2025-522378-36-00招募中2 期
A Randomised, Placebo-Controlled, Double-Blind, Single-Ascending Dose and Multiple-Ascending Dose First-In-Human Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Orally Administered ALE1 With or Without Food in Healthy Adult Participants and Adult Patients With Hypophosphatasia
Alesta Therapeutics B.V.1 个研究点 分布在 1 个国家目标入组 24 人开始时间: 2025年11月19日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 24
- 试验地点
- 1
- 主要终点
- Number and severity of treatment emergent adverse events (TEAEs) in healthy participants and patients with hypophosphatasia (HPP) with increasing oral doses of ALE1
研究概览
简要总结
To investigate the safety and tolerability of ALE1 in healthy adult participants and adult patients with HPP following single and multiple-ascending oral dose administrations.
入排标准
- 年龄范围
- 18 years 至 64 years（18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Aged ≥ 18 to ≤ 50 years (inclusive) at the time of signing the ICF.
- •Part 1: 1) Participants are overtly healthy as determined by a medical evaluation.
- •Part 1: 2) No concurrent medical conditions or significant medical history, in the opinion of the investigator.
- •Part 2: 1) Documented ALPL gene variant.
排除标准
- •Part 1: History of conditions affecting bone or mineral metabolism.
- •Part 2: 1) Previous treatment with an enzyme replacement therapy (ERT) or any advanced therapeutic agent (e.g., gene therapy) for the treatment of HPP or any treatment for osteoporotic diseases.
- •Part 2: 2) Previous exposure to any medication or investigational agent potentially affecting bone structure, muscle volume, muscle strength, or muscle or nerve function.
- •Part 2: 3) Diagnosis of hyperparathyroidism.
- •Part 2: 4) Diagnosis of hypoparathyroidism, unless secondary to HPP.
- •Part 2: 5) New fracture within 12 weeks before first dosing.
结局指标
主要结局
Number and severity of treatment emergent adverse events (TEAEs) in healthy participants and patients with hypophosphatasia (HPP) with increasing oral doses of ALE1
Number and severity of treatment emergent adverse events (TEAEs) in healthy participants and patients with hypophosphatasia (HPP) with increasing oral doses of ALE1
次要结局
- Pharmacokinetic parameters of single and multiple ascending doses of ALE1.
- Pharmacodynamic biomarker levels in blood samples of single and multiple ascending doses of ALE1.
- Dose proportionality in pharmacokinetic parameters in single and multiple ascending doses of ALE1.
- Effect of food on ALE1 pharmacokinetic parameters.
研究者
Medical Information
Scientific
Alesta Therapeutics B.V.
研究点 (1)
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