2023-506677-36-00招募中2 期
A Phase 1-2 multicenter study to evaluate the safety and tolerability of intravenous ATA-100, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMDR9, formerly LGMD2I)
Atamyo Therapeutics, Atamyo Therapeutics2 个研究点 分布在 2 个国家目标入组 20 人开始时间: 2023年11月10日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 20
- 试验地点
- 2
- 主要终点
- Safety and tolerability of ATA-100, as measured by incidence of treatment emergent adverse events, incidence of serious adverse events, and incidence of clinically significant laboratory changes.
研究概览
简要总结
To assess the safety and tolerability of intravenous administration of ATA-100 in ambulant patients with LGMDR9 at two different dosage levels and to select the recommended dose for future studies
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Dose escalation phase
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •Ambulant male or female patients at least 16 years old
- •Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
- •Able to: i) Perform the 10-meter walk test (10MWT) within 30 sec with unilateral help, such as cane, or bilateral help, such as elbow crutches or orthotic devices below the knees ii) Rise from a standard-height chair with or without arm support
- •Diaphragmatic muscle impairment defined as forced vital capacity (FVC) between 40 and 80% (inclusive) of the expected value
- •Effective contraception
- •Signed written informed consent before any study related procedure is performed
- •Patient medical status sufficiently stable and ability of patient and parents/legal guardian, in the opinion of the Investigator, to adhere to the study visit schedule and other protocol requirements.
排除标准
- •Detectable serum neutralizing antibodies against AAV9
- •Current participation in a clinical trial of another investigational medicinal product
- •Previous participation in gene and cell therapy trials
- •Any condition that would contraindicate treatment with immunosuppressant therapy
- •Presence of any permanent items (e.g., metal braces) precluding undergoing MRI
- •Any vaccination 1 month prior to the planned IMP administration
- •Serology consistent with HIV exposure or active hepatitis B or C infection
- •Grade 2 or higher lab abnormalities for LFT, bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT, and a PTT, according to current version of CTCAE.
- •Known hypersensitivity to IMP excipients, to eculizumab, murine proteins, or any excipients in eculizumab formulation
- •Cardiomyopathy based on physical and cardiological examination and echocardiography with Left Ventricular Ejection Fraction (LVEF) below 50%
- •Any respiratory assistance, including non-invasive daytime or nocturnal ventilation
- •Inability to cooperate with muscle testing or to perform respiratory function tests
- •Presence or history of concomitant muscular or other medical condition that might interfere with LGMDR9 evolution or that would confound scientific rigor or interpretation of results, e.g., current infectious episode (pulmonary, ENT,...), abnormal laboratory test if clinically significant
- •Acute illness within 4 weeks of the anticipated IMP administration which may interfere with study assessments
- •Recent immunosuppressive treatment within 3 months prior to screening
- •Current or history of significant heart, lung, hepato-biliary or renal disease or impairment that jeopardize the safety of the subject according to the investigator
结局指标
主要结局
Safety and tolerability of ATA-100, as measured by incidence of treatment emergent adverse events, incidence of serious adverse events, and incidence of clinically significant laboratory changes.
Safety and tolerability of ATA-100, as measured by incidence of treatment emergent adverse events, incidence of serious adverse events, and incidence of clinically significant laboratory changes.
次要结局
- Change from baseline in muscular function tests (NSAD score, 10MWT, etc.) and pulmonary function test (FVC) at one year post-IMP administration
- Change from baseline in muscle MRI parameters (fat fraction, T2 water content) at one year post-IMP administration
- Change from baseline in other respiratory assessments (IC, MIP/MEP, SNIP) at one year post-IMP administration
- Change from baseline in muscle biomarkers (histological features, biodistribution, transgene expression) at one year post-IMP administration
- Change from baseline in patient reported outcome and quality-of-life assessment (gNMD, Activlim) at one year post-IMP administration
- Change from baseline in biomarkers (creatine kinase, myomesin-3, circulating microRNA) at one year post-IMP administration
研究者
Genethon Clinical Development Department
Scientific
Atamyo Therapeutics
研究点 (2)
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