A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Depen… (NCT03342404) | Clinical Trial Compass
CompletedPhase 2
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
United States, Greece145 participantsStarted 2018-02-05
Plain-language summary
This is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion dependent beta (β)-thalassemia. The study is divided into the Screening Period, Double-blind Treatment Period (DBTP), Open-label Phase (OLP), and Post-Treatment Follow-up Period (PTFP).
It is planned to randomize approximately 150 subjects at a 2:1 ratio of luspatercept versus placebo.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. Subjects must be ≥ 18 years of age at the time of signing the informed consent document (ICF).
✓. Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
✓. Subject is willing and able to adhere to the study visit schedule (eg, not scheduled to receive hematopoietic stem cell transplantation) and other protocol requirements.
✓. Subject must have documented diagnosis of β-thalassemia or hemoglobin E/ β-thalassemia. Concomitant alpha globin mutation and/or duplication are allowed.
✓. Subject must be non-transfusion dependent, defined as 0 to 5 units of RBCs received during the 24-week period prior to randomization. Note: 1 unit defined for this entry criterion as approximately 200 to 350 mL of transfused packed RBCs.
✓. Subject must not be on a regular transfusion program and must be RBC transfusion-free for at least ≥ 8 weeks prior to randomization
✓. Subject must have mean baseline hemoglobin ≤ 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.
✓. Subject must have performance status: Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 1.
Exclusion criteria
✕. Any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.
✕
What they're measuring
1
Percentage of Participants Achieving Erythroid Response (Week 13 to Week 24)
Timeframe: From Week 13 to Week 24 of study treatment
. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
✕. Any condition that confounds the ability to interpret data from the study.
✕. Diagnosis of hemoglobin S/β-thalassemia or alpha (α)-thalassemia (eg,Hemoglobin H).
✕. Active hepatitis C (HCV) infection
✕. Deep vein thrombosis (DVT) or stroke requiring medical intervention ≤ 24 weeks prior to randomization.
✕. Subjects on chronic anticoagulant therapy are excluded, unless they stopped the treatment at least 28 days prior to randomization. Anticoagulant therapies for prophylaxis and for surgery or high-risk procedures as well as low molecular weight (LMW) heparin for superficial vein thrombosis (SVT) and chronic aspirin are allowed before and during the study.
✕. Treatment with another investigational drug or device ≤ 28 days prior to randomization.