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
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: ALE1, Placebo.
- Who it may be relevant to
- Registry conditions: Hypophosphatasia (HPP). Basic parameters: 18 years — 50 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Germany, New Zealand, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
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
Overview
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
- Drug ALE1
Specified dose on specified days - Drug Placebo
Specified dose on specified days
Primary outcome measures
- Evaluate the safety of ALE1 by assessing the number of treatment emergent adverse events (TEAEs) [Time frame: From baseline up to day 16]
- Evaluate safety of ALE1 by assessing the presence of clinically significant changes in participants haematology parameters post-dose [Time frame: From baseline up to day 16]
- Evaluate safety of ALE1 by assessing the presence of clinically significant changes in participants biochemistry parameters post-dose [Time frame: From baseline up to day 16]
- Evaluate safety of ALE1 by assessing changes in heart rhythms via electrocardiogram [Time frame: From baseline up to day 16]
- Evaluate safety of ALE 1 by assessing the presence of clinically significiant changes in participants vital signs [Time frame: From baseline up to day 16]
Secondary outcome measures (10)
- Pharmacokinetic parameter: area under the plasma concentration versus time curve (AUC (D0 - INF)) [Time frame: From baseline up to day 16]
- Pharmacokinetic parameter: time at which maximum plasma concentration occurs (Tmax) [Time frame: From baseline up to day 16]
- Pharmacokinetic parameter: terminal elimination phase half-life (t(1/2)) [Time frame: From baseline up to day 16]
- Pharmacokinetic parameter: total clearance (CL/F) [Time frame: From baseline up to day 16]
- Pharmacokinetic parameter: volume of distribution (Vd/F) [Time frame: From baseline up to day 16]
- Change in pharmacodynamic biomarker levels in blood samples of ALE1 [Time frame: From baseline up to day 16]
- Dose proportionality of maximum observed plasma concentration (Cmax) Time at which maximum plasma concentration occurs (Tmax) [Time frame: From baseline up to day 16]
- Dose proportionality of area under the plasma concentration versus time curve (AUC (D0 - INF)) [Time frame: From baseline up to day 16]
- Effect of food on area under the plasma concentration versus time curve (AUC (D0 - INF)) [Time frame: From baseline up to 16 days post dose]
- Effect of food on maximum observed plasma concentration (Cmax) [Time frame: From baseline up to day 16]
Eligibility criteria
Key Inclusion Criteria Part 1:
- Participants are overtly healthy as determined by a medical evaluation
- 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:
- 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
- Previous exposure to any medication or investigational agent potentially affecting bone structure, muscle volume, muscle strength, or muscle or nerve function
- Diagnosis of hyperparathyroidism
- Diagnosis of hypoparathyroidism, unless secondary to HPP
- New fracture within 12 weeks before first dosing
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
Germany · 1 center
- Universitätsklinikum Würzburg — Würzburg
New Zealand · 1 center
- New Zealand Clinical Research — Grafton
United Kingdom · 1 center
- Fortrea Clinical Research Unit — Leeds
Identifiers
NCT: NCT07179640 · ALE1-101