Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 200
- 试验地点
- 21
- 主要终点
- Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC
研究概览
简要总结
This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
详细描述
Patients will receive standard of care treatments and assessments under the care of their healthcare provider. Biologic samples will be collected annually to measure cardiac and other related biomarkers. Clinical and observational data will be collected prospectively for up to 5 years from the date of enrollment, or until the patient withdraws consent/assent, undergoes heart transplantation, or dies.
If consent is provided, there may be a one-time sample collection to evaluate genetics for research purposes. Quality of Life (QoL) questionnaires will be used to assess a patient's wellbeing and quality of life. If not included as part of a patient's standard of care, diagnostic Holter (or equivalent) monitoring will be required annually. No investigational product will be administered. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 时间视角
- Other
入排标准
- 年龄范围
- 14 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Ages 14-65 years, inclusive, at the time of consent
- •Pathogenic or likely pathogenic PKP2 gene mutation
- •Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC as affected.
- •Functioning ICD
排除标准
- •Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy
- •History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection
- •Previously dosed with any investigational or approved gene therapy product at any time
- •Concurrent participation in another interventional clinical trial unless approved by the Sponsor. Participation in a noninterventional study may be allowed at the investigator's discretion.
- •History of cardiac transplant.
结局指标
主要结局
Investigate the seroprevalence of pre-existing antibodies to AAV9 in patients with PKP2-associated ARVC
时间窗: 5 years
次要结局
- To characterize the burden of illness in patients with pathogenic or likely pathogenic PKP2 mutations(5 years)
- To evaluate functional status and Quality of Life (QoL) in patients with pathogenic or likely pathogenic PKP2 mutations(5 years)
- To characterize arrhythmic risk in patients with pathogenic or likely pathogenic PKP2 mutations(5 years)
- To evaluate heart function as assessed by imaging in patients with pathogenic or likely pathogenic PKP2 mutations(5 years)