SAD/MAD FIH study of ALE1 in in healthy adult subjects and adult patients with HPP
Research type
Research Study
Full title
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
IRAS ID
1012437
Contact name
Aaron Deveney
Contact email
Sponsor organisation
Alesta Therapeutics B.V.
Clinicaltrials.gov Identifier
Research summary
This study is testing a new experimental drug called ALE1, developed to treat hypophosphatasia (HPP)—a rare genetic condition that affects bone and tooth strength due to a faulty enzyme.
The primary aim of the study is to test the safety and tolerability of ALE1 in healthy adults and in adult patients with HPP after single and multiple doses of ALE1 (the study drug) at increasing strengths.
The secondary aims of the study are to measure the amount of ALE1 in the blood and urine (called pharmacokinetics or PK), and to measure the effect of ALE1 on the body (called pharmacodynamics or PD) in healthy adults and in adult patients with HPP who receive single and multiple doses of ALE1 at increasing strengths. The study will also measure whether having a meal before taking ALE1 affects ALE1 PK.
The study is divided into four parts:
Parts 1A & 1B: Healthy adults will receive single or multiple doses of ALE1. (conducted in New Zealand only)
Parts 2A & 2B: Adults with HPP will receive single or multiple doses of ALE1. (conducted in UK and EU)
Participants will be randomly assigned to receive either ALE1 or a placebo (a capsule with no active ingredients). Neither participants nor study staff will know who receives which—this is called a double-blind, placebo-controlled study.
To be eligible for the study, participants must be aged 18 to 50 years, with a BMI in the range 18 to 32 and no previous or current medical conditions or treatments that could affect the safety or the study results.
Additionally, HPP patients in Parts 2A and 2B must have a known genetic mutation.
Safety checks include heart monitoring, blood tests, and follow-up visits. ALE1 has shown promising safety results in animal studies, though some risks remain, including possible allergic reactions and unknown effects on pregnancy.
Participation is voluntary, and individuals can leave the study at any time. By joining, participants are helping researchers explore a potential new treatment for HPP.REC name
Wales REC 1
REC reference
25/WA/0306
Date of REC Opinion
12 Nov 2025
REC opinion
Further Information Favourable Opinion