Etavopivat for Sickle Cell Anemia

(FLORAL Trial)

Recruiting at 92 trial locations

Overseen By Novo Nordisk

Age: Any Age

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Novo Nordisk A/S

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking into how safe treatment with etavopivat is and how well it works over a long period of time. The study will last for up to 264 weeks, but it will end earlier if etavopivat is approved in the participant's country.

Research Team

Clinical Transparency (dept. 2834)

Principal Investigator

Novo Nordisk A/S

Eligibility Criteria

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.

Inclusion Criteria

I am currently taking hydroxyurea, crizanlizumab, or Endari without changing the dose recently.

I have shown improvement with etavopivat treatment as judged by my doctor.

Participant must have ongoing participation in an etavopivat parent study for treatment of sickle cell disease (SCD) or thalassaemia and have completed at least a treatment period of the parent study

Exclusion Criteria

I am on a lower dose or have temporarily stopped my treatment.

I am not taking, nor will I need, strong drugs that affect liver enzymes during the study.

I have not used experimental drugs for blood cell disorders in this or any study.

Treatment Details

Interventions

• Etavopivat (Other)

Trial Overview 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.

Participant Groups

Group I: Participants ≥ 12 years old with transfusion-dependent thalassaemia Experimental Treatment 1 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) transfusions Experimental Treatment 1 Intervention

Participants will receive an oral dose of Etavopivat A.

Group III: Participants ≥ 12 years old with non-transfusion dependent thalassaemia Experimental Treatment 1 Intervention

Participants will receive an oral dose of Etavopivat A.

Group IV: Participants ≥ 11 months to less than (<) 12 years old with sickle cell disease Experimental Treatment 2 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 disease Experimental Treatment 1 Intervention

Participants will receive an oral dose of Etavopivat A.

Who Is Running the Clinical Trial?

Novo Nordisk A/S

Lead Sponsor

Trials

1,578

Recruited

3,813,000+

Lars Fruergaard Jørgensen

Novo Nordisk A/S

Chief Executive Officer since 2017

MSc in Finance and Business Administration, Aarhus School of Business, Aarhus University, Denmark

Martin Holst Lange

Novo Nordisk A/S

Chief Medical Officer since 2021

MD from University of Copenhagen