What side effects are associated with this type of intervention?

Hydroxyurea, a common treatment for SCD, can cause side effects such as bone marrow suppression, leading to neutropenia, thrombocytopenia, and anemia. It may also cause gastrointestinal disturbances, skin and nail changes, and potential teratogenic effects, thus it is contraindicated during pregnancy. Chronic transfusions can lead to iron overload, alloimmunization, and increased risk of infections. Investigational therapies like Etavopivat, a pyruvate kinase activator, are still under study, but potential side effects could include those related to changes in red blood cell metabolism and oxidative stress. Patients should discuss these risks with their healthcare provider to make informed decisions.

What prior FDA approvals exist?

Hydroxyurea is one of the primary FDA-approved treatments for Sickle Cell Disease, known for its efficacy in reducing vaso-occlusive pain and other complications. It is recommended for individuals with severe genotypes such as homozygous SCD (Hb SS) and sickle beta0 thalassemia. Other treatments include chronic transfusions and hematopoietic stem cell transplantation, which are used in specific cases. While the context does not mention FDA-approved pyruvate kinase activators like Etavopivat, it highlights the ongoing research and clinical trials aimed at exploring new therapeutic options for SCD.

What other types of research exist?

Promising novel alternatives to Etavopivat for Sickle Cell Disease patients in 2024 include: 1) l-glutamine (Endari), which has shown a significant reduction in pain crises; 2) Hydroxyurea, recommended for its efficacy in reducing vaso-occlusive pain and other complications; 3) Pentoxifylline and SQ22536, which improve red blood cell deformability; 4) Mitapivat, which increases ATP and decreases oxidative stress in SCD models; and 5) Sevuparin, which is being evaluated for its effectiveness in treating acute pain crises.

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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

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 weeks for reporting.

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 more

Awards & 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

~40

Do I qualify?Apply below to find out

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.