Phase 2 Study of Luspatercept in Adults and Adolescents with Alpha (α)-thalassemia
Key facts
- Where
- Athens, Larissa, Patras és Thessaloniki, GörögországSee the 5 sites
- Phase
- 2. fázis
Where in Görögország
- Hippokration HospitalAthens, GörögországNo longer taking part
- Nosokomeio Paidon I Agia SofiaAthens, GörögországNo longer taking part
- General Hospital Of Larissa Koutlibaneio And TriantafylleioLarissa, GörögországNo longer taking part
- General University Hospital Of PatrasPatras, GörögországNo longer taking part
- Ippokratio General Hospital of ThessalonikiThessaloniki, GörögországNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Celgene Corp.
- Trial code
- EU 2022-502328-35-00
- Record updated
- 2026. március 25.
- Original description
Adult cohorts: To compare the erythroid response of luspatercept plus BSC vs placebo plus BSC on anemia in participants with A-THAL HbH disease. Adolescent cohorts: To confirm the recommended safe and tolerable dose of luspatercept in adolescent participants with A-THALRead full original descriptionShow less
A Phase 2 Study for the Treatment of Anemia with Alpha (α)-Thalassemia to Determine the Efficacy and Safety of Luspatercept (BMS-986346/ACE-536) in Adults and Evaluate the Safety and Pharmacokinetics in Adolescents
Adult cohorts: To compare the erythroid response of luspatercept plus BSC vs placebo plus BSC on anemia in participants with A-THAL HbH disease. Adolescent cohorts: To confirm the recommended safe and tolerable dose of luspatercept in adolescent participants with A-THAL
Inclusion: Adult participant ≥ 18 years with documented diagnosis of α-thalassemia HbH disease with Transfusion dependence defined as: - TD participant: ≥ 6 RBC units during the 24 weeks prior to randomization and no transfusion-free period for > 56 days during the 24 weeks prior to randomization - NTD participant: < 6 RBC units during the 24 weeks prior to randomization and, RBC transfusion-free during at least 8 weeks prior to randomization and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to randomization; hemoglobin values within 21 days post-transfusion will be excluded. Adolescent participant 12 years to < 18 years with documented diagnosis of α- thalassemia HbH disease with transfusion dependence defined as: - TD participant: ≥ 4 RBC events during the 24 weeks prior to enrollment and, no transfusion-free period for > 56 days during the 24 weeks prior to enrollment. Participants must have a history of regular transfusions for at least 2 years - NTD participant: < 4 RBC events during the 24 weeks prior to enrollment and RBC transfusion-free during at least 8 weeks prior to enrollment and, mean baseline Hb ≤ 10 g/dL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to enrollment, hemoglobin values within 21 days post-transfusion will be excluded. - Participant has Karnofsky (age ≥16 years) or Lansky (age < 16 years) performance status score ≥ 50 at screening.