ALE1 safety, dosing, and activity in adults with and without hypophosphatasia
Official 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 Subjects And Adult Patients With Hypophosphatasia
Run by Alesta Therapeutics · NCT07179640 · Data updated 2026-11-12
Researchers will try different doses of ALE1 to check safety and how the drug acts in healthy adults and adults with hypophosphatasia.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years to 50 Years |
| Sex | All |
| Enrollment | 120 (estimated) |
| Sponsor | Alesta Therapeutics Industry-sponsored |
| Locations | 3 sites (Würzburg and 2 other locations) |
| Status | Recruiting |
| Started | September 30, 2025 |
| Main results expected | January 2027 |
| Study ends | January 2027 |
| Registry record updated | August 3, 2026 |
| Trial ID | NCT07179640 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal participants are adults with a documented ALPL gene variant who are medically stable, have not received prior enzyme replacement or advanced HPP therapies, and are willing to attend in-person study visits; the trial also enrolls healthy adult volunteers for safety comparisons.
✕Probably not a fit
Patients with other bone or mineral disorders, recent fractures, uncontrolled parathyroid disease, or prior exposure to ERT or gene therapies are unlikely to benefit from this early-phase dosing study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This randomized, placebo-controlled, double-blind Phase 1/2a trial tests single and multiple doses of ALE1 to characterize safety, tolerability, pharmacokinetics, and pharmacodynamics. The study enrolls overtly healthy adults and adults with genetically confirmed hypophosphatasia to identify suitable dose regimens. Key exclusions include prior enzyme replacement or other advanced therapies for HPP, recent fractures, and disorders affecting bone or mineral metabolism. Safety labs, PK sampling, and PD markers related to alkaline phosphatase activity will guide dose selection.
Why it matters
Potential benefit: If successful, ALE1 could offer a new treatment option that improves biochemical markers and symptoms of hypophosphatasia in adults.
How similar studies have gone: Enzyme replacement therapy such as asfotase alfa has improved outcomes in HPP, so targeting alkaline phosphatase pathways is a proven approach, but ALE1 itself is a novel agent with limited clinical data to date.
Who can join: the full rules
Show full inclusion / exclusion criteria
Key Inclusion Criteria Part 1: 1. Participants are overtly healthy as determined by a medical evaluation 2. No concurrent medical conditions or significant medical history, in the opinion of the investigator. Key Inclusion Criteria Part 2: 1\. Documented ALPL gene variant Key Exclusion Criteria Part 1: 1\. History of conditions affecting bone or mineral metabolism Key Exclusion Criteria Part 2: 1. Previous treatment with an enzyme replacement therapy (ERT) or any advanced therapeutic agent (e.g., gene therapy) for the treatment of hypophosphatasia (HPP) or any treatment for osteoporotic diseases 2. Previous exposure to any medication or investigational agent potentially affecting bone structure, muscle volume, muscle strength, or muscle or nerve function 3. Diagnosis of hyperparathyroidism 4. Diagnosis of hypoparathyroidism, unless secondary to HPP 5. New fracture within 12 weeks before first dosing
Where it's running
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Universitätsklinikum Würzburg
Würzburg, Germany Recruiting -
New Zealand Clinical Research
Grafton, Auckland, New Zealand Recruiting -
Fortrea Clinical Research Unit
Leeds, United Kingdom Recruiting
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.