Recruiting
Phase 3

Etavopivat

Sponsor:

Novo Nordisk A/S

Code:

NCT06609226

Conditions

Sickle Cell Disease

Thalassemia

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Interventions

Etavopivat A

Etavopivat B

Etavopivat C

Study Details

Brief summary:

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.

Conditions

Sickle Cell Disease

Thalassemia

Study ID

NCT06609226

Start date

Jan 10, 2025

Status verified date

Aug, 2026

Completion date

Dec 30, 2030

Anticipated

Primary completion date

Dec 30, 2030

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Inclusion Criteria:

  • 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.
  • Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator.
  • Any participant with dose reduction or temporary discontinuation will need to be successfully rechallenged to the full dose of etavopivat before transferring.
  • Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they have been on a stable dose in the parent study as defined at the investigator's discretion. Necessary adjustments related to weight or age are accepted. Participants with temporary dose reductions or pauses due to medical reasons may still be considered to have a stable dose, as determined by the investigator, who will assess the impact of these adjustments based on clinical context and the participant's overall health status.

Exclusion Criteria:

  • Any disorder, except for conditions associated with SCD or thalassaemia, which in the investigator's opinion might jeopardise participant's safety or compliance with the protocol.
  • Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study.
  • Participants on permanent dose reduction (greater than \[>\] 28 days or more) or ongoing temporary treatment discontinuation.
  • Use of any of the following within the timeframes prior to the transfer visit as stated:
  • Use of haemoglobin S (HbS) polymerisation inhibitors within participation of the parent study or anticipated need for this agent during this study.
  • Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within the parent study or anticipated need for such agents during this study.
  • Use of erythropoietin or other haematopoietic growth factor treatment for more than 4 consecutive weeks during the parent study or anticipated need of such agents for a maintenance treatment during this study.
  • Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4 within 2 weeks of the transfer visit or anticipated need for such agents during the study.
  • Current participation in a study that is not a designated parent study, or planned participation in any other clinical study, for the duration of FLORAL.

Study Design

Enrollment

480 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Participants greater than or equal to (≥) 12 years old with sickle cell disease

Participants will receive an oral dose of Etavopivat A or C.

experimental: Participants ≥ 12 years old with sickle cell disease transfusion dependent

Participants will receive an oral dose of Etavopivat A or C.

experimental: Participants ≥ 12 years old with transfusion-dependent thalassaemia

Participants will receive an oral dose of Etavopivat A or C.

experimental: Participants ≥ 12 years old with non-transfusion dependent thalassaemia

Participants will receive an oral dose of Etavopivat A or C.

experimental: Participants ≥ 2 years to less than (<) 12 years old with sickle cell disease

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

Interventions

Etavopivat A

Participants will receive an oral dose of Etavopivat A.

Etavopivat B

Participants will receive an oral dose of Etavopivat B.

Etavopivat C

Participants will receive an oral dose of Etavopivat C.

Primary outcome measure

  • Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately [ Time Frame: Baseline (week 0 of FLORAL) up to end of study (up to week 316) ]
  • Number of adverse reactions, reported for each indication and age group separately [ Time Frame: Baseline (week 0 of FLORAL) up to end of study (up to week 316) ]

Central Contacts and Locations

Central contacts

Novo Nordisk

(+1) 866-867-7178clinicaltrials@novonordisk.com

Locations

Children's Hospital Los Angeles - Endocrinology

Recruiting

Los Angeles, California, United States, 90027

UCSF Oakland Benioff ChildHosp

Recruiting

Oakland, California, United States, 94609

Children's Healthcare Atlanta

Recruiting

Atlanta, Georgia, United States, 30329

Univer Of Illinois at Chicago

Recruiting

Chicago, Illinois, United States, 60612

Columbia University Medical Center_New York_0

Recruiting

New York, New York, United States, 10032

Duke University_Durham

Recruiting

Durham, North Carolina, United States, 27710

Texas Children's Hospital_Houston

Recruiting

Houston, Texas, United States, 77030

UT Health University of Texas

Recruiting

Houston, Texas, United States, 77030

Versiti, CCBD_Milwaukee

Recruiting

Milwaukee, Wisconsin, United States, 53226

More Information

Sponsor

Novo Nordisk A/S

Last update posted

Aug 13, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Novo Nordisk A/S on 2026-08-13.