CTIS2022-500281-10-02招募中1 期
An Open-label, Dose-Finding, Phase 1/2 Study to Evaluate the Safety and Tolerability of a Single Intravenous Dose of LY3884961 in Subjects with Peripheral Manifestations of Gaucher Disease - J3Z-MC-OJAE
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 15
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
- •1. Age 18-65 years inclusive at the time of informed consent. 2. Bi-allelic GBA1 mutations must be centrally confirmed. 3. On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening. 4. Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. 5. Females and males will be eligible for this study. Men and women of childbearing potential must use a highly effective method of contraception consistently and correctly for the duration of the study, including the long-term follow-up. 6. Patients must agree to abstain from blood donations for at least the first year of the study
排除标准
- •1. Clinically significant neurological signs and symptoms and/or behavioral disturbances. 2. Active and progressive bone disease expected to require surgical treatment in the next 6 months 3. History of total splenectomy or planned total splenectomy during the first 18 months of the study 4. Splenomegaly > 10 MN as evaluated by centrally read abdominal MRI 5. Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins 6. Thrombocytopenia with platelet count < 40 × 103 per µL 7. Severe hyperlipidemia (triglycerides > 1,000 mg/dL) 8. Current diagnosis of unstable or clinically significant cardiovascular conditions based on Investigator assessment 9. History of certain cancers within 5 years of Screening 10. Concomitant disease, condition or treatment which, in the opinion of the Investigator, would pose an unacceptable risk to the patient or interfere with the patient's ability to comply with study procedures or interfere with the conduct of the study. 11. Women, who are pregnant (i.e., positive serum pregnancy result at Screening and Day 1) or breastfeeding or intending to become pregnant during the course of the trial 12. Use of any GD-related chaperone therapy within 4 weeks prior to Screening or expected need to initiate chaperone therapy during at least the first 18 months of the study 13. Any type of prior gene or cell therapy 14. Use of systemic immunosuppressant or steroid therapy other than protocol-specified immunosuppression 15. Participation in another therapeutic investigational drug or device study within 3 months or 5 half-lives of the study agent, whichever is longer (unless it can be documented that the patient received placebo) 16. Have an anti-AAV9 antibody titer of >1:40 as determined by the central laboratory. 17. Clinically significant abnormalities in laboratory test results at Screening 18.Have any contraindications for magnetic resonance imaging (MRI), including claustrophobia or the presence of contraindicated metal (ferromagnetic) implants/cardiac pacemaker
研究者
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