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

Status: Recruiting
Location: See all (100) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: f
View:

• 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.

Locations
United States
Alabama
Univ of Alabama Birmingham
ACTIVE_NOT_RECRUITING
Birmingham
Arizona
Phoenix Children's Hsptl
NOT_YET_RECRUITING
Phoenix
California
Children's Hospital Los Angeles - Endocrinology
RECRUITING
Los Angeles
Children's Hospital Los Angeles - Endocrinology
ACTIVE_NOT_RECRUITING
Los Angeles
UCSF Oakland Benioff ChildHosp
RECRUITING
Oakland
UCSF Oakland Benioff ChildHosp
ACTIVE_NOT_RECRUITING
Oakland
Children's Hosp Of Orange
WITHDRAWN
Orange
University Of California Irvine
RECRUITING
Orange
Connecticut
University of Connecticut
ACTIVE_NOT_RECRUITING
Farmington
Washington, D.c.
Children's National Medical Center
NOT_YET_RECRUITING
Washington D.c.
Florida
Foundation for Sickle Cell Disease Research
RECRUITING
Hollywood
Univ of Miami/SCCC
WITHDRAWN
Miami
Georgia
Children's Healthcare Atlanta
RECRUITING
Atlanta
Emory University School of Medicine
RECRUITING
Atlanta
Center for Blood Disorders Augusta University
RECRUITING
Augusta
Illinois
Univer Of Illinois at Chicago
NOT_YET_RECRUITING
Chicago
Louisiana
Children's Hosp-New Orleans
WITHDRAWN
New Orleans
Massachusetts
Boston Medical Center
ACTIVE_NOT_RECRUITING
Boston
Missouri
Washington University-St.Louis
RECRUITING
St Louis
North Carolina
Atrium Levine Children's/Atrium Health
NOT_YET_RECRUITING
Charlotte
Duke University_Durham
RECRUITING
Durham
East Carolina Univ-Greenville
RECRUITING
Greenville
East Carolina University_Greenville
WITHDRAWN
Greenville
Atrium Health-Wake Forest Bapt
NOT_YET_RECRUITING
Winston-salem
New York
NYC Health+Hospitals
RECRUITING
Brooklyn
Columbia University Medical Center_New York_0
NOT_YET_RECRUITING
New York
Columbia University Medical Center_New York_0
RECRUITING
New York
Weill Cornell Med Coll-NYPH
RECRUITING
New York
Jacobi Medical Center
ACTIVE_NOT_RECRUITING
The Bronx
Montefiore Medical Center
NOT_YET_RECRUITING
The Bronx
Ohio
Cincinnati Child's Hsp Med Ctr
ACTIVE_NOT_RECRUITING
Cincinnati
Neuro-Behavioral Clinical Research
ACTIVE_NOT_RECRUITING
North Canton
South Carolina
Medical University Of South Carolina_Charleston
NOT_YET_RECRUITING
Charleston
Tennessee
Methodist University Hospital
NOT_YET_RECRUITING
Memphis
Texas
Texas Children's Hospital_Houston
RECRUITING
Houston
UT Health University of Texas
RECRUITING
Houston
Virginia
Virginia Comm Univ Medical Ctr
NOT_YET_RECRUITING
Richmond
Washington
Mary Bridge Children's Health
ACTIVE_NOT_RECRUITING
Tacoma
Wisconsin
Versiti, CCBD_Milwaukee
NOT_YET_RECRUITING
Milwaukee
Other Locations
Canada
CHU Ste-Justine
RECRUITING
Montreal
The Hospital for Sick Children
ACTIVE_NOT_RECRUITING
Toronto
University Health Network - Toronto General Hospital
RECRUITING
Toronto
St Pauls Hospital
RECRUITING
Vancouver
Egypt
Alexandria University Hospital
NOT_YET_RECRUITING
Alexandria, Egypt
Zagazig University Hospital
NOT_YET_RECRUITING
Alsharkia, Egypt
Faculty of Medicine Ain Shams Medical Research Institute (MASRI)
NOT_YET_RECRUITING
Cairo
Abu El-Reesh El-Mounira Children University Hospital
RECRUITING
Cairo, Egypt
Cairo University
RECRUITING
Cairo, Egypt
France
Ap-Hp-Hopital Henri Mondor
NOT_YET_RECRUITING
Créteil
Hospices Civils de Lyon-Hopital Edouard Herriot
NOT_YET_RECRUITING
Lyon
Ap-Hp-Hopital Robert Debre
RECRUITING
Paris
Ap-Hp-Hopital Robert Debre
NOT_YET_RECRUITING
Paris
Germany
Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie
NOT_YET_RECRUITING
Berlin
Universitätsklinikum Freiburg - Kinder- und Jugendklinik
RECRUITING
Freiburg Im Breisgau
Ghana
Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH)
NOT_YET_RECRUITING
Accra
Kintampo Health Research Centre (KHRC), Kintampo, Ghana
NOT_YET_RECRUITING
Kintampo
Greece
Hippokration Hospital
RECRUITING
Athens
General Hospital Of Larissa Koutlibaneio And Triantafylleio
RECRUITING
Larissa
General University Hospital of Patras
RECRUITING
Pátrai
'Ippokrateio' General Hospital of Thessaloniki
NOT_YET_RECRUITING
Thessaloniki
India
Victoria Hospital, Bangalore
NOT_YET_RECRUITING
Bangalore
Nirmal Hospital Pvt. Ltd.
NOT_YET_RECRUITING
Gujarat
Suretech Hospital and Research Centre Ltd.
NOT_YET_RECRUITING
Maharashtra
K.J Somaiya Hospital and Research Centre
NOT_YET_RECRUITING
Mumbai
All India Institute of Medical Sciences_Delhi
NOT_YET_RECRUITING
New Delhi
All India Institute of Medical Sciences (AIIMS), Raipur
NOT_YET_RECRUITING
Raipur
Italy
Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche
RECRUITING
Orbassano
Azienda Ospedale Universita Padova
RECRUITING
Padua
Fondazione IRCCS Policlinico San Matteo
RECRUITING
Pavia
Kenya
KEMRI-Walter-Reed Kericho
NOT_YET_RECRUITING
Kericho
Ahero Clinical Trials Unit, Kisumu, Kenya
RECRUITING
Kisumu
KEMRI Kondele Children Hospital, Kisumu
NOT_YET_RECRUITING
Kisumu
Kombewa Clinical Research Centre
NOT_YET_RECRUITING
Kisumu
Gertrude's Children's Hospital, Nairobi
NOT_YET_RECRUITING
Nairobi
KEMRI Siaya Clinical Research Annex Siaya County Referral Hospital, Siaya
NOT_YET_RECRUITING
Siaya
Lebanon
American University of Beirut Medical Centre
RECRUITING
Hamra
Chronic Care Center
RECRUITING
Hazmiyeh
Hospital Nini
RECRUITING
Tripoli
Nigeria
University of Abuja Teaching Hospital, Gwagwalada, Abuja
NOT_YET_RECRUITING
Abuja
University of Nigeria Teaching Hospital, Enugu
NOT_YET_RECRUITING
Enugu
University College Hospital Paediatric Haematology and Oncology Unit, Ibadan
NOT_YET_RECRUITING
Ibadan
Barau Dikko Teaching Hospital, Kaduna
NOT_YET_RECRUITING
Kaduna
Aminu Kano Teaching Hospital (AKTH)
NOT_YET_RECRUITING
Kano
Lagos University Teaching Hospital, Lagos
NOT_YET_RECRUITING
Lagos
Oman
Sultan Qaboos University Hospital
RECRUITING
Muscat
Saudi Arabia
Prince Mohammad Bin Naser Hospital
NOT_YET_RECRUITING
Jizan
King Khalid University Hospital
NOT_YET_RECRUITING
Riyadh
Spain
Hospital Universitario de Cruces
ACTIVE_NOT_RECRUITING
Barakaldo
Hospital Vall d'Hebron
RECRUITING
Barcelona
Hospital Universitario La Paz
RECRUITING
Madrid
Turkey
Baskent Universitesi Adana
NOT_YET_RECRUITING
Adana
Hacettepe University Hematology
NOT_YET_RECRUITING
Ankara
Mersin University Medical Faculty Hospital, Hematology
NOT_YET_RECRUITING
Mersin
Mersin University Medical Faculty Pediatric Hematology
NOT_YET_RECRUITING
Mersin
United Kingdom
Guy's Hospital
NOT_YET_RECRUITING
London
Imperial College London
RECRUITING
London
Kings College Hospital
NOT_YET_RECRUITING
London
Kings College Hospital
RECRUITING
London
Manchester Royal Infirmary_Manchester_0
RECRUITING
Manchester
Royal Hallamshire Hospital
RECRUITING
Sheffield
Contact Information
Primary
Novo Nordisk
clinicaltrials@novonordisk.com
(+1) 866-867-7178
Time Frame
Start Date: 2025-01-10
Estimated Completion Date: 2030-12-30
Participants
Target number of participants: 480
Treatments
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.
Sponsors
Leads: Novo Nordisk A/S

This content was sourced from clinicaltrials.gov