← Back to Search

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 48 weeks

Awards & highlights

Study Summary

This trial will evaluate the safety and clinical activity of a new drug, etavopivat, in patients with thalassemia or sickle cell disease. The goal is to see if the drug can lower the number of red blood cell transfusions required and increase hemoglobin.

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.Check my eligibility

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.See study design

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

Select...

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

Select...

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

Select...

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

Select...

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

Select...

I have been diagnosed with a form of thalassemia.

Select...

I have been diagnosed with sickle cell disease.

Select...

I regularly receive blood transfusions.

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

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 outcome measures

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 / 7Eligibility criteria met

Find a Location

Who is running the clinical trial?

Forma Therapeutics, Inc.Lead Sponsor

12 Previous Clinical Trials

1,189 Total Patients Enrolled

Novo Nordisk A/SLead Sponsor

1,514 Previous Clinical Trials

2,415,008 Total Patients Enrolled

Clinical Transparency (dept. 2834)Study DirectorNovo Nordisk A/S

106 Previous Clinical Trials

139,766 Total Patients Enrolled

Media Library

Etavopivat (Small Molecule) Clinical Trial Eligibility Overview. Trial Name: NCT04987489 — Phase 2

Thalassemia Research Study Groups: Etavopivat 400 mg daily - SCD with transfusions, Etavopivat 400 mg daily - Thalassemia with transfusions, Etavopivat 400 mg daily - Thalassemia

Thalassemia Clinical Trial 2023: Etavopivat Highlights & Side Effects. Trial Name: NCT04987489 — Phase 2

Etavopivat (Small Molecule) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04987489 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is enrollment for this research project still available?

"According to the latest update on clinicaltrials.gov, enrollment for this research is ongoing. This trial was initially advertised in December of 2021 and its details were most recently amended on October 12th 2022."

Answered by AI

How many participants are enrolled in this clinical trial at its current capacity?

"This clinical trial requires 60 eligible participants to be recruited. There are a variety of medical sites in which one can partake such as East Carolina University located in Greenville, NC and Children's Hospital Los Angeles situated in LA, CA."

Answered by AI

May I partake in this clinical trial?

"In order to be considered for participation in this medical trial, candidates must possess thalassemia and fall between the ages of 12 and 65. Thus far, 60 individuals have been recruited into the study."

Answered by AI

Is this research open to elderly individuals aged 75 or more?

"This medical trial is open to participants of 12 years or greater and those under 65."

Answered by AI

Are there any potential risks associated with consuming Etavopivat tablets?

"Our team at Power judged the safety of Etavopivat tablets to be a 2, as this is only Phase 2 trial, meaning there exists some evidence for its security but none that it has any beneficial effect."

Answered by AI

What is the geographical range of implementation for this trial?

"The trial is being administered at East Carolina University, Children's Hospital Los Angeles, and Weill Medical College of Cornell University in addition to 10 other research sites."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

2021. "A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04987489.

All > Sickle Cell Disease > Sickle Cell Anemia