| Protocol No: | ECCT/25/01/01 | Date of Protocol: | 03-05-2024 |
| Study Title: | 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. |
| Study Objectives: | To investigate long-term safety of etavopivat in adults, adolescents and children with SCD, SCDTD, TDT or NTDT transferring from other studies with etavopivat To investigate long-term clinical efficacy measures of etavopivat treatment in adults, adolescents and children with SCD transferring from other studies with etavopivatb To evaluate the effects of etavopivat on hospitalisations in adults, adolescents and children with SCD transferring from other studies with etavopivatb To investigate long-term clinical efficacy measures of etavopivat treatment in adults and adolescents with NTDT transferring from other studies with etavopivat To investigate long-term clinical efficacy measures of etavopivat treatment in adults and adolescents with TDT or SCDTD, transferring from other studies with etavopivat |
| Laymans Summary: |
A research study looking at long-term treatment with etavopivat in people with sickle cell disease or thalassaemia.
The purpose of this rollover study is to investigate the long-term safety of etavopivat in participants 11 months of age and older with SCD or thalassaemia who have completed a treatment period in previous etavopivat studies.
Long-term clinical efficacy measures of etavopivat treatment will also be assessed. This study will also ensure that participants who are benefiting from etavopivat treatment have prolonged access to the drug in the time before it is commercially available in their country.
Etavopivat is an orally bioavailable, small-molecule activator of pyruvate kinase red blood cell (PKR) isozyme. The investigational medicinal product (IMP) is being developed for the treatment of inherited haemoglobinopathies, such as Sickle cell disease (SCD) and thalassemia.
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| Abstract of Study: |
This is an interventional, multi-national, multi-centre, open-label, phase 3b study in adults (participants ≥ 18 years old), adolescents (participants ≥ 12 to < 18 years old) and children(participants ≥ 11 months to < 12 years old) with sickle cell disease (SCD), with SCD who have been on chronic transfusions to prevent primary stroke or recurrence of stroke (SCDTD), non-transfusion-dependent thalassaemia (NTDT) or transfusion-dependent thalassaemia (TDT).
The purpose of this rollover study is to investigate the long-term safety of etavopivat in participants 11 months of age and older with SCD or thalassaemia who have completed a treatment period in previous etavopivat studies. Long-term clinical efficacy measures of etavopivat treatment will also be assessed.
Etavopivat is an orally bioavailable, selective activator of erythrocyte pyruvate kinase (PKR), currently under development in clinical studies as a potential treatment for inherited haemoglobinopathies, including SCD and thalassemia
Approximately 325 participants are planned to be enrolled in the study in about 120 sites worldwide. All participants will receive etavopivat. Participants ≥ 12 years old will receive an oral dose of 400 mg QD administered as 2 × 200 mg tablets. Participants < 12 years old will receive an age- and weight-adjusted dose (400 mg QD equivalent dose) administered as granules
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