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Accepting PatientsPhase 3 (Comparison)NCT06609226

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

Sickle Cell Disease, Thalassemia

Phase:Phase 3 (Comparison)
Status:Accepting Patients
Age:2+
Gender:All
Locations:106 sites

What This Study Is About

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.

Who Can Participate

You May Qualify If
  • 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.
You Cannot Join If
  • 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.
  • +4 more...
Key Requirements
  • Able to provide informed consent
View Full Eligibility Criteria
Full Criteria (from ClinicalTrials.gov)

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.

Important: This is a summary. The final determination of eligibility will be made by the trial coordinator after reviewing your complete medical history.

Study Locations

Univ of Alabama Birmingham — Birmingham, Alabama
Phoenix Children's Hsptl — Phoenix, Arizona
Children's Hospital Los Angeles - Endocrinology — Los Angeles, California
Show all 106 locations
Children's Hospital Los Angeles - Endocrinology — Los Angeles, California
UCSF Oakland Benioff ChildHosp — Oakland, California
UCSF Oakland Benioff ChildHosp — Oakland, California
Children's Hosp Of Orange — Orange, California
University Of California Irvine — Orange, California
University of Connecticut — Farmington, Connecticut
Children's National Medical Center — Washington D.C., District of Columbia
Foundation for Sickle Cell Disease Research — Hollywood, Florida
Univ of Miami/SCCC — Miami, Florida
Emory University School of Medicine — Atlanta, Georgia
Children's Healthcare Atlanta — Atlanta, Georgia
Center for Blood Disorders Augusta University — Augusta, Georgia
Univer Of Illinois at Chicago — Chicago, Illinois
Children's Hosp-New Orleans — New Orleans, Louisiana
Boston Medical Center — Boston, Massachusetts
Washington University-St.Louis — St Louis, Missouri
NYC Health+Hospitals — Brooklyn, New York
Columbia University Medical Center_New York_0 — New York, New York
Columbia University Medical Center_New York_0 — New York, New York
Weill Cornell Med Coll-NYPH — New York, New York
Jacobi Medical Center — The Bronx, New York
Montefiore Medical Center — The Bronx, New York
Atrium Levine Children's/Atrium Health — Charlotte, North Carolina
Duke University_Durham — Durham, North Carolina
East Carolina Univ-Greenville — Greenville, North Carolina
East Carolina University_Greenville — Greenville, North Carolina
Atrium Health-Wake Forest Bapt — Winston-Salem, North Carolina
Cincinnati Child's Hsp Med Ctr — Cincinnati, Ohio
Neuro-Behavioral Clinical Research — North Canton, Ohio
Medical University Of South Carolina_Charleston — Charleston, South Carolina
Methodist University Hospital — Memphis, Tennessee
Texas Children's Hospital_Houston — Houston, Texas
UT Health University of Texas — Houston, Texas
Virginia Comm Univ Medical Ctr — Richmond, Virginia
Mary Bridge Children's Health — Tacoma, Washington
Versiti, CCBD_Milwaukee — Milwaukee, Wisconsin
St Pauls Hospital — Vancouver, British Columbia
The Hospital for Sick Children — Toronto, Ontario
University Health Network - Toronto General Hospital — Toronto, Ontario
CHU Ste-Justine — Montreal, Quebec
Alexandria University Hospital — Alexandria, Egypt
Zagazig University Hospital — Alsharkia, Egypt
Faculty of Medicine Ain Shams Medical Research Institute (MASRI) — Cairo
Cairo University — Cairo, Egypt
Abu El-Reesh El-Mounira Children University Hospital — Cairo, Egypt
Ap-Hp-Hopital Henri Mondor — Créteil
Hospices Civils de Lyon-Hopital Edouard Herriot — Lyon
Ap-Hp-Hopital Robert Debre — Paris
Ap-Hp-Hopital Robert Debre — Paris
Charité - Campus Virchow-Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie — Berlin
Universitätsklinikum Freiburg - Kinder- und Jugendklinik — Freiburg im Breisgau
Kintampo Health Research Centre (KHRC) — Kintampo, Bono East
Ghana Institute of Clinical Genetics, Korle Bu Teaching Hospital (KBTH) — Accra
General Hospital Of Larissa Koutlibaneio And Triantafylleio - Thalassemia and SCD Unit — Larissa, Thessaly
Hippokration Hospital — Athens
General University Hospital of Patras — Pátrai
'Ippokrateio' General Hospital of Thessaloniki — Thessaloniki
All India Institute of Medical Sciences (AIIMS), Raipur — Raipur, Chhattisgarh
K.J Somaiya Hospital and Research Centre — Mumbai, Maharashtra
Victoria Hospital, Bangalore — Bangalore
Nirmal Hospital Pvt. Ltd. — Gujarat
Suretech Hospital and Research Centre Ltd. — Maharashtra
All India Institute of Medical Sciences_Delhi — New Delhi
Azienda Ospedaliera Universitaria San Luigi Gonzaga - S.C.D.O. Microcitemie e malattie rare ematologiche — Orbassano, Torino
Azienda Ospedale Universita Padova — Padova
Fondazione IRCCS Policlinico San Matteo — Pavia
Gertrude's Children's Hospital — Nairobi, Nairobi County
KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital — Siaya, Siaya County
KEMRI-Walter-Reed Kericho — Kericho
Kombewa Clinical Research Centre — Kisumu
Kombewa Clinical Research Centre — Kisumu
KEMRI Kondele Children Hospital, Kisumu — Kisumu
Ahero Clinical Trials Unit — Kisumu
American University of Beirut Medical Centre — Hamra
Chronic Care Center — Hazmiyeh
Hospital Nini — Tripoli
University of Nigeria Teaching Hospital (UNTH) — Ituku-Ozalla, Enugu State
University College Hospital Paediatric Haematology and Oncology Unit, Ibadan — Ibadan, Oyo State
University of Abuja Teaching Hospital, Gwagwalada, Abuja — Abuja
Barau Dikko Teaching Hospital, Kaduna — Kaduna
Aminu Kano Teaching Hospital (AKTH) — Kano
Lagos University Teaching Hospital, Lagos — Lagos
Sultan Qaboos University Hospital — Muscat, Sultanet of Oman/Muscat/Al Khoud
Prince Mohammad Bin Naser Hospital — Jizan
King Khalid University Hospital — Riyadh
Hospital Universitario de Cruces — Barakaldo
Hospital Vall d'Hebron — Barcelona
Hospital Universitario La Paz — Madrid
Baskent Universitesi Adana — Adana
Başkent Üniversitesi Adana-Hematoloji — Adana
Hacettepe University Hematology — Ankara
Hacettepe Üniversitesi Hastanesi- Hematoloji — Ankara
Mersin University Medical Faculty Pediatric Hematology — Mersin
Mersin Üniversitesi Tip Fakültesi Hastanesi- Çiftlikköy Yerleşkesi- Hematoloji — Mersin
VM Medical Park Mersin Hastanesi- Hematoloji — Mersin
Evelina London Children's Hospital - Haemophilia — London
Guy's Hospital - Haematology — London
Guy's Hospital — London
King's College Hospital - Paediatric Research — London
Kings College Hospital - Haematology — London
Imperial College London — London
Manchester Royal Infirmary — Manchester
Royal Hallamshire Hospital — Sheffield

Contact

Contact

Novo Nordisk

Experimental Treatment

This trial is testing a new treatment. Results are not guaranteed. Talk to your doctor before applying.

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