CTIS2023-506440-16-00进行中（未招募）1 期
A Phase I/II, open-label, dose escalation study to evaluate the safety of 2 doses of intravenous ATA-200, an adeno-associated viral vector carrying the human gamma-sarcoglycan gene, in patients with gamma-sarcoglycanopathy (limb-girdle muscular dystrophy LGMDR5, formerly LGMD2C). - ATA-003-GSAR
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 6
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 17（—）
- 性别
- All
入选标准
- •Ambulant male or female patients at least 6 and less than 12 years of age at screening, Confirmed LGMDR5 diagnosis before age of 10, based on clinical presentation and genotyping identifying the SGCG gene mutations, Able to Perform the 10-meter walk test (10MWT) within 15 sec without help, such as cane, or bilateral help, such as elbow crutches or orthotic devices below the knees and to rise from a standard-height chair with or without arm support, 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 visits schedule and other protocol requirements., Seronegative patients for neutralizing antibodies against AAV8
排除标准
- •Previous participation in gene and cell therapy trials, 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 LGMDR5 evolution or that would confound scientific rigor or interpretation of results, e.g., current infectious episode (pulmonary, ENT, etc.), abnormal laboratory test if clinically significant, 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, Acute illness within 4 weeks of the anticipated IMP administration which may interfere with study assessments, Current participation in a clinical trial of another investigational medicinal product, 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 (except isolated AST increase), bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT, and a PTT, according to current version of CTCAE. Isolated AST increase associated with CK levels > 800 U/L, ALT < 2 x ULN, and GLDH within normal range, is not exclusionary., Known hypersensitivity to IMP excipients, to eculizumab, murine proteins, or any excipients in eculizumab formulation, and to contrast media, 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
研究者
相似试验
进行中（未招募）
1 期
Clinical study using the drug GSK525762 in Subjects with cancerT Midline Carcinoma and other solid tumorsMedDRA version: 18.0Level: LLTClassification code 10007284Term: CarcinomaSystem Organ Class: 100000004864EUCTR2014-004982-25-FRGlaxoSmithKline Research and Development Ltd196
进行中（未招募）
1 期
Clinical study using the drug GSK525762 in subjects with hematologic malignanciesPatients with relapsed and/or refractory haematological malignancies (lymphoma, leukemia, or multiple myeloma)MedDRA version: 19.0Level: LLTClassification code 10066481Term: Hematological malignancySystem Organ Class: 100000004864EUCTR2013-000445-39-ESGlaxoSmithKline, S.A.111
进行中（未招募）
1 期
Clinical study using the drug GSK525762 in Subjects with cancerEUCTR2014-004982-25-NLGlaxoSmithKline Research and Development Ltd196
进行中（未招募）
1 期
Clinical study using the drug GSK525762 in Subjects with cancerT Midline Carcinoma and other solid tumors.MedDRA version: 18.0 Level: LLT Classification code 10007284 Term: Carcinoma System Organ Class: 100000004864EUCTR2014-004982-25-ESGlaxoSmithKline, S.A.196
进行中（未招募）
1 期
Clinical study using the drug GSK525762 in subjects with hematologic malignanciesPatients with relapsed and/or refractory haematological malignancies (lymphoma, leukemia, or multiple myeloma)MedDRA version: 18.0Level: LLTClassification code 10066481Term: Hematological malignancySystem Organ Class: 100000004864EUCTR2013-000445-39-GBGlaxoSmithKline Research and Development Limited180