EUCTR2015-003225-33-GB进行中（未招募）1 期
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 (ß)-Thalassemia - The BEYOND Study
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 150
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Subjects must be = 18 years of age at the time of signing the informed consent document (ICF).
- •2. Subject must understand and voluntarily sign an ICF prior to any study-related assessments/procedures being conducted.
- •3. 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.
- •4. Subject must have documented diagnosis of ß-thalassemia or hemoglobin E/ ß-thalassemia. Concomitant alpha globin mutation and/or duplication are allowed.
- •5. 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.
- •6. Subject must not be on a regular transfusion program and must be RBC transfusion-free for at least = 8 weeks prior to randomization
- •7. 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.
- •8. Subject must have performance status: Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 1.
- •9. A female of childbearing potential (FCBP) for this study is defined as a female who: 1) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months). A FCBP participating in the study must:
- •a. Have 2 negative pregnancy tests as verified by the Investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the study, and after end of study treatment. This applies even if the subject practices true abstinence from heterosexual contact.
- •b. Either commit to true abstinence from heterosexual contact (which must be reviewed on a monthly basis and source documented). If a FCBP engages in sexual activity that may result in a pregnancy, she must agree to use, and be able to comply with, effective contraception without interruption, 28 days prior to starting IP, during the study therapy (including dose interruptions), and for 12 weeks (approximately 5 times the mean terminal half-life of luspatercept based on multiple-dose pharmacokinetics [PK] data) after discontinuation of study therapy.
- •10. Male subjects must:
- •a. Practice true abstinence (which must be reviewed on a monthly basis) or agree to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 12 weeks (approximately 5 times the mean terminal half-life of luspatercept based on multiple-dose PK data) following IP discontinuation, even if he has undergone a successful vasectomy
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 148
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. Subject has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from participating in the study.
- •2. Subject has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study.
- •3. Subject has any condition that confounds the ability to interpret data from the study.
- •4. Subject has a diagnosis of hemoglobin S/ß-thalassemia or alpha (a)-thalassemia (eg, Hemoglobin H).
- •5. Subject has active hepatitis C (HCV) infection as demonstrated by a positive HCV-RNA test of sufficient sensitivity, or active infectious hepatitis B as demonstrated by the presence of hepatitis B surface antigen (HBsAG) and/or hepatitis B virus DNA (HBV-DNA) positive, or known positive human immunodeficiency virus (HIV).
- •Note: Subjects receiving antiviral therapies should have 2 negative HCV-RNA test 3 months apart before ICF signature, ie, one test at the end of the anti-viral therapy and second test 3 months following the first test.
- •6. Subject had deep vein thrombosis (DVT) or stroke requiring medical intervention = 24 weeks prior to randomization.
- •7. 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.
- •8. Subject has received treatment with another investigational drug or device = 28 days prior to randomization.
- •9. Subject had prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536).
- •10. Subject has platelet count > 1000 x 10^9/L.
- •11. Subjects on iron chelation therapy (ICT) at the time of ICF signature must have initiated the treatment with ICT at least 24 weeks before the predicted randomization date. ICT can be initiated at any time during treatment and should be used according to the label.
- •12. Subject had Hydroxyurea and ESA treatment = 24 weeks prior to randomization, and no prior gene therapy.
- •13. Subject is pregnant or a lactating female.
- •14. Subject has uncontrolled hypertension. Controlled hypertension for this protocol is considered = Grade 1 according to National Cancer Institute Common Terminology for Adverse Events (NCI CTCAE) version 4.0 (current active minor version).
- •15. Subject has major organ damage, including:
- •a. Liver disease with alanine aminotransferase (ALT) > 3 x upper limit of normal (ULN) or history/evidence of cirrhosis, as well as presence of masses/tumor detected by ultrasound at screening.
- •b. Heart disease, heart failure as classified by the New York Heart Association (NYHA) classification 3 or higher, or significant arrhythmia requiring treatment, or recent myocardial infarction (MI) within 6 months of randomization.
- •c. Severe lung disease, including pulmonary fibrosis or pulmonary hypertension, ie, =G3 NCI CTCAE version 4.0 (current active minor version).
- •d. Estimated glomerular filtration rate (eGFR) < 60 ml/min/1.73 m2 (per Modification of Diet in Renal Disease [MDRD] formula).
- •16. Subject has received chronic systemic glucocorticoids = 12 weeks prior to randomization (physiologic replacement therapy for adrenal insufficiency is allowed).
- •17. Subject had major surgery = 12 weeks prior to randomization (subjects must have completely recovered fr
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