What side effects are associated with this type of intervention?

Common treatments for Thalassemia that aim to improve red blood cell function or production include hydroxyurea and erythropoietin. Hydroxyurea can cause side effects such as bone marrow suppression, leading to neutropenia, thrombocytopenia, and anemia. It may also cause gastrointestinal disturbances and skin reactions. Erythropoietin therapy can lead to hypertension, increased risk of thromboembolic events, and potential for pure red cell aplasia. Both treatments require careful monitoring to manage these side effects effectively.

What prior FDA approvals exist?

The FDA has approved several treatments for Thalassemia that focus on improving red blood cell function or production. Erythropoiesis-stimulating agents (ESAs) like epoetin alfa and darbepoetin alfa have been used to treat anemia in Thalassemia by stimulating the bone marrow to produce more red blood cells. Additionally, luspatercept-aamt (Reblozyl) has been approved to reduce the need for red blood cell transfusions in adult patients with beta-thalassemia. These treatments aim to enhance the production and function of red blood cells, similar to the investigational drug Etavopivat.

What other types of research exist?

Promising novel alternatives to Etavopivat for Thalassemia patients include Vadadustat, which is an oral hypoxia-inducible factor prolyl hydroxylase inhibitor that stimulates erythropoiesis, and Luspatercept, which has shown efficacy in improving hemoglobin levels and reducing transfusion dependence in patients with lower-risk myelodysplastic syndromes, a condition with similar hematologic complications.

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Small Molecule

Etavopivat for Sickle Cell Disease

Phase 2

Recruiting

Research Sponsored by Forma Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

At least 24 months of chronic monthly red blood cell transfusions for secondary stroke prevention/treatment of primary stroke

Chronically red blood cell transfused for primary stroke prevention or due to previous stroke

Must not have

Active hepatitis B or hepatitis C infection

Severe renal dysfunction or on chronic dialysis

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 48 weeks

Awards & highlights

Summary

This trial is testing a new oral medicine called etavopivat in patients with sickle cell disease or thalassemia. The medicine helps red blood cells produce energy more efficiently. The goal is to reduce the need for blood transfusions and increase hemoglobin levels.

Who is the study for?

This trial is for individuals with thalassemia or sickle cell disease who consent to participate and agree to use contraception if applicable. It's not for those who are pregnant, breastfeeding, have significant liver issues (high ALT or bilirubin levels), HIV, active hepatitis B/C, severe kidney dysfunction, recent malignancies requiring intense treatment unless considered cured of certain cancers, or unstable heart/lung conditions.

What is being tested?

The study tests Etavopivat tablets in a Phase 2 trial to see if they're safe and effective for patients with thalassemia or sickle cell disease. The goal is to reduce the need for red blood cell transfusions and increase hemoglobin levels in participants.

What are the potential side effects?

While specific side effects of Etavopivat aren't listed here, common types of side effects from similar treatments may include gastrointestinal symptoms like nausea or diarrhea, potential liver enzyme elevations, fatigue, headache and possible allergic reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been receiving monthly blood transfusions for at least 2 years to prevent or treat a stroke.

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I regularly receive blood transfusions to prevent or because of a previous stroke.

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I have been receiving regular blood transfusions for at least a year to prevent strokes.

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I've had regular blood transfusions to manage my HbS levels below 45% for the last 3 months.

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I have been diagnosed with a form of thalassemia.

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I have been diagnosed with sickle cell disease.

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I regularly receive blood transfusions.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have an active hepatitis B or C infection.

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I have severe kidney problems or am on long-term dialysis.

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I am HIV positive.

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I have had cancer treatment with chemo or radiation in the last 2 years.

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I have liver issues, including high enzyme levels or cirrhosis.

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I am currently pregnant or breastfeeding.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 48 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~48 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 48 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

Cohort C: Hemoglobin response rate at Week 12 (increase of ≥ 1.0 g/dL from baseline)

Cohorts A: Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history

Cohorts B: Proportion of patients with ≥ 20% reduction in red blood cell transfusions over a continuous 12-week treatment period versus baseline red blood cell transfusion history

Secondary study objectives

Change from baseline in hemoglobin over 12 weeks

Body Weight Changes

Change from baseline in hemoglobin over 48 weeks

+14 more

Trial Design

3Treatment groups

Experimental Treatment

Group I: Etavopivat 400 mg daily - Thalassemia with transfusionsExperimental Treatment1 Intervention

Patients with thalassemia on chronic red blood cell transfusions

Group II: Etavopivat 400 mg daily - ThalassemiaExperimental Treatment1 Intervention

Patients with thalassemia not on chronic red blood cell transfusions

Group III: Etavopivat 400 mg daily - SCD with transfusionsExperimental Treatment1 Intervention

Patients with sickle cell disease on chronic red blood cell transfusions

0 / 13Eligibility criteria met

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

The most common treatments for Thalassemia aim to improve red blood cell function or increase their production. Etavopivat, for example, works by enhancing the function of red blood cells and increasing hemoglobin levels, which can reduce the need for frequent blood transfusions. This is crucial for Thalassemia patients as it helps manage anemia and reduces complications associated with chronic transfusions, such as iron overload. Other treatments, like erythropoiesis-stimulating agents (ESAs), also aim to boost red blood cell production, thereby improving oxygen delivery and reducing transfusion dependency. These mechanisms are vital for improving the quality of life and long-term outcomes for Thalassemia patients.

The practical aspects of therapy with rHuEPO.Efficacy of cytotoxic agents used in the treatment of testicular germ cell tumours under normoxic and hypoxic conditions in vitro.Red blood cells.