A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1

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

Status

Recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT07179640

Enrollment

120

Registered

2025-09-18

Start date

2025-09-30

Completion date

2027-01-01

Last updated

2026-08-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hypophosphatasia (HPP)

Keywords

hypophosphatasia

Brief summary

This is a phase 1/2a randomised, placebo controlled, double-blind study investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALE1 on healthy adult subjects and adult patients with Hypophosphatasia (HPP).

Interventions

DRUGALE1

Specified dose on specified days

DRUGPlacebo

Specified dose on specified days

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

18 Years to 50 Years

Healthy volunteers

Yes

Inclusion 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

Design outcomes

Primary

Measure	Time frame
Evaluate the safety of ALE1 by assessing the number of treatment emergent adverse events (TEAEs)	From baseline up to day 16
Evaluate safety of ALE1 by assessing the presence of clinically significant changes in participants haematology parameters post-dose	From baseline up to day 16
Evaluate safety of ALE1 by assessing the presence of clinically significant changes in participants biochemistry parameters post-dose	From baseline up to day 16
Evaluate safety of ALE1 by assessing changes in heart rhythms via electrocardiogram	From baseline up to day 16
Evaluate safety of ALE 1 by assessing the presence of clinically significiant changes in participants vital signs	From baseline up to day 16

Secondary

Measure	Time frame
Pharmacokinetic parameter: area under the plasma concentration versus time curve (AUC (D0 - INF))	From baseline up to day 16
Pharmacokinetic parameter: time at which maximum plasma concentration occurs (Tmax)	From baseline up to day 16
Pharmacokinetic parameter: terminal elimination phase half-life (t(1/2))	From baseline up to day 16
Pharmacokinetic parameter: total clearance (CL/F)	From baseline up to day 16
Pharmacokinetic parameter: volume of distribution (Vd/F)	From baseline up to day 16
Change in pharmacodynamic biomarker levels in blood samples of ALE1	From baseline up to day 16
Dose proportionality of maximum observed plasma concentration (Cmax) Time at which maximum plasma concentration occurs (Tmax)	From baseline up to day 16
Dose proportionality of area under the plasma concentration versus time curve (AUC (D0 - INF))	From baseline up to day 16
Effect of food on area under the plasma concentration versus time curve (AUC (D0 - INF))	From baseline up to 16 days post dose
Effect of food on maximum observed plasma concentration (Cmax)	From baseline up to day 16

Countries

Germany, New Zealand, United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Aug 4, 2026