RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2025-01-10
Study Completion:2029-11-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06609226

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.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * 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.
  2. * Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator.
  3. * Any participant with dose reduction or temporary discontinuation will need to be rechallenged before transferring.
  4. * Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they:
  5. * 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).
  6. * Have been compliant with the treatment regimen at the discretion of the investigator during participation of the parent study.
  1. * 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.
  2. * Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study.
  3. * Participants on permanent dose reduction or temporary treatment discontinuation.
  4. * Use of any of the following within the timeframes prior to the transfer visit as stated:
  5. * Use of voxelotor within participation of the parent study or anticipated need for this agent during this study.
  6. * 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.
  7. * Use of erythropoietin or other haematopoietic growth factor treatment within the parent study or anticipated need for such agents during this study.
  8. * 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.
  9. * 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.

Contacts and Locations

Study Contact

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

Principal Investigator

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

Study Locations (Sites)

Univ of Alabama Birmingham
Birmingham, Alabama, 35233
United States
Phoenix Children's Hsptl
Phoenix, Arizona, 85016
United States
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, 90027
United States
UCSF Oakland Benioff ChildHosp
Oakland, California, 94609
United States
Children's Hosp Of Orange
Orange, California, 92868
United States
University Of California Irvine
Orange, California, 92868
United States
University of Connecticut
Farmington, Connecticut, 06030
United States
Children's National Medical Center
Washington, District of Columbia, 20010
United States
Foundation for Sickle Cell Disease Research
Hollywood, Florida, 33021
United States
Univ of Miami/SCCC
Miami, Florida, 33136
United States
Emory University School of Medicine
Atlanta, Georgia, 30303
United States
Children's Healthcare Atlanta
Atlanta, Georgia, 30329
United States
Center for Blood Disorders Augusta University
Augusta, Georgia, 30912
United States
Children's Hosp-New Orleans
New Orleans, Louisiana, 70118
United States
Boston Medical Center
Boston, Massachusetts, 02118
United States
Washington University-St.Louis
Saint Louis, Missouri, 63110
United States
Jacobi Medical Center
Bronx, New York, 10461
United States
Montefiore Medical Center
Bronx, New York, 10467
United States
NYC Health+Hospitals
Brooklyn, New York, 11203
United States
Columbia University Medical Center_New York_0
New York, New York, 10032
United States
Weill Cornell Med Coll-NYPH
New York, New York, 10065
United States
Duke University_Durham
Durham, North Carolina, 27705
United States
East Carolina University_Greenville
Greenville, North Carolina, 27834
United States
Atrium Health-Wake Forest Bapt
Winston-Salem, North Carolina, 27157
United States
Cincinnati Child's Hsp Med Ctr
Cincinnati, Ohio, 45229
United States
Neuro-Behavioral Clinical Research
North Canton, Ohio, 44720
United States
Medical University Of South Carolina_Charleston
Charleston, South Carolina, 29425
United States
East Carolina Univ-Greenville
Greenville, South Carolina, 27858
United States
UTHSC-Memphis
Memphis, Tennessee, 38104
United States
Texas Children's Hospital_Houston
Houston, Texas, 77030
United States
UT Health University of Texas
Houston, Texas, 77030
United States
Virginia Comm Univ Medical Ctr
Richmond, Virginia, 23298
United States
Mary Bridge Children's Health
Tacoma, Washington, 98405
United States
Versiti, CCBD_Milwaukee
Milwaukee, Wisconsin, 53226
United States

Collaborators and Investigators

Sponsor: Novo Nordisk A/S

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-01-10
Study Completion Date2029-11-30

Study Record Updates

Study Start Date2025-01-10
Study Completion Date2029-11-30

Terms related to this study

Additional Relevant MeSH Terms

  • Sickle Cell Disease, Thalassemia