All > Anemia > Sickle Cell Anemia

Etavopivat tablets for Thalassemia

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
University of California, Los Angeles, Los Angeles, CAThalassemia+1 MoreEtavopivat tablets - Drug
Eligibility
12 - 65
All Sexes
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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.

Eligible Conditions
  • Thalassemia
  • Sickle Cell Disease

Treatment Effectiveness

Effectiveness Progress

1 of 3

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1
FT-4202 Tablets
1
Transfusion
1
REGN7257

Study Objectives

3 Primary · 20 Secondary · Reporting Duration: 48 weeks

12 weeks
Change from baseline in hemoglobin over 12 weeks
Body Weight Changes
Changes in serum ferritin levels at 12 weeks versus baseline.
Cohort A: Proportion of patients with ≥ 33% reduction in red blood cell transfusion over a continuous 12-week treatment period versus baseline red blood cell transfusion history
Cohort A: Reduction in red blood cell transfusions over 12 weeks
Red Blood Cell Transfusion
Cohort B: Reduction in red blood cell transfusions over 12 weeks
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
24 weeks
Body Weight Changes
Body Weight Changes
Changes in serum ferritin levels at 24 weeks versus baseline.
Cohort A: Reduction in red blood cell transfusions over 24 weeks
Cohort B: Reduction in red blood cell transfusions over 24 weeks
Cohort C: Hemoglobin response rate at Week 24 (increase of ≥ 1.0 g/dL from baseline).
48 weeks
Change from baseline in hemoglobin over 48 weeks
Changes in liver iron concentration at 48 weeks versus baseline
Body Weight Changes
Changes in serum ferritin levels at 48 weeks versus baseline.
Cohort A: Reduction in red blood cell transfusions over 48 weeks
Cohort B: Reduction in red blood cell transfusions over 48 weeks
Cohort C: Hemoglobin response rate at Week 48 (increase of ≥ 1.0 g/dL from baseline).

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Similar Trials

2
FT-4202 Tablets
2
Transfusion
1
REGN7257

Trial Design

3 Treatment Groups

Etavopivat 400 mg daily - SCD with transfusions
1 of 3
Etavopivat 400 mg daily - Thalassemia with transfusions
1 of 3
Etavopivat 400 mg daily - Thalassemia
1 of 3

Experimental Treatment

60 Total Participants · 3 Treatment Groups

Primary Treatment: Etavopivat tablets · No Placebo Group · Phase 2

Etavopivat 400 mg daily - SCD with transfusions
Drug
Experimental Group · 1 Intervention: Etavopivat tablets · Intervention Types: Drug
Etavopivat 400 mg daily - Thalassemia with transfusions
Drug
Experimental Group · 1 Intervention: Etavopivat tablets · Intervention Types: Drug
Etavopivat 400 mg daily - Thalassemia
Drug
Experimental Group · 1 Intervention: Etavopivat tablets · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: 48 weeks

Who is running the clinical trial?

Forma Therapeutics, Inc.Lead Sponsor
10 Previous Clinical Trials
1,093 Total Patients Enrolled
Leila J Clay, MDStudy DirectorForma Therapeutics, Inc.
Von Potter, MDStudy DirectorForma Therapeutics
1 Previous Clinical Trials
25 Total Patients Enrolled
Patrick Kelly, MDStudy DirectorForma Therapeutics, Inc.
5 Previous Clinical Trials
538 Total Patients Enrolled

Eligibility Criteria

Age 12 - 65 · All Participants · 5 Total Inclusion Criteria

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References

Frequently Asked Questions

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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