood cell transfusion in the last 60 days.I do not have serious heart or lung conditions.I have been on a stable dose of hydroxyurea for 3 months without blood-related side effects.I have liver problems.
Research Study Groups:
This trial has the following groups:- Group 1: Etavopivat
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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