Multicenter Epidemiological Study for the Evaluation of the Seroprevalence of Total and Neutralizing Antibodies Against Adenoassociated Virus Serotypes Used in Gene Therapy in Patients With Neuromuscular Diseases of Genetic Origin. (SeroDysMyo)
研究概览
详细说明
This research aims to determine the seroprevalence and immunological profiles of recent and/or emerging AAV capsids that could be used in future gene therapy programs. It will thus make it possible to: 1/ improve knowledge of the prevalence of anti-AAV antibodies (total and neutralizing) in these specific populations; 2/ identify the clinical, demographic or biological factors likely to influence this seroprevalence (age, sex, type of pathology, immunosuppressive treatment followed); 3/ to characterize the phenomena of cross-reactivity between different serotypes or variants of capsids, a major challenge for the design of new generations of vectors.
The data generated will contribute to a better estimation of the populations potentially eligible for gene therapies, by making it possible to anticipate exclusion rates related to pre-existing immunity. They may also guide the rational selection of alternative capsids that are less immunogenic or evade cross-immunity, as well as the development of immunomodulation or re-assay strategies.
研究类型
注册 (估计的)
阶段
- 不适用
联系人和位置
学习地点
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-
Île-de-France Region
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Évry、Île-de-France Region、法国、9100
- GENETHON
-
接触：
- Philippe VERON, Master of Sciences
- 邮箱：veron@genethon.fr
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接触：
- Céline LABETOULE, Master of Sciences
- 邮箱：clabetoule@genethon.fr
-
-
参与标准
资格标准
适合学习的年龄
- 孩子
- 成人
接受健康志愿者
描述
Inclusion Criteria:
- Pediatric patient 6 years of age or older or adult under 60 years of age with genetic neuromuscular disease
- Informed patient who signed informed consent
- No opposition from the holders of parental authority or guardian, for minor patients.
- Affiliated/beneficiary of a national health insurance scheme
Exclusion Criteria:
- Gene or cell therapy treatment prior to blood collection
学习计划
研究是如何设计的？
设计细节
- 主要用途：放映
- 分配：不适用
- 介入模型：单组作业
- 屏蔽：无（打开标签）
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
|
其他：Cohort of 450 patients with neuro-muscular disease for AAV sero-analysis
|
In each clinical site, the principal investigator will propose to his patients with neuromuscular disease of genetic origin to take a blood sample (4 mL of blood) to determine their level of immunity to certain AAVs used in gene therapy. The collected samples will be prepared and aliquoted on site and then sent frozen to the GENETHON laboratory for analysis. Only one sample per patient will be analyzed. All samples will be analyzed in batches. |
研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
Percentage of patients with anti-AAV neutralizing antibodies
大体时间：Day 1
|
The primary endpoint is the percentage of patients who tested positive for neutralizing antibodies against multiple AAVs that can be used in gene therapy.
|
Day 1
|
次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
|
To estimate seroprevalence in subgroups of patients (age; type of neuromuscular disease; gender; immunosuppressive therapy)
大体时间：Day 1
|
Estimation of seroprevalence (positive or negative) of anti-AAV neutralizing antibodies in different subgroups (age, sex, type of neuromuscular disease, immunosuppressive therapy)
|
Day 1
|
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To analyze the co-prevalence between several AAV serotypes
大体时间：Day 1
|
Quantification of neutralizing antibody titers against various capsids using cellular tests
|
Day 1
|
|
Analyze the potential correlation between the presence of neutralizing antibodies and total antibody titer using cellular tests and ELISA method
大体时间：Day 1
|
Prevalence of cross-reactions between multiple capsids
|
Day 1
|
合作者和调查者
赞助
研究记录日期
研究主要日期
学习开始 (估计的)
初级完成 (估计的)
研究完成 (估计的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (实际的)
研究记录更新
最后更新发布 (实际的)
上次提交的符合 QC 标准的更新
最后验证
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- GNT-020-SDM
- ID RCB N° 2026-A00986-45 (其他标识符：French health authority (ANSM))
计划个人参与者数据 (IPD)
计划共享个人参与者数据 (IPD)？
IPD 计划说明
药物和器械信息、研究文件
研究美国 FDA 监管的药品
研究美国 FDA 监管的设备产品
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