NL-OMON56084招募中3 期
A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T) - AG348-C-018 (ENERGIZE-T)
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 21
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. >=18 years of age at the time of providing informed consent.
- •2. Documented diagnosis of thalassemia (β-thalassemia with or without a-globin
- •mutations, HbE/β-thalassemia, or a-thalassemia/HbH disease) based on DNA
- •from the subject*s medical record. If this information is not available from
- •the subject*s
- •medical record, DNA analysis can be performed by a local laboratory during the
- •Screening Period. If a local laboratory is unable to perform the test, results
- •comprehensive a- and β-globin genotyping performed by the study central
- •laboratory can
- •3. Transfusion dependent, defined as 6 to 20 RBC units transfused and a <=6-week
- •transfusion-free period during the 24-week period before randomization.
- •4. If taking hydroxyurea, the hydroxyurea dose must be stable for >=16 weeks
- •randomization.
- •5. Women of childbearing potential (WOCBP) must be abstinent of sexual
- •activities that may induce pregnancy as part of their usual
- •lifestyle or agree to use 2 forms of contraception, 1 of which must be
- •considered highly
- •effective, from the time of providing informed consent, throughout the study,
- •28 days after the last dose of study drug. The second form of contraception can
- •be an acceptable barrier
- •6. Written informed consent before any study-related procedures are conducted
- •and willing
- •to comply with all study procedures for the duration of the study.
排除标准
- •1. Pregnant, breastfeeding or parturient.
- •2. Documented history of homozygous or heterozygous HbS or HbC.
- •3. Prior exposure to gene therapy or prior bone marrow or stem cell
- •transplantation.
- •4. Currently receiving treatment with luspatercept; the last dose must have
- •been administered >=36 weeks before randomization.
- •5. Currently receiving treatment with hematopoietic stimulating agents; the
- •last dose must have been administered >=36 weeks before randomization.
- •6. History of malignancy (active or treated) <=5 years before providing informed
- •consent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in
- •situ, or breast carcinoma in situ.
- •7. History of active and/or uncontrolled cardiac or pulmonary disease <=6 months
- •before providing informed consent, including but not limited to:
- •a. New York Heart Association Class III or IV heart failure or clinically
- •significant dysrhythmia
- •b. Myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or
- •thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism
- •c. Heart rate-corrected QT interval using Fridericia*s method >=450 milliseconds
- •(males) or >=470 milliseconds (females), except for right or left bundle branch
- •d. Severe pulmonary fibrosis as defined by severe hypoxia, evidence of
- •right-sided heart failure, and radiographic pulmonary fibrosis >50%
- •e. Severe pulmonary hypertension as defined by severe symptoms associated with
- •hypoxia, right-sided heart failure, and oxygen indicated
- •8. Hepatobiliary disorders, including but not limited to:
- •a. Liver disease with histopathological evidence of cirrhosis or severe fibrosis
- •b. Clinically symptomatic cholelithiasis or cholecystitis (prior
- •cholecystectomy is not exclusionary)
- •c. History of drug-induced cholestatic hepatitis
- •d. Aspartate aminotransferase >2.5 × upper limit of normal (ULN); unless due to
- •hemolysis and hepatic iron deposition) and alanine aminotransferase >2.5 × ULN
- •(unless due to hepatic iron deposition)
- •9. Estimated glomerular filtration rate <45 mL/min/1.73 m2 by Chronic Kidney
- •Disease Epidemiology Collaboration creatinine equation.
- •10. Nonfasting triglycerides >440 mg/dL (5 mmol/L).
- •11. Active infection requiring systemic antimicrobial therapy at the time of
- •providing informed consent. If antimicrobial therapy is required during the
- •Screening Period, screening procedures should not be performed while
- •antimicrobial therapy is being administered, and the last dose of antimicrobial
- •therapy must be administered >=7 days before randomization.
- •12. Positive test for hepatitis C virus (HCV) antibody (Ab) with evidence of
- •active HCV infection, or positive test for hepatitis B surface antigen.
- •13. Positive test for HIV-1 Ab or HIV-2 Ab.
- •14. History of major surgery (including splenectomy) <=6 months before providing
- •informed consent and/or a major surgical procedure planned during the study.
- •15. Current enrollment or past participation (<=12 weeks before administration
- •of the first dose of study drug or a time frame equivalent to 5 half-lives of
- •the investigational treatment, whichever is longer) in any other clinical study
- •involving an investigational treatment or device.
- •16. Receiving strong cytochrome P450 (CYP)3A4/5 inhibitors that have not been
- •stopped for >=5 days or a time frame equivalent to 5 half-lives (
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