CTIS2023-509748-89-00进行中（未招募）1 期
A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-DUX4 in Adult Patients with Facioscapulohumeral Muscular Dystrophy Type 1 - ARODUX4-1001
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 70
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 65+（—）
- 性别
- All
入选标准
- •Genetically confirmed FSHD1 (based on Screening evaluation or source verifiable medical record). If available, the number of repeats (1 to 10) via assessment of the size of the D4Z4 array on chromosome 4 should be provided. Confirmation must be obtained prior to the Screening MRI and baseline muscle biopsy., Clinical severity score (CSS) between 3 and 8 (scale, 0 to 10), Must have an eligible lower extremity muscle for biopsy as determined from MRI by a central reader, with MFF =10% and less than approximately 40%., Males or nonpregnant, nonlactating females =18 years of age who do not plan to become pregnant during the study, with an upper age limit of =70 years., Able and willing to provide written informed consent prior to the performance of any study specific procedures., Subjects with a body mass index (BMI) between 18.0 and 30.0 kg/m2, inclusive. A subject with FSHD1 and a BMI outside this range may be allowed into the study at the discretion of the PI., A 12-lead ECG at Screening with no abnormalities that may compromise the subject’s safety in this study per PI discretion., Subjects of childbearing potential and their partners must agree to use highly effective contraception during the study and for at least 12 weeks following the end of the study or last dose of IP, whichever is later. Males must not donate sperm during the study from Day 1 until at least 12 weeks following the end of the study or last dose of IP, whichever is later., Must be willing and able to comply with all study assessments and adhere to the protocol schedule.
排除标准
- •Is unable to comply with the study requirements, including the number of required visits to the clinical site., Use of an investigational agent or device within 30 days (or longer as per local regulations) prior to dosing or current participation in an investigational study., Blood donation (500 mL) within 7 days prior to study treatment administration. Donation or loss of whole blood (excluding the volume of blood that will be drawn during the Screening procedures of this study) prior to administration of the study treatment as follows: 50 mL to 499 mL of whole blood within 30 days, or more than 499 mL of whole blood within 56 days prior to study treatment administration., Any concomitant medical or psychiatric condition or social situation that would make it difficult to comply with protocol requirements or put the subject at additional safety risk., Estimated glomerular filtration rate (eGFR) <60 mL/min/1.73m2 calculated by using the Chronic Kidney Disease Epidemiology Collaboration creatinine equation (CKD-EPI)., Current concomitant use of theophylline (including duration of study)., History of thromboembolic events including deep vein thrombosis, thrombotic stroke, pulmonary embolism, or atrial thrombi., Platelet count less than the lower limit of normal at Screening., History or presence of any of the following based on source verifiable medical record and physical exam or reported medical history when applicable: • a hypercoagulable state including factor V Leiden mutation, increased factor VIII, increased proteins C and S, and antithrombin deficiency • nephrotic range proteinuria • antiphospholipid antibody syndrome or myeloproliferative diseases (polycythemia vera and essential thrombocythemia) • inability to ambulate • use of hormone-based contraceptives (including oral, transdermal patch, vaginal ring, and injectables) =16 weeks prior to Day 1, peri- and postmenopausal hormone replacement therapy =16 weeks prior to Day 1. Note: Use of intrauterine device (IUD) with levonorgestrel (single hormone) is allowed., ALT or AST >3×ULN at Screening., Any contraindications to muscle biopsy., HIV infection, as shown by the presence of anti-HIV antibody (seropositive) at Screening., Any contraindications to MRI., History of any illness or any clinical condition that, in the opinion of the PI, might confound the results of the study or pose an additional risk in administering study drug to the subject. This may include, but is not limited to, a history of relevant drug or food allergies; history of cardiovascular or central nervous system disease; neuromuscular diseases except FSHD (eg, myopathy, neuropathy, neuromuscular junction disorders); or clinically significant history of mental disease., For subjects who are on drug(s) or supplements that may affect muscle function, as determined by the treating physician, or that are listed in Section 8.2.3, subjects must be on a stable dose of that drug(s) or supplement for at least 28 days prior to the first dose of study drug and with no plans to change dose or treatment regimens during the duration of the study. Changes to the dose or treatment discontinuation during the study can only be done for medical reasons as part of standard management by the treating physician with clear documentation and notification to the sponsor., Seropositive for hepatitis B (positive HBsAg at Screening) or hepatitis C (HCV) at Screening, (positive for anti-HCV antibody must be confirmed with positive HCV-RNA te
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