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

ClinicalTrials.gov processed this data on October 8, 2024. Link to the current ClinicalTrials.gov record.

Recruitment Status

NOT YET RECRUITING (See Contacts and Locations)
Verified October 2024 by Novo Nordisk A/S

Sponsor

Novo Nordisk A/S

Information Provided by (Responsible Party)

Novo Nordisk A/S

Clinicaltrials.gov Identifier

NCT06609226
Other Study ID Numbers: NN7535-7822
First Submitted: September 20, 2024
First Posted: September 24, 2024
Last Update Posted: October 9, 2024
Last Verified: October 2024
History of Changes

Listing a study on this site does not mean it has been evaluated by the U.S. Federal Government. The safety and scientific validity of a study listed on ClinicalTrials.gov is the responsibility of the study sponsor and investigators. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating.

ClinicalTrials.gov, a resource provided by the U.S. National Library of Medicine (NLM), is a registry and results information database of clinical research studies sponsored or funded by a broad range of public and private organizations around the world. Not all studies listed on ClinicalTrials.gov are funded by the National Institutes of Health (NIH) or other agencies of the U.S. Federal Government. Not all listed studies are regulated and/or reviewed by the U.S. Food and Drug Administration or other governmental entities.

Information on ClinicalTrials.gov is provided by study sponsors and investigators, and they are responsible for ensuring that the studies follow all applicable laws and regulations. NLM staff do not verify the scientific validity or relevance of the submitted information beyond a limited quality control review for apparent errors, deficiencies, or inconsistencies.

Choosing to participate in a study is an important personal decision. Before you participate in a study, discuss all options with your health care provider and other trusted advisors. For more information about participating in clinical studies, see Learn About Clinical Studies, which includes questions that you might want to ask before deciding to participate in a study.

For more information about using the information on ClinicalTrials.gov, please also see Terms and Conditions.

See also the Web Policies and Notices for the NIH web site.

Study Description

Not Provided
Condition or Disease Intervention/Treatment
  • Sickle Cell Disease, Thalassemia
  • Drug: Etavopivat A
  • Drug: Etavopivat B

Study Design

Study TypeInterventional
Anticipated Enrollment325 participants
Design AllocationNon-Randomized
Interventional ModelParallel Assignment
MaskingNone (Open Label)
Primary PurposeTreatment
Official TitleAn 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
Study Start DateDecember 9, 2024
Anticipated Primary Completion DateNovember 30, 2029
Anticipated Study Completion DateNovember 30, 2029

Groups and Cohorts

Group/ CohortIntervention/ Treatment
  • Participants greater than or equal to (≥) 12 years old with sickle cell disease
    • Participants will receive an oral dose of Etavopivat A.
  • Drug: Etavopivat A
    • Participants will receive an oral dose of Etavopivat A.
  • Participants ≥ 12 years old with sickle cell disease on chronic red blood cell (RBC) transfusions
    • Participants will receive an oral dose of Etavopivat A.
  • Drug: Etavopivat A
    • Participants will receive an oral dose of Etavopivat A.
  • Participants ≥ 12 years old with transfusion-dependent thalassaemia
    • Participants will receive an oral dose of Etavopivat A.
  • Drug: Etavopivat A
    • Participants will receive an oral dose of Etavopivat A.
  • Participants ≥ 12 years old with non-transfusion dependent thalassaemia
    • Participants will receive an oral dose of Etavopivat A.
  • Drug: Etavopivat A
    • Participants will receive an oral dose of Etavopivat A.
  • Participants ≥ 11 months to less than (<) 12 years old with sickle cell disease
    • Participants ≥ 12 years of age will receive an oral dose of Etavopivat A and participants < 12 years of age will receive an oral dose of Etavopivat B.
  • Drug: Etavopivat A
    • Participants will receive an oral dose of Etavopivat A.
  • Drug: Etavopivat B

    Outcome Measures

    Primary Outcome Measures

    1. Number of treatment emergent adverse events (TEAEs), reported for each indication and age group separately [Baseline (week 0 of FLORAL) to end of study (week 264, or earlier)]
      Measured as number of events.
    2. Number of adverse reactions, reported for each indication and age group separately [Baseline (week 0 of FLORAL) to end of study (week 264, or earlier)]
      Measured as number of adverse reactions.

    Secondary Outcome Measures

    1. Annualised vaso-occlusive crisis (VOC) rates, reported for each age group separately [Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier]
      Measured as count.
    2. Change in VOCs, reported for each age group separately [Baseline (of parent study) to end of treatment at week 260, or earlier]
      Measured as count.
    3. Change in hemoglobin (Hb) concentration, reported for each age group separately [Baseline (of parent study) to end of treatment at week 260, or earlier]
      Measured as grams per deciliter (g/dL).
    4. Annualised number of hospitalisations, reported for each age group separately [Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier]
      Measured as count.
    5. Average length of stay of hospitalisations, reported for each age group separately [Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier]
      Measured as days.
    6. Change in Hb concentration [Baseline (of parent study) to end of treatment at week 260, or earlier]
      Measured as g/dL.
    7. Number of red blood cell (RBC) units transfused, reported for each indication separately [Baseline (week 0 of FLORAL) to end of treatment at week 260, or earlier]
      Measured as units.
    8. Change in RBC units transfused, reported for each indication separately [Baseline (of parent study) to end of treatment at week 260, or earlier]
      Measured as units.

    Eligibility Criteria

    Ages Eligible for Study (Child, Adult, Older Adult)
    Sexes Eligible for Study All
    Accepts Healthy Volunteers No
    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 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.
    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 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.

    Contacts and Locations

    Sponsors and Collaborators Novo Nordisk A/S
    Locations
    • Univ of Alabama Birmingham | Birmingham, Alabama, United States, 35233
    • Phoenix Children's Hsptl | Phoenix, Arizona, United States, 85016
    • University Of California Irvine | Irvine, California, United States, 92697
    • Children's Hospital Los Angeles - Endocrinology | Los Angeles, California, United States, 90027
    • UCSF Oakland Benioff ChildHosp | Oakland, California, United States, 94609
    • Children's Hosp Of Orange | Orange, California, United States, 92868
    • University of Connecticut | Farmington, Connecticut, United States, 06030
    • Children's National Medical Center | Washington, District of Columbia, United States, 20010
    • Univ Miami-Miller School Med | Miami, Florida, United States, 33136
    • Emory University School of Medicine | Atlanta, Georgia, United States, 30303
    • Children's Healthcare Atlanta | Atlanta, Georgia, United States, 30342
    • Children's Hosp-New Orleans | New Orleans, Louisiana, United States, 70118
    • Boston Medical Center | Boston, Massachusetts, United States, 02118
    • Jacobi Medical Center | Bronx, New York, United States, 10461
    • Montefiore Medical Center | Bronx, New York, United States, 10467
    • NYC Health+Hospitals | Brooklyn, New York, United States, 11203
    • Columbia University Medical Center_New York_0 | New York, New York, United States, 10032
    • Weill Cornell Med Coll-NYPH | New York, New York, United States, 10065
    • Duke University_Durham | Durham, North Carolina, United States, 27705
    • East Carolina University_Greenville | Greenville, North Carolina, United States, 27834
    • Atrium Health-Wake Forest Bapt | Winston-Salem, North Carolina, United States, 27157
    • Cincinnati Child's Hsp Med Ctr | Cincinnati, Ohio, United States, 45229
    • Neuro-Behavioral Clinical Research | North Canton, Ohio, United States, 44720
    • Medical University Of South Carolina_Charleston | Charleston, South Carolina, United States, 29425
    • East Carolina University_Greenville_0 | Greenville, South Carolina, United States, 27858
    • UTHSC-Memphis | Memphis, Tennessee, United States, 38104
    • UT Health University of Texas | Houston, Texas, United States, 77030
    • Virginia Comm Univ Medical Ctr | Richmond, Virginia, United States, 23298
    • Mary Bridge Children's Health | Tacoma, Washington, United States, 98405
    • Providence Hematolgy | Vancouver, British Columbia, Canada, V6E 1M7
    • The Hospital for Sick Children | Toronto, Ontario, Canada, M5G 1X8
    • University Health Network - Toronto General Hospital | Toronto, Ontario, Canada, M5G 2C4
    • CHU Ste-Justine | Montreal, Quebec, Canada, H3T 1C5
    • Ap-Hp-Hopital Henri Mondor | Créteil, France, 94000
    • Hospices Civils de Lyon-Hopital Edouard Herriot | Lyon Cedex 03, France, 69437
    • Ap-Hp-Hopital Robert Debre | Paris, France, 75019
    • Charité Campus Virchow Klinikum - Klinik für Pädiatrie mit Schwerpunkt Onkologie und Hämatologie | Berlin, Germany, 13353
    • Universitätsklinikum Freiburg, Kinder- und Jugendklinik | Freiburg, Germany, 79106
    • General Hospital Of Larissa Koutlibaneio And Triantafylleio | Larissa, Thessaly, Greece, 412 21
    • Hippokration Hospital | Athens, Greece, 11527
    • General University Hospital of Patras | Patra, Greece, 26504
    • 'Ippokrateio' General Hospital of Thessaloniki | Thessaloniki, Greece, 54642
    • Azienda Ospedaliera Universitaria San Luigi Gonzaga | Orbassano, Torino, Italy, 10043
    • Azienda Ospedale Universita Padova | Padova, Italy, 35128
    • Fondazione IRCCS Policlinico San Matteo | Pavia, Italy, 27100
    • American University of Beirut Medical Centre | Hamra, Lebanon,
    • Chronic Care Center | Hazmieh, Lebanon, 21211
    • Hospital Nini | Tripoli, Lebanon, 1434
    • Sultan Qaboos University Hospital | Muscat, Oman, 123
    • Prince Mohammad Bin Naser Hospital | Jizan, Saudi Arabia, 82943
    • King Khalid University Hospital | Riyadh, Saudi Arabia, 12372
    • Hospital Universitario de Cruces | Baracaldo, Spain, 48903
    • Hospital Vall d'Hebron | Barcelona, Spain, 08035
    • Hospital Universitario La Paz | Madrid, Spain, 28046
    • Baskent Universitesi Adana | Adana, Turkey, 01250
    • Hacettepe University Hematology | Ankara, Turkey, 06230
    • Mersin University Medical Faculty Hospital, Hematology | Mersin, Turkey, 33110
    • Mersin University Medical Faculty Pediatric Hematology | Mersin, Turkey, 33110
    Investigators

      More Information

      Additional Relevant MeSH Terms

      • Anemia, Sickle Cell
      • Thalassemia
      • Anemia, Hemolytic, Congenital
      • Anemia, Hemolytic
      • Anemia
      • Hematologic Diseases
      • Hemoglobinopathies
      • Genetic Diseases, Inborn