2022-500281-10-02招募中2 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
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 3
- 主要终点
- Incidence and severity of TEAEs and SAEs, including clinically significant changes in vital signs, clinical laboratory parameter assessments, 12-lead ECGs, results of abdominal and bone MRIs, physical and neurological examinations, and waist circumference and weight over time.
研究概览
简要总结
To evaluate the safety and tolerability of LY3884961
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Expansion Period
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Age ≥ 18years at the time of informed consent.
- •Bi-allelic pathogenic GBA1 variants must be centrally confirmed.
- •On ERT or SRT for at least 2 years and on a stable, maximum tolerated dose, for at least 3 months prior to screening.
- •Capable of giving signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- •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.
- •Patients must agree to abstain from blood donations for at least the first year of the study; and must agree to abstain from tissue and organ donation for the duration of the study, including long-term follow-up.
排除标准
- •Clinically significant neurological signs and symptoms and/or behavioral disturbances.
- •Active and progressive bone disease expected to require surgical treatment in the next 6 months
- •History of total splenectomy or planned total splenectomy during the first 18 months of the study
- •Splenomegaly > 10 MN as evaluated by centrally read abdominal magnetic resonance imaging (MRI).
- •Evidence of clinically significant liver disease, fragile liver, or history of exposure to hepatotoxins
- •Thrombocytopenia with platelet count < 40 × 103 per μL
- •Severe hyperlipidemia (triglycerides > 1,000 mg/dL)
- •Current diagnosis of unstable or clinically significant cardiovascular conditions based on Investigator assessment
- •History of certain cancers within 5 years of Screening
- •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.
- •Women, who are pregnant (i.e., positive serum or urine pregnancy result at Screening and or Check-in) or breastfeeding or intending to become pregnant during the course of the trial.
- •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
- •Any type of prior gene or cell therapy
- •Use of systemic immunosuppressant or steroid therapy other than protocol-specified immunosuppression
- •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)
- •Have an anti-AAV9 antibody titer of >1:40 as determined by the central laboratory.
- •Clinically significant abnormalities in laboratory test results at Screening 18.Have any contraindications for MRI, including claustrophobia or the presence of contraindicated metal (ferromagnetic) implants/cardiac pacemaker
结局指标
主要结局
Incidence and severity of TEAEs and SAEs, including clinically significant changes in vital signs, clinical laboratory parameter assessments, 12-lead ECGs, results of abdominal and bone MRIs, physical and neurological examinations, and waist circumference and weight over time.
Incidence and severity of TEAEs and SAEs, including clinically significant changes in vital signs, clinical laboratory parameter assessments, 12-lead ECGs, results of abdominal and bone MRIs, physical and neurological examinations, and waist circumference and weight over time.
次要结局
- Change and percent change from baseline in spleen volume (MN)
- Change from baseline in platelet count
- Change from baseline in GCase enzyme activity and protein levels and GluSph levels
- Time from LY3884961 to ERT/SRT discontinuation.
- Time from discontinuation of ERT/SRT to re-initiation of ERT/SRT.
研究者
Regulatory Department
Scientific
Prevail Therapeutics Inc.
研究点 (3)
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