Recruiting

Trial ID CA056-015 | NCT05664737

A Study to Determine the Efficacy and Safety of Luspatercept in Adult and Adolescent Participants With Alpha (α)-Thalassemia

About This Trial
Key Eligibility Criteria
Trial Site Locations

Summary

Phase 2
12+

Age Range
4

Locations
Recruiting

Overview

The purpose of the study is to evaluate the efficacy and safety of luspatercept plus best supportive care (BSC) vs placebo plus BSC on anemia in adult participants with α-thalassemia hemoglobin H (HbH) disease and determine the safety and drug levels in adolescent participants.

Nearest Recruiting Site

Key Eligibility Criteria

Key

Inclusion Criteria

Adult participant ≥ 18 years with documented diagnosis of α-thalassemia HbH disease with Transfusion dependence for TD participants defined as ≥ 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.
Adult participant ≥ 18 years with documented diagnosis of α-thalassemia HbH disease with Transfusion dependence for NTD participants defined as < 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 for TD participants defined as ≥ 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.
Adolescent participant 12 years to < 18 years with documented diagnosis of α-thalassemia HbH disease with Transfusion dependence for NTD participants defined as < 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.
Adolescent participant has Karnofsky (age ≥16 years) or Lansky (age < 16 years) performance status score ≥ 50 at screening. Key

Exclusion Criteria

Participants must not have a 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.
Participants must not have anemia related to nutritional deficiency, anemia of chronic disease, autoimmune hemolytic anemia, or any other hemolytic anemias.
Participants must not have bleeding disorders manifested by frequent bleeding episodes.
Participants must not have undergone episodes of hemolysis not related to alpha-thalassemia within the 8 weeks prior to randomization.
Participants must not have prior exposure to gene therapy to treat α-thalassemia.
Participants must not have undergone hematopoietic stem cell transplantation (HSCT) Note: Other protocol-defined inclusion/exclusion criteria apply.

Additional Information *

Treatment Options

Study Arms

ASSIGNED INTERVENTION

Study Arms

Placebo Comparator: Adult NTD Cohort: Placebo + BSC

ASSIGNED INTERVENTION

Drug: Placebo

Study Arms

Placebo Comparator: Adult TD Cohort: Placebo + BSC

ASSIGNED INTERVENTION

Drug: Placebo

Study Arms

Experimental: Non-transfusion Dependent (NTD): Luspatercept + BSC

ASSIGNED INTERVENTION

Biological: Luspatercept

Study Arms

Experimental: Transfusion Dependent (TD): Luspatercept + Best supportive care (BSC)

ASSIGNED INTERVENTION

Biological: Luspatercept

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