ETAVOPIVAT for Sickle cell disease | Thalassaemia

Inclusion criteria

• {"criterion_text":"- 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."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Participant must have derived clinical benefit from treatment with etavopivat, as determined by the investigator."}
• {"criterion_text":"- Any participant with dose reduction or temporary discontinuation will need to be rechallenged before transferring."}
• {"criterion_text":"- 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 patient’s overall health status."}

Exclusion criteria

• {"criterion_text":"- Previous participation in this study. Participation is defined as signed informed consent."}
• {"criterion_text":"- 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)."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Participant withdrew or had permanent treatment discontinuation from an etavopivat clinical study."}
• {"criterion_text":"- Participants on permanent treatment dose reduction (>28 days or more) or ongoing temporary treatment discontinuation."}
• {"criterion_text":"- Use of any of the following within the timeframes prior to the transfer visit as stated: a) Use of hemoglobin S (HbS) polymerization inhibitors 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 for more than 4 consecutive weeks during the parent study or anticipated need of such agent for a maintenance treatment 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. For guidance on strong inducers of CYP 3A4, see Section 6.8"}
• {"criterion_text":"- 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"}

Primary endpoints

• {"endpoint_text":"- Number of TEAEs, reported for each indication and age group separately","definition_or_measurement_approach":"Reported for each indication and age group separately"}
• {"endpoint_text":"- Number of adverse reactions, reported for each indication and age group separately","definition_or_measurement_approach":"Reported for each indication and age group separately"}

Secondary endpoints

• {"endpoint_text":"- Annualised VOC rates, reported for each age group separately","definition_or_measurement_approach":"Reported for each age group separately"}
• {"endpoint_text":"- Change in VOCs, reported for each age group separately","definition_or_measurement_approach":"Reported for each age group separately"}
• {"endpoint_text":"- Change in Hb concentration, reported for each age group separately","definition_or_measurement_approach":"Reported for each age group separately"}
• {"endpoint_text":"- Annualised number of hospitalisations, reported for each age group separately","definition_or_measurement_approach":"Reported for each age group separately"}
• {"endpoint_text":"- Average length of stay of hospitalisations, reported for each age group separately","definition_or_measurement_approach":"Reported for each age group separately"}
• {"endpoint_text":"- Change in Hb concentration","definition_or_measurement_approach":""}
• {"endpoint_text":"- Number of RBC units transfused, reported for each indication separately","definition_or_measurement_approach":"Reported for each indication separately"}
• {"endpoint_text":"- Change in RBC units transfused, reported for each indication separately","definition_or_measurement_approach":"Reported for each indication separately"}

Spain

Earliest CTIS Part Ii Submission Date

22-10-2024

Latest Decision Or Authorization Date

24-02-2026

Processing Time Days

490

Number Of Sites

3

Number Of Participants

10

Germany

Earliest CTIS Part Ii Submission Date

27-09-2024

Latest Decision Or Authorization Date

24-02-2026

Processing Time Days

515

Number Of Sites

2

Number Of Participants

2

Greece

Earliest CTIS Part Ii Submission Date

21-08-2024

Latest Decision Or Authorization Date

24-02-2026

Processing Time Days

552

Number Of Sites

4

Number Of Participants

16

Italy

Earliest CTIS Part Ii Submission Date

24-10-2024

Latest Decision Or Authorization Date

17-03-2026

Processing Time Days

509

Number Of Sites

3

Number Of Participants

5

France

Earliest CTIS Part Ii Submission Date

12-11-2024

Latest Decision Or Authorization Date

25-02-2026

Processing Time Days

470

Number Of Sites

3

Number Of Participants

11

Primary sponsor

Full Name

Novo Nordisk A/S

Organisation Type

Pharmaceutical company

Country Of Registered Address

Denmark

Contract research organisations

Name

4G Clinical B.V.

Responsibilities

RTSM supplier and RTSM Helpdesk

Name

Icon Clinical Research Limited

Responsibilities

iRIS data entry service

Name

Oracle Danmark ApS

Responsibilities

CRF Supplier

Third parties

• {"country":"Denmark","full_name":"Oracle Danmark ApS","duties_or_roles":"CRF Supplier","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Netherlands","full_name":"4G Clinical B.V.","duties_or_roles":"RTSM supplier and RTSM Helpdesk","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Greece","full_name":"Affidea Piraeus Biopathological","duties_or_roles":"Health care,Hospital/Clinic/Other health care facility","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"iRIS data entry service","organisation_type":"Pharmaceutical company"}