跳至主要内容
临床试验/CTRI/2025/02/080626
CTRI/2025/02/080626招募中Phase 3 4

Long-term Follow-up of Patients with Spinal Muscular Atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials

Novartis Healthcare Pvt Ltd5 个研究点 分布在 1 个国家目标入组 175 人开始时间: 2025年2月28日最近更新:

试验速览

阶段
Phase 3 4
状态
招募中
发起方
入组人数
175
试验地点
5
主要终点
What is the long-term effect of OAV101/ Zolgensma on safety in patients with SMA who were treated with this drug in past trials?

研究概览

简要总结

This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue from date of OAV101 administration in the previous clinical trial. The study is comprised of a Baseline Period and 3 Follow-up Periods. Follow-up Periods 1 and 2 consist of in-person visits and Period 3 consists of tele-visits. Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. During Follow-up Period 3 (Year 6 to up to Year 15 after OAV101 administration), participants/caregivers will be contacted using tele-visits annually for remote assessments. All patients will enter the study at the baseline visit and continue until 15 years since OAV101 administration is reached. Total duration of participation in the study will be dependent upon time of enrollment relative to OAV101 administration and will vary by participant.

研究设计

研究类型
Interventional
分配方式
Na
盲法
None

入排标准

年龄范围
2.00 Year(s) 至 18.00 Year(s)(—)
性别
All

入选标准

  • Participants eligible for inclusion in this study must meet all of the following criteria:
  • Participated in an OAV101 clinical trial.
  • Written informed consent must be obtained before any assessment is performed.
  • 3.Patient/Parent/legal guardian willing and able to comply with study procedures.

排除标准

  • There are no specific exclusion criteria for this study.

结局指标

主要结局

What is the long-term effect of OAV101/ Zolgensma on safety in patients with SMA who were treated with this drug in past trials?

时间窗: Every 6 months for 2 years and then every year till the 5th Year

次要结局

  • To explore other potential effects of OAV101 on exploratory efficacy & safety biomarkers(Every 6 months for 2 years & then every year till 5th Year)
  • To describe the time-course of the elevation of anti-AAV9 antibodies after administration of OAV101(Every 6 months for 2 years & then every year till 5th Year)
  • To assess long-term caregiver experience(Every 6 months for 2 years & then every year till 5th Year)

研究者

发起方
Novartis Healthcare Pvt Ltd
申办方类型
Pharmaceutical industry-Global
责任方
Principal Investigator
主要研究者

Murugananthan K

Novartis Healthcare Private

研究点 (5)

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