Hypophosphatasia (HPP)Phase 1/2NCT07179640
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 Phase 1/2 study testing ALE1 in people with Hypophosphatasia (HPP), run by Alesta Therapeutics at 3 sites in 3 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Hypophosphatasia (HPP)
- What is being tested?
- ALE1
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 120 participants
- Where
- 3 sitesin 3 countries
- Study dates
- 30 Sep 2025to Jan 2027
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Participants are overtly healthy as determined by a medical evaluation
- No concurrent medical conditions or significant medical history, in the opinion of the investigator.
- 1. Documented ALPL gene variant
Who cannot join
- 1. History of conditions affecting bone or mineral metabolism
- 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
Trial locations and contacts
3 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
3 sites in 3 countries
Germany1 site, 1 recruiting
- Recruiting
Universitätsklinikum Würzburg
Würzburg, 97074
Dr. Lothar Seefried, +49 931 803 3575, lothar.seefried@uni-wuerzburg.de
New Zealand1 site, 1 recruiting
- Recruiting
New Zealand Clinical Research
Grafton, Auckland, 1010
Julia O'Sullivan, +64 0800 788 3437, horizon.auckland@nzcr.co.nz
United Kingdom1 site, 1 recruiting
- Recruiting
Fortrea Clinical Research Unit
Leeds
Firas Almazedi, MBChB, MSc, CPI, DipPharmMed, 0113 394 5200
About this study
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
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).
- Conditions
- Hypophosphatasia (HPP)
- Being tested
- ALE1, Placebo
- Keywords
- hypophosphatasia
- Medical subject headings
- Hypophosphatasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALE1Drug
Specified dose on specified days
PlaceboDrug
Specified dose on specified days
| Study group | Role | What they receive |
|---|---|---|
| Part 1 Cohort A | Experimental | Not described Receives: Drug: ALE1; Drug: Placebo |
| Part 1 Cohort B | Experimental | Not described Receives: Drug: ALE1; Drug: Placebo |
| Part 2 Cohort A | Experimental | Not described Receives: Drug: ALE1; Drug: Placebo |
| Part 2 Cohort B | Experimental | Not described Receives: Drug: ALE1; Drug: Placebo |
| Part 1 Cohort C | Experimental | Not described Receives: Drug: ALE1; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the safety of ALE1 by assessing the number of treatment emergent adverse events (TEAEs)
Measured: From baseline up to day 16
Evaluate safety of ALE1 by assessing the presence of clinically significant changes in participants haematology parameters post-dose
Measured: From baseline up to day 16
Evaluate safety of ALE1 by assessing the presence of clinically significant changes in participants biochemistry parameters post-dose
Measured: From baseline up to day 16
Evaluate safety of ALE1 by assessing changes in heart rhythms via electrocardiogram
Measured: From baseline up to day 16
Evaluate safety of ALE 1 by assessing the presence of clinically significiant changes in participants vital signs
Measured: From baseline up to day 16
Secondary outcomes (10)
Pharmacokinetic parameter: area under the plasma concentration versus time curve (AUC (D0 - INF))
Measured: From baseline up to day 16
Pharmacokinetic parameter: time at which maximum plasma concentration occurs (Tmax)
Measured: From baseline up to day 16
Pharmacokinetic parameter: terminal elimination phase half-life (t(1/2))
Measured: From baseline up to day 16
Pharmacokinetic parameter: total clearance (CL/F)
Measured: From baseline up to day 16
Pharmacokinetic parameter: volume of distribution (Vd/F)
Measured: From baseline up to day 16
Change in pharmacodynamic biomarker levels in blood samples of ALE1
Measured: From baseline up to day 16
Dose proportionality of maximum observed plasma concentration (Cmax) Time at which maximum plasma concentration occurs (Tmax)
Measured: From baseline up to day 16
Dose proportionality of area under the plasma concentration versus time curve (AUC (D0 - INF))
Measured: From baseline up to day 16
Effect of food on area under the plasma concentration versus time curve (AUC (D0 - INF))
Measured: From baseline up to 16 days post dose
Effect of food on maximum observed plasma concentration (Cmax)
Measured: From baseline up to day 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2025
- Study start30 Sep 2025
- Primary completionJan 2027 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hypophosphatasia (HPP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alesta Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07179640
- Sponsor's protocol ID
- ALE1-101
- Status last verified
- Jan 2026
- First submitted
- 4 Aug 2025
- Last updated
- 3 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07179640
- Acronym / sponsor ID
- ALE1-101
- Conditions
- Hypophosphatasia (HPP)
- Keywords
- hypophosphatasia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- Alesta TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Sep 2025 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 18 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Part 1 Cohort A | Experimental | Drug: ALE1, Drug: Placebo |
| Part 1 Cohort B | Experimental | Drug: ALE1, Drug: Placebo |
| Part 2 Cohort A | Experimental | Drug: ALE1, Drug: Placebo |
| Part 2 Cohort B | Experimental | Drug: ALE1, Drug: Placebo |
| Part 1 Cohort C | Experimental | Drug: ALE1, Drug: Placebo |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- Participants are overtly healthy as determined by a medical evaluation
- No concurrent medical conditions or significant medical history, in the opinion of the investigator.
- 1. Documented ALPL gene variant
Key exclusion criteria (6 listed)
- 1. History of conditions affecting bone or mineral metabolism
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALE1Specified dose on specified days | Drug | Part 1 Cohort A, Part 1 Cohort B, Part 1 Cohort C, Part 2 Cohort A, Part 2 Cohort B |
| PlaceboSpecified dose on specified days | Drug | Part 1 Cohort A, Part 1 Cohort B, Part 1 Cohort C, Part 2 Cohort A, Part 2 Cohort B |
Primary outcome
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
10 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Aug 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ALE1 in people with Hypophosphatasia (HPP), run by Alesta Therapeutics at 3 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Germany, New Zealand, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate the safety of ALE1 by assessing the number of treatment emergent adverse events (TEAEs) (time frame: From baseline up to day 16). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Aug 2026.