Table of Contents
- Trial overview
- Who is being studied
- What researchers are measuring
- Study design and phase
- Trial status and size
Trial overview
The clinical trial for ALE1 is an interventional study in healthy adult participants and adult patients with hypophosphatasia.[1] The study is designed to assess safety, tolerability, pharmacokinetics, and pharmacodynamics, and to help identify suitable doses of ALE1.[1]
Who is being studied
This study includes two main groups: healthy adults and adults with hypophosphatasia.[1] Healthy participants help researchers understand how ALE1 behaves in people without the disease, while the patient group helps show what happens in the target condition.[1]
Hypophosphatasia is the condition listed in the trial data, and it is the disease being studied in the patient group.[1] The source data do not list any other conditions or age groups beyond adults.[1]
What researchers are measuring
The main outcome is the number and severity of treatment-emergent adverse events (TEAEs) in healthy participants and patients with hypophosphatasia, with increasing oral doses of ALE1.[1] TEAEs are medical problems that start or become worse after the study treatment begins.[1]
The study also looks at pharmacokinetics and pharmacodynamics.[1] Pharmacokinetics means how the body handles the study treatment, and pharmacodynamics means the early effects seen in the body.[1]
Study design and phase
This is a Phase 1/2 trial.[1] In simple terms, Phase 1/2 studies are early trials that first focus on safety and dose finding, and may also look for early signs of benefit.[1]
The study uses single-ascending dose and multiple-ascending dose oral administration.[1] This means the trial tests one dose first, then higher doses, and also repeated doses over time to learn more about the medicine in the body.[1]
The interventions listed are ALE1 matched placebo and oral ALE1 dose groups, including ALE1 I and ALE1 III.[1] The source data do not explain these dose labels further, so they should be understood only as different study groups in the trial.[1]
Trial status and size
The trial status is Authorised.[1] The planned enrollment is 120 participants.[1]
Because this is an early-stage study, the main purpose is to collect research data that can guide later trials.[1] The key question is whether ALE1 can be studied safely enough, at what doses, and with what early body response in adults with and without hypophosphatasia.[1]
