← Back to Search

Other

Etavopivat for Sickle Cell Anemia (FLORAL Trial)

Phase 3

Waitlist Available

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline (week 0 of floral) to end of study (week 264, or earlier)

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

"This trial is testing a new medication called Etavopivat for treating blood disorders like sickle cell disease and thalassaemia. The medication aims to improve the function of haemoglobin,

Who is the study for?

This trial is for individuals with inherited blood disorders, specifically sickle cell disease or thalassaemia. Participants will be involved in the study for up to 264 weeks unless etavopivat gets approved sooner in their country.

What is being tested?

The study tests long-term safety and effectiveness of a new medication called etavopivat, which aims to treat anemia caused by sickle cell disease and thalassaemia by improving haemoglobin function.

What are the potential side effects?

As etavopivat is under development, specific side effects are being studied. Generally, potential side effects may include issues related to blood chemistry changes or reactions at the site of administration.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline (week 0 of floral) to end of study (week 264, or earlier)

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline (week 0 of floral) to end of study (week 264, or earlier)

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline (week 0 of floral) to end of study (week 264, or earlier) for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of adverse reactions, reported for each indication and age group separately

Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately

Secondary study objectives

Annualised number of hospitalisations, reported for each age group separately

Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately

Average length of stay of hospitalisations, reported for each age group separately

+5 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

5Treatment groups

Experimental Treatment

Group I: Participants ≥ 12 years old with transfusion-dependent thalassaemiaExperimental Treatment1 Intervention

Participants will receive an oral dose of Etavopivat A.

Group II: Participants ≥ 12 years old with sickle cell disease on chronic red blood cell (RBC) transfusionsExperimental Treatment1 Intervention

Participants will receive an oral dose of Etavopivat A.

Group III: Participants ≥ 12 years old with non-transfusion dependent thalassaemiaExperimental Treatment1 Intervention

Participants will receive an oral dose of Etavopivat A.

Group IV: Participants ≥ 11 months to less than (<) 12 years old with sickle cell diseaseExperimental Treatment2 Interventions

Participants ≥ 12 years of age will receive an oral dose of Etavopivat A and participants \< 12 years of age will receive an oral dose of Etavopivat B.

Group V: Participants greater than or equal to (≥) 12 years old with sickle cell diseaseExperimental Treatment1 Intervention

Participants will receive an oral dose of Etavopivat A.

Do I qualify?Apply below to find out