For physicians

Do you have a patient with thalassemia or sickle cell disease (SCD)?

The primary purpose of the Gladiolus Study is to evaluate the safety and efficacy of an investigational medication in people 12 to 65 years of age with thalassemia or SCD, as measured by improvement in anemia (Hb) and reduction in red blood cell (RBC) transfusions.

Study design

This is a Phase 2, open-label study in which up to 60 participants will be enrolled to receive 400 mg of etavopivat, which is taken as 2 tablets once a day. The total duration of the study for each participant is up to 58 weeks. This includes screening (up to 6 weeks), open-label study treatment (up to 48 weeks), and an end-of-study visit 4 weeks after the last dose of the investigational medication.

The study is enrolling 3 groups of patients:

Cohort A

Up to 20 patients with SCD on chronic RBC transfusion therapy to prevent stroke or recurrence of stroke.

Cohort B

Up to 20 patients with thalassemia on chronic RBC transfusion therapy to prevent symptomatic anemia.

Cohort C

Up to 20 patients with thalassemia not receiving chronic RBC transfusion therapy.

Key eligibility criteria

All cohorts

12 to 65 years of age (inclusive) at the time of first dose.

Cohort A (SCD transfusion cohort)

Study patients living with a confirmed diagnosis of SCD (all genotypes).
At least 24 months of chronic, monthly RBC transfusions by straight transfusions for primary stroke prevention or due to previous stroke. Chronic RBC transfusion is defined as: ≥ 6 RBC units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for > 35 days during that period.
On iron chelation therapy for > 3 months prior to enrollment.

Cohort B (thalassemia transfusion cohort)

Study patients living with documented diagnosis of β-thalassemia, hemoglobin E/β-thalassemia, or hemoglobin H (α-thalassemia).
Chronically and regularly transfused, defined as: ≥ 6 RBC units in the previous 24 weeks before the first dose of study treatment and no transfusion-free period for > 35 days during that period.
On iron chelation therapy for > 3 months prior to enrollment.

Cohort C (thalassemia non-transfused cohort)