A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Description

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

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Study Overview

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Study Details

Study overview

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.

An Open-label, Multi-centre, Rollover Study to Characterise Long-term Safety and Efficacy of Etavopivat in Adults, Adolescents and Children Who Have Sickle Cell Disease or Thalassaemia and Have Completed a Treatment Period in an Etavopivat Study

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

Condition
Sickle Cell Disease, Thalassemia
Intervention / Treatment

-

Contacts and Locations

Birmingham

Univ of Alabama Birmingham, Birmingham, Alabama, United States, 35233

Phoenix

Phoenix Children's Hsptl, Phoenix, Arizona, United States, 85016

Los Angeles

Children's Hospital Los Angeles - Endocrinology, Los Angeles, California, United States, 90027

Oakland

UCSF Oakland Benioff ChildHosp, Oakland, California, United States, 94609

Orange

Children's Hosp Of Orange, Orange, California, United States, 92868

Orange

University Of California Irvine, Orange, California, United States, 92868

Farmington

University of Connecticut, Farmington, Connecticut, United States, 06030

Washington

Children's National Medical Center, Washington, District of Columbia, United States, 20010

Hollywood

Foundation for Sickle Cell Disease Research, Hollywood, Florida, United States, 33021

Miami

Univ of Miami/SCCC, Miami, Florida, United States, 33136

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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 rechallenged 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 during participation in the parent study (i.e., no changes to the dose except for changes to weight or age reasons).
  • * Have been compliant with the treatment regimen at the discretion of the investigator during participation of the parent study.
  • * 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 or temporary treatment discontinuation.
  • * Use of any of the following within the timeframes prior to the transfer visit as stated:
  • * Use of voxelotor 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 within the parent study or anticipated need for such agents 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 trial, for the duration of FLORAL.

Ages Eligible for Study

to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Novo Nordisk A/S,

Clinical Transparency (dept. 2834), STUDY_DIRECTOR, Novo Nordisk A/S

Study Record Dates

2029-11-30