A research study looking at long-term treatment with etavopivat in people with sickle cell disease or thalassaemia

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novo Nordisk A/S

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

9 Jan 2025 → ongoing

Decision date (initial)

2024-11-15

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Novo Nordisk A/S

External identifiers

EU CT number

2024-510805-27-00

WHO UTN

U1111-1301-8130

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To investigate long-term safety of etavopivat in adults, adolescents and children with SCD, SCDTD, TDT or NTDT transferring from the Hibiscus, HibiscusKids, Hibiscus3 and Gladiolus studies

Secondary objectives 4

1. To investigate long-term clinical efficacy measures of etavopivat treatment in adults, adolescents and children with SCD transferring from the Hibiscus, HibiscusKids, Hibiscus3 and Gladiolus studies
2. To evaluate the effects of etavopivat on hospitalisations in adults, adolescents and children with SCD transferring from the Hibiscus, HibiscusKids, Hibiscus3 and Gladiolus studies
3. To investigate long-term clinical efficacy measures of etavopivat treatment in adults and adolescents with NTDT transferring from the Gladiolus study
4. To investigate long-term clinical efficacy measures of etavopivat treatment in adults and adolescents with TDT or SCDTD, transferring from the Gladiolus study

Conditions and MedDRA coding

Sickle Cell Disease and Thalassaemia

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002924-PIP02-23

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

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.
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 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 7

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 (>28 days or more) or ongoing temporary treatment discontinuation.
6. 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
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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

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 endpoints 8

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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novo Nordisk A/S

Sponsor organisation

Novo Nordisk A/S

Address

Novo Alle 1

City

Bagsvaerd

Postcode

2880

Country

Denmark

Scientific contact point

Organisation

Novo Nordisk A/S

Contact name

EU Submission Hub

Public contact point

Organisation

Novo Nordisk A/S

Contact name

EU Submission Hub

Third parties 4

Locations

5 EU/EEA countries · 15 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 34 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications