2022-502328-35-00Active, not recruitingPhase 2
Phase 2 Study of Luspatercept in Adults and Adolescents with Alpha (α)-thalassemia
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
Source: EU CTIS (euclinicaltrials.eu) · Last updated 25 Mar 2026
Brief summary (registry, in English)
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
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 41 (estimated)
- First submitted
- 20 Apr 2023
- Start date
- 30 Jan 2023
- Lead sponsor
- Celgene Corp.
- Diseases
- Thalassaemia
- EU CT number
- 2022-502328-35-00
- EudraCT number
- 2021-004928-15
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 41
- 9 sites in 2 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of EU CTIS (euclinicaltrials.eu). It describes the study design.
Who cannot take part
Copied verbatim from EU CTIS (euclinicaltrials.eu) and grouped by topic. Only the trial team decides who can take part.
Who the study is looking for
- 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.
Sex: All · Does not accept healthy volunteers
Sites (9)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Nosokomeio Paidon I Agia Sofia | Athens | Greece | Active, not recruiting |
| Hippokration Hospital | Athens | Greece | Active, not recruiting |
| General Hospital Of Larissa Koutlibaneio And Triantafylleio | Larissa | Greece | Active, not recruiting |
| General University Hospital Of Patras | Patras | Greece | Active, not recruiting |
| Ippokratio General Hospital of Thessaloniki | Thessaloniki | Greece | Active, not recruiting |
| Ente Ospedaliero Ospedali Galliera Di Genova | Genoa | Italy | Active, not recruiting |
| Azienda Ospedaliera Universitaria - Universita degli Studi della Campania Luigi Vanvitelli | Naples | Italy | Active, not recruiting |
| Azienda Ospedaliera di Rilievo Nazionale Antonio Cardarelli | Naples | Italy | Active, not recruiting |
| Azienda Ospedaliero-Universitaria San Luigi Gonzaga | Orbassano | Italy | Active, not recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.