NCT07354724招募中1 期
A Phase 1, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 32
- 试验地点
- 4
- 主要终点
- Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
This is a Phase 1, multicenter, open-label study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of DNL952 in adult participants with late-onset Pompe disease. The principal aim of this study is to obtain safety and tolerability data across varous dose levels of DNL952 in participants with late-onset Pompe disease (LOPD).
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Body weight ≥40 kg
- •Diagnosis of LOPD
- •Upright FVC ≥ 30% of predicted normal value
- •Able to ambulate ≥ 40 meters (use of assistive devices is acceptable)
- •[Cohorts A1-A4 only] Have received avalglucosidase alfa or cipaglucosidase alfa at a dose of 20 mg/kg every 2 weeks for at least 12 months prior to screening
- •[Cohorts B1-B2 only] Must not have received any enzyme-replacement therapy for Pompe disease in the 12 months prior to screening
排除标准
- •Any ongoing, clinically significant, unstable, or poorly controlled neurological, psychiatric, endocrine, pulmonary, cardiovascular, gastrointestinal, hepatic, pancreatic, renal, metabolic, hematological, immunological, allergic, or ophthalmic disease not related to Pompe disease, or other major disorders. Well-controlled conditions are permitted if investigator and Sponsor agree.
- •Wheelchair-dependent
- •Require noninvasive ventilation for an average of more than 6 hours per day while awake or any invasive ventilation. Use of noninvasive ventilation during sleep is acceptable.
- •Received an experimental gene therapy at any time or participation in any other investigational drug trial or use of investigational drug within 60 days or 5 half-lives, whichever is longer, before screening
研究组 & 干预措施
Cohort A3 (Optional)
Experimental
Participants with LOPD
干预措施: DNL952 (Drug)
Cohort B2 (Optional)
Experimental
Participants with LOPD
干预措施: DNL952 (Drug)
Cohort A1
Experimental
Participants with LOPD
干预措施: DNL952 (Drug)
Cohort B1 (Optional)
Experimental
Participants with LOPD
干预措施: DNL952 (Drug)
Cohort A2
Experimental
Participants with LOPD
干预措施: DNL952 (Drug)
Cohort A4 (Optional)
Experimental
Participants with LOPD
干预措施: DNL952 (Drug)
结局指标
主要结局
Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs)
时间窗: 48 weeks
Incidence and severity of infusion-related reacations (IRRs)
时间窗: 48 weeks
次要结局
- PK parameter: Maximum concentration (Cmax) of DNL952 in serum(48 weeks)
- PK Parameter: Time to reach maximum concentration (tmax) of DNL952 in serum(48 weeks)
- PK Parameter: Area under the concentration-time curve (AUC) from time zero to time of last measurable concentration (AUClast) of DNL952 in serum(48 weeks)
- PK Parameter: AUC from time 0 to infinity (AUC∞) of DNL952 in serum(48 weeks)
- PK parameter: AUC from time zero to time t (AUCt) of DNL952 in serum(48 weeks)
- PK Parameter: terminal elimination half-life (t1/2) of DNL952 in serum(48 weeks)
研究者
研究点 (4)
Loading locations...
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