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

Phase 1

Recruiting

• Conditions: Alpha (a)-thalassemia; MedDRA version: 20.1Level: LLTClassification code: 10054659Term: Thalassemia alpha Class: 10010331; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2022-502328-35-00
• Lead Sponsor: Celgene Corp.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-04-20
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 177

Inclusion Criteria

Adult participant = 18 years with documented diagnosis of a-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 a- 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.

Exclusion Criteria

Key Exclusion Criteria - Adult and Adolescent participants: - Medical Conditions: Diagnosis of a-thalassemia Trait, Hb Bart hydrops, ATRx a-thalassemia, hemoglobin S/ß-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 a-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 a-thalassemia. Undergone hematopoietic stem cell transplantation (HSCT)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified