Open-Label Study of Zopapogene Imadenovec Retreatment and Vector Shedding Evaluation in Adult Patients With Recurrent Respiratory Papillomatosis
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 30
- 试验地点
- 3
- 主要终点
- Cohort 1 Vector shedding
研究概览
简要总结
This open-label study evaluates safety, vector shedding, and retreatment efficacy of Zopapogene imadenovec (Zopa) in adults with recurrent respiratory papillomatosis (RRP). Two cohorts will be enrolled (n=30): Cohort 1 to assess the magnitude and duration of adenoviral vector shedding in urine, feces, skin, and nasal tissue; Cohort 2 to assess the complete response rate following retreatment.
详细描述
This is a single-arm, open-label confirmatory study of PRGN-2012 in RRP patients with a history of repeated surgical debulking for the management of their disease. Patients will be treated with PRGN-2012 and followed for at least 12 months to evaluate for efficacy and safety endpoints.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age 18 years and older.
- •Clinical diagnosis of recurrent respiratory papillomatosis with histological confirmation of papilloma.
- •Cohort 1: Treatment-naïve with respect to Zopa.
- •Cohort 2: Received a minimum of four administrations of Zopa at 5 × 10^11 PU per injection and require clinically indicated debulking procedures.
- •Presence of laryngotracheal papillomas accessible for endoscopic cleanout.
- •ECOG performance status 0 or
- •Sexually active participants of reproductive potential must agree to use contraception during treatment and for 120 days for males and 6 months for females after last dose.
- •Ability to understand and sign informed consent.
排除标准
- •Conditions or therapies that increase risk or interfere with participation per investigator judgment.
- •Systemic corticosteroids >10 mg prednisone equivalent or other immunosuppressive medications within 14 days prior to dosing.
- •Other systemic RRP treatments or investigational agents within 30 days.
- •History of heparin-induced thrombocytopenia or vaccine-induced thrombotic thrombocytopenia.
- •Active uncontrolled HIV, hepatitis B, or hepatitis C infection.
- •Pregnant or nursing women.
- •Known allergy to any study drug component.
研究组 & 干预措施
Cohort 1 Vector Shedding
Zopa administered subcutaneously at 5 × 10^11 particle units on Day 1, Week 2, Week 6, and Week 12. Samples for vector shedding to be collected from urine, feces, skin, and nasal tissue.
干预措施: Zopapogene imadenovec (Zopa) (Drug)
Cohort 2 Retreatment
Patients with prior treatment with Zopa (5 × 10^11 PU per injection) and require clinically indicated debulking procedure
干预措施: Zopapogene imadenovec (Zopa) (Drug)
结局指标
主要结局
Cohort 1 Vector shedding
时间窗: Up to 6 weeks following last administration, with additional collections if needed until two consecutive measurements at or below limit of detection
Magnitude and duration of Zopa vector shedding measured in urine, fecal, skin, and nasal tissue
Cohort 2 Complete Response Rate
时间窗: 12 months following last administration
Percentage of subjects achieving a complete response defined as no clinically indicated debulking procedures during the 12 months following completion of Zopa retreatment.
次要结局
- Interval to First Debulking Procedure (Cohort 2)(3 years following last administration)
- Safety and Tolerability of Zopa (Cohort 1 and Cohort 2)(Up to 28 days after last administration, with extended monitoring during follow-up.)