CTIS2023-506677-36-00招募中1 期
A Phase 1-2 multicenter study (2-stages) to evaluate the safety and efficacy of intravenous GNT0006, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMDR9, formerly LGMD2I) - ATA-001-FKRP
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 40
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
- 0 至 65+（—）
- 性别
- All
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
研究者
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