A Phase 3, Double-Blind, Randomized Trial Evaluating the Efficacy and Safety of Elritercept (TAK‑226) Compared to Placebo in Participants With Myelofibrosis and Anemia on Concurrent Ruxolitinib Therapy

Condition studied

Myelofibrosis

Investigational medicinal product(s)

Every trial of Elritercept and Ruxolitinib across our registers →

Eligibility

Primary endpoint

Proportion of participants who are RBC-TI for any consecutive ≥12-week period during the 36-week double-blinded treatment period.

Endpoint detail

1a. During the 36-week double-blinded treatment period: • Proportion of participants who achieve ≥50% reduction in RBC transfusion burden from baseline over any consecutive 12-week period. • Proportion of participants who are RBC-TI for any consecutive ≥16-week period, 1b. During the 36-week double-blinded treatment period: • Proportion of participants who are RBC-TI for any consecutive ≥12-week period with concurrent mean Hgb increase ≥1.5 g/dL from baseline., 2a. During the 36-week double-blinded treatment period: • Proportion of participants that have achieved anemia response. Anemia response is evaluated using multiple definitions, including:, 2b. RBC-TI for any consecutive 24-week period, RBC-TI for any consecutive 12-week period with a concurrent mean Hgb increase of ≥1.0 g/dL and ≥2.0 g/dL from baseline, RBC-TI for any consecutive 16- or 24-week period with a concurrent mean Hgb increase of ≥1.0 g/dL, ≥1.5 g/dL, and ≥2.0 g/dL from baseline., 2c. During the 36-week double-blinded treatment period: • Duration of RBC-TI ≥12 weeks. • Duration of RBC-TI ≥12 weeks with a concurrent mean Hgb increase of ≥1.5 g/dL from baseline. • Time to onset of anemia response., 3a. At 36 weeks: • Mean change in PROMIS Short Form v1.0 - Fatigue 7a T-score from baseline. • Mean change from baseline in EORTC QLQ-C30 fatigue scale score., 3b. During the 36-week double-blinded treatment period: • Proportion of participants with meaningful improvement and meaningful deterioration in PROMIS Short Form v1.0 - Fatigue 7a T-score., 3c. During the 36-week double-blinded treatment period: • Time to meaningful improvement and meaningful deterioration in PROMIS Short Form v1.0 - Fatigue 7a T-score from baseline. • Proportion of participants with meaningful improvement and meaningful deterioration in EORTC QLQ-C30 fatigue scale score., 3d. During the 36-week double-blinded treatment period: • Time to meaningful improvement and meaningful deterioration in EORTC QLQ-C30 fatigue scale score from baseline., 4a. At 36 weeks: • Mean changes in MFSAF v4.0 7-item Total Symptom Score from baseline., 4b. During the 36-week double-blinded treatment period: • Proportion of participants achieving confirmed MFSAF TSS50. • Time to symptom response in MFSAF TSS50 from baseline., 5a. At 36 weeks: • Mean change from baseline EORTC QLQ-C30 physical functioning scale score., 5b. During the 36-week double-blinded treatment period: • Proportion of participants with meaningful improvement and meaningful deterioration in EORTC QLQ-C30 physical functioning scale score. • Time to meaningful improvement and meaningful deterioration in EORTC QLQ-C30 physical functioning scale score from baseline., 6. Proportion of participants with platelet response e during the 36-week double-blinded treatment period., 7. Proportion of participants achieving SVR from baseline, as determined by blinded independent central review using computed tomography scan or magnetic resonance imaging during the 36-week double-blinded treatment period., 8. • Proportion of participants and time to progression in participants who develop AP MF. • Proportion of participants and time to progression in participants who develop BP MF., 9. Concentration-time data for participants treated with elritercept., 10. Proportion of participants treated with elritercept who have antidrug antibodies., 11. OS, defined as the time from randomization to the date of death due to any cause.