Authorised Therapeutic exploratory (Phase II) Alpha (α)-thalassemia

Phase 2 Study of Luspatercept in Adults and Adolescents with Alpha (α)-thalassemia

EU CTIS ID: 2022-502328-35-00

What this study is testing

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

  • Therapeutic exploratory (Phase II)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • 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.

You likely can't join if

  • Key Exclusion Criteria - Adult and Adolescent participants: - Medical Conditions: Diagnosis of α-thalassemia Trait, Hb Bart hydrops, ATRx α-thalassemia, hemoglobin S/β-thalassemia, myelodysplasia subtype anemia, or with HbE homozygous beta gene mutation. Anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias. Bleeding disorders manifested by frequent bleeding episodes. Undergone episodes of hemolysis not related to α-thalassemia within the 8 weeks prior to randomization. - Reproductive Status: Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding. - Prior/Concomitant Therapy: Prior exposure to gene therapy to treat α-thalassemia. Undergone hematopoietic stem cell transplantation (HSCT)

The study team makes the final eligibility decision.

Where it's taking place

  • Thailand
  • China
  • Turkey
  • Taiwan
  • Hong Kong

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 18-64 years, 0-17 years, 65+ years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Thailand; China; Turkey; Taiwan; Hong Kong. Enter your location above to see the nearest site and check your eligibility.

Explore other conditions

BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.