Share Icon Health Study Area: Cardiovascular Disease Chevron Icon Health Study Area: Genitourinary For Patients Health Study Area: Lung Cancer Page Icon Phone Icon For Caregivers Health Study Area: AutoImmune Disease Health Study Area: Melanoma Location Icon Print YouTube Icon For Parents Health Study Area: Lung Cancer Print Created with Sketch. Help Icon Green Check Icon Search Icon Instagram Created with sketchtool. Direction Arrow Icon Error Icon For Parents Health Study Area: Blood Cancer Help Icon Health Study Area: NASH Gender Both Bookmark Icon Health Study Area: Melanoma Created with Sketch. Glossary Print Health Study Area: Blood Cancer Health Study Area: Genitourinary Health Study Area: Gastrointestinal Cancer Mobile Menu Icon Created with Sketch. Health Study Area: Cardiovascular Disease Health Study Area: Women's Cancer Communities Map Icon Created with Sketch. For Caregivers Health Study Area: Fibrosis Health Study Area: AutoImmune Disease FAQs Health Study Area: Head and Neck Cancer Created with Sketch. For Clinicians Chevron Right Icon Gender Female Health Study Area: Breast Cancer Direction Arrow Icon Gender Both Right Arrow Icon LinkedIn Icon Green Check Icon Gender Male Health Study Area: Fibrosis For Patients Twitter Icon Email Icon Facebook Icon Health Study Area: Gastrointestinal Cancer Health Study Area: Head and Neck Cancer For Clinicians External Link Icon

Menu

Search BMS Clinical Trials
BMS Recruiting Study icon

Recruiting

Trial ID CA056-015  |   NCT05664737

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

Summary

  • Phase 2
  • Male and Female Gender icon
  • 12+
    Age Range
  • 1
    Locations
  • BMS Recruiting Study icon
    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.

print Print
email Email

Nearest Recruiting Site

Key Eligibility Criteria

Key

Inclusion Criteria

Inclusion Criteria Icon img
:
  • Adult participant≥ 18 years with documented diagnosis of A-Thal HbH disease with Transfusion dependence defined as:. 1. TD participant: ≥ 6 RBC units during the 24 weeks prior to randomization. 2. NTD participant:< 6 RBC units during the 24 weeks prior to randomization(transfusion due to conditions other than A-Thal will not be considered)and, RBC transfusion-free during at least 8 weeks prior to randomization(unless transfusion was required to treat an acute medical condition other than A-Thal) 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.
    1. Adult participant has Eastern Cooperative Oncology Group (ECOG) 34 score of 0 or 1.
      1. Adolescent participant 12 years to < 18 years with documented diagnosis of A-Thal HbH disease with transfusion dependence defined as:. 1. 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. 2. 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. 3. Participant has Karnofsky (age ≥16 years) or Lansky (age < 16 years) performance status score ≥ 50 at screening. Key

        Exclusion Criteria

        Exclusion Criteria Icon
        :
        • Medical Conditions: Diagnosis of A-ThalTrait, Hb Bart hydrops, ATRx A-Thal, 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. Undergone episodes of hemolysis not related to A-Thal within the 8 weeks prior to randomization.
          1. Participant has deep vein thrombosis (DVT), stroke or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24weeks prior to randomization.
            1. Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered: blood pressure value corresponding to ≤Grade 1 according to NCI CTCAE Version 5.0. with or without pharmacological treatment.
              1. Reproductive Status: Women who are pregnant, plan to get pregnant during the study, or who are breastfeeding.
                1. Prior/Concomitant: Undergone HSCTs or gene therapy (candidates for HSCT or gene therapy with waiting period of ≥ 12 months are eligible).
                  1. Use of hydroxyurea treatment ≤ 12 weeks prior to enrollment for NTD participants and ≤ 24 weeks for TD participants.
                    1. Participant who has extramedullary hematopoiesis (EMH) complications requiring treatment to control the growth of EMH mass(es) during the screening period.
                      1. Any medical or psychiatric condition (including active infections, recent surgery, sequelae of diseases or interventions, clinically significant laboratory abnormalities or concurrent treatment) that in the opinion of the investigator would put the participant at unacceptable risk of participating in the study or that could affect interpretability of data.
                        1. 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
                          Please select a study site to register
                          Select a Study Site Location
                          Select a study site location that is convenient for you
                          Register
                          Provide your contact details for the study site to connect with you.

                          Help us enhance your website experience! Share your feedback with our team at BMS Clinical Trials to improve yours and others’ experience. Thank you