NCT05973630进行中（未招募）1 期
A Phase 1-2, Open-label Study to Evaluate the Safety of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients With Gamma-sarcoglycanopathy (LGMDR5)
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 4
- 试验地点
- 2
- 主要终点
- Incidence of treatment-emergent adverse events,
研究概览
简要总结
The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5).
详细描述
This is a multicenter Phase 1b assessing the safety and tolerability of intravenous ATA-200 for the treatment of LGMDR5.
The study will enroll ambulant patients with LGMDR5. Patients will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments. Patients will receive a potentially effective dose corresponding to the minimum effective dose (MED) in preclinical studies.
Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months.
All subjects will be followed up for an additional 4.5 years after completion of the initial evaluation period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 13 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping
- •Ambulant male or female patients aged 6 to less than 12 years of age at screening
- •Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support
排除标准
- •Detectable neutralizing antibodies against AAV8
- •Cardiomyopathy with left ventricular ejection fraction (LVEF) < 50%
- •Respiratory assistance
- •Concomitant medical condition that might interfere with LGMDR5 evolution
- •Acute illness within 4 weeks of anticipated IMP administration
- •Current participation in another clinical trial with investigational medicinal product
- •Previous participation in gene and cell therapy trials
- •Any condition that would contraindicate immunosuppressant treatment
- •Presence of any permanent items (e.g., metal braces) precluding undergoing MRI
- •Any vaccination 1 month prior to planned IMP administration
- •Serology consistent with HIV exposure or active hepatitis B or C infection
- •Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and coagulation
研究组 & 干预措施
Treatment arm
Experimental
ATA-200 Dose : 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
干预措施: ATA-200 (Biological)
结局指标
主要结局
Incidence of treatment-emergent adverse events,
时间窗: 0-6 months
Collection of adverse events at each visit
Incidence of adverse events
时间窗: 0-6 months
Collection of adverse events at each visit
Incidence of serious adverse events
时间窗: 0-6 months
Collection of adverse events at each visits
次要结局
未报告次要终点
研究者
研究点 (2)
Loading locations...
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相关资讯
Atamyo Therapeutics Reports Promising Early Results for ATA-200 Gene Therapy in Rare Muscular Dystrophy- Atamyo Therapeutics announced encouraging safety and efficacy results for ATA-200 gene therapy in the first four patients with LGMD-R5, a severe childhood muscular dystrophy that leads to loss of walking ability before adulthood.
- The Phase 1b/2 trial showed over 90% of muscle fibers expressing the therapeutic protein in the first two patients at six months, with significant reductions in muscle damage biomarkers and clinical improvements in functional tests at nine months.
- No serious adverse events were observed in the four treated patients, demonstrating the safety profile of the single intravenous AAV-based gene therapy targeting gamma-sarcoglycan deficiency.
- The results represent rare biological data in neuromuscular diseases at such an early trial stage, offering potential hope for approximately 2,000 patients with LGMD-2C/R5 in Europe and the US who currently have no curative treatment options.6 months agoAtamyo Therapeutics' ATA-200 Gene Therapy Receives FDA Clearance for US Clinical Trial in LGMD2C/R5- The FDA has cleared Atamyo Therapeutics' IND application for ATA-200, a gene therapy for Limb-Girdle Muscular Dystrophy Type 2C/R5 (LGMD2C/R5).
- ATA-200 delivers a functional copy of the SGCG gene and has shown promise in preclinical studies, correcting symptoms and disease biomarkers in mice.
- The Phase 1b dose-escalation trial (NCT05973630) will now expand to the US, supported by funding from The Dion Foundation, with plans to open the first US center before year-end.
- ATA-200 has also been granted orphan drug designation by the FDA, highlighting the unmet need for treatments for this rare pediatric disease.last yearFDA Clears Atamyo's ATA-200 Gene Therapy Trial for LGMD2C/R5 in Children- The FDA has approved Atamyo Therapeutics' Investigational New Drug application for ATA-200, a gene therapy for limb-girdle muscular dystrophy type 2C/R5 (LGMD2C/R5).
- ATA-200 is the first potential treatment for LGMD2C/R5 to enter clinical development in the U.S., offering hope for children with this debilitating condition.
- The Phase 1b trial will enroll approximately six children aged 6-13 across sites in the U.S., France and Italy, evaluating the safety and tolerability of ATA-200.
- ATA-200 delivers a healthy copy of the _SGCG_ gene to muscle cells, aiming to restore gamma-sarcoglycan protein production and improve muscle function in affected individuals.last yearFDA Approves Autolus' Obecabtagene Autoleucel (Aucatzyl) for R/R B-cell ALL• The FDA has approved obecabtagene autoleucel (obe-cel), a CD19-directed CAR-T therapy, for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia (r/r B-cell ALL).
• Approval was based on the Phase 1b/2 FELIX trial, which demonstrated overall complete remission rates above 60% and durable remissions exceeding 12 months.
• Clinical holds on CARsgen's BCMA-directed CAR-T therapy zevorcabtagene autoleucel and Claudin18.2-directed CAR-T satricabtagene autoleucel have been removed by the FDA.last yearAtamyo Therapeutics Advances Gene Therapy Programs for Limb-Girdle Muscular Dystrophy- Atamyo Therapeutics completed recruitment for the dose escalation phase of the Phase 1b trial of ATA-100 for LGMD2I/R9.
- The company submitted an IND application to the FDA for ATA-200, a gene therapy for LGMD2C/R5, expanding its clinical trial to the U.S.
- Initial results from the ATA-100 trial show promising functional improvements and a marked decline in creatine kinase levels in treated patients.
- The FDA granted rare pediatric disease designation to ATA-200, highlighting the unmet need for treatments for these rare muscular dystrophies.2 years ago