A Long-term Follow-up Study for Subjects Previously Treated with Autologous ex vivo Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 38
- 试验地点
- 1
- 主要终点
- To characterize the long-term safety of the gene therapy treatment with Telethon003 as measured by: recording of adverse event (AE) and serious adverse event (SAE) related to gene therapy including: − insertional mutagenesis and oncogenesis (blood and solid malignancies); − transgene immunogenicity; − development of replication-competent lentiviruses (RCL);
研究概览
简要总结
- To characterize the long-term safety of Telethon003 treatment.
- To characterize the long-term clinical efficacy of Telethon003 treatment.
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 18-64 Years, 65+ Years）
- 接受健康志愿者
- 是
入选标准
- •The patient has been treated with Telethon003
- •The patient or, if applicable, the patient’s parent(s)/legal guardian(s), are able and willing to provide informed consent.
- •Willingness to attend the study visit schedule and be compliant with the protocol.
排除标准
- •Participation in an ongoing clinical study with an investigational drug (other to gene therapy study or early access, object for this study) prior to enrolment in this study or willingness to participate in another interventional clinical trial. If in deviation to this exclusion criteria, a subject took or takes another investigational drug or other available therapy (e.g. hematopoietic stem cell transplant) he will be followed anyway to collect safety events.
结局指标
主要结局
To characterize the long-term safety of the gene therapy treatment with Telethon003 as measured by: recording of adverse event (AE) and serious adverse event (SAE) related to gene therapy including: − insertional mutagenesis and oncogenesis (blood and solid malignancies); − transgene immunogenicity; − development of replication-competent lentiviruses (RCL);
To characterize the long-term safety of the gene therapy treatment with Telethon003 as measured by: recording of adverse event (AE) and serious adverse event (SAE) related to gene therapy including: − insertional mutagenesis and oncogenesis (blood and solid malignancies); − transgene immunogenicity; − development of replication-competent lentiviruses (RCL);
To evaluate the overall survival (OS) as primary efficacy endpoint up to 15 years of follow-up post treatment with Telethon003
To evaluate the overall survival (OS) as primary efficacy endpoint up to 15 years of follow-up post treatment with Telethon003
次要结局
- Use of IgRT
- Event free survival
- Annualized rate of severe infections compared with 1 year prior to gene therapy;
- Annualized rate of moderate and severe bleeding episodes compared with 1 year prior to gene therapy
- Eczema
- Autoimmunity
- Hematologic disorders (i.e. neutropenia and/or other cytopenias);
- Malignancies
- New or exacerbations of pre-existing neurological disorders
- Variation in height and weight (for paediatric patients)
- Karnofsky/Lansky performance scales
- Platelet count and MPV
- Vector Copy Number (VCN)
- Insertion site analysis (ISA)
- PedsQL (Pediatric Quality of Life Inventory) health-related quality of life scales
- EQ-5D, and SF-10 or SF-36 health-related quality of life questionnaires
研究者
Regulatory Affairs
Scientific
Fondazione Telethon Ets