Haematological Disorders
Conditions
Interventions
Sponsors
Novo Nordisk
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Informed consent obtained before any study-related activities. Study-related activities are any procedures that are carried out as part of the study, including activities to determine suitability for the study. United Kingdom: Please see country-specific requirements in Appendix 8 2. Participant must have ongoing participation in an etavopivat parent study (Table 4-1) for treatment of SCD or thalassaemia and have completed at least a treatment period of the parent study. 3. Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator. 4. Any participant with dose reduction or temporary discontinuation will need to be rechallenged before transferring. 5. Participants on hydroxyurea (HU), crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they: a. 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). b. Have been compliant with the treatment regimen at the discretion of the investigator during participation of the parent study.
Exclusion criteria
Exclusion criteria: 1. Previous participation in this study. Participation is defined as signed informed consent. 2. Female who is pregnant or intends to become pregnant or is of childbearing potential and not using adequate contraceptive method, as defined in Appendix 4 (Section 10.4). 3. 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. 4. Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study. 5. Participants on permanent treatment dose reduction or temporary treatment discontinuation. 6. Use of any of the following within the timeframes prior to the transfer visit as stated: a. Use of voxelotor within participation of the parent study or anticipated need for this agent during this study. b. 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. c. Use of erythropoietin or other haematopoietic growth factor treatment within the parent study or anticipated need for such agents during this study. d. 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. 7. 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Number of TEAEs, reported for each indication and age group separately 2. Number of adverse reactions, reported for each indication and age group separately | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Annualised VOC rates, reported for each age group separately 2. Change in VOCs, reported for each age group separately 3. Change in Hb concentration, reported for each age group separately 4. Annualised number of hospitalisations, reported for each age group separately 5. Average length of stay of hospitalisations, reported for each age group separately 6. Change in Hb concentration 7. Number of RBC units transfused, reported for each indication separately 8. Change in RBC units transfused, reported for each indication separately | — |
Countries
Ghana, Kenya, Nigeria
Contacts
Public ContactMichell Botha
Assc Dr Global site Activation
Outcome results
None listed