NCT07179640

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).

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
120

participants targeted

Target at P75+ for phase_1

Timeline
8mo left

Started Sep 2025

Geographic Reach
2 countries

2 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress48%
Sep 2025Jan 2027

First Submitted

Initial submission to the registry

August 4, 2025

Completed
2 months until next milestone

First Posted

Study publicly available on registry

September 18, 2025

Completed
12 days until next milestone

Study Start

First participant enrolled

September 30, 2025

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2027

Last Updated

January 30, 2026

Status Verified

January 1, 2026

Enrollment Period

1.3 years

First QC Date

August 4, 2025

Last Update Submit

January 28, 2026

Conditions

Hypophosphatasia (HPP)

Keywords

hypophosphatasia

Outcome Measures

Primary Outcomes (5)

  • 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 Outcomes (10)

  • 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

  • +5 more secondary outcomes

Study Arms (5)

Part 1 Cohort A

EXPERIMENTAL
Drug: ALE1Drug: Placebo

Part 1 Cohort B

EXPERIMENTAL
Drug: ALE1Drug: Placebo

Part 2 Cohort A

EXPERIMENTAL
Drug: ALE1Drug: Placebo

Part 2 Cohort B

EXPERIMENTAL
Drug: ALE1Drug: Placebo

Part 1 Cohort C

EXPERIMENTAL
Drug: ALE1Drug: Placebo

Interventions

ALE1DRUG

Specified dose on specified days

Part 1 Cohort APart 1 Cohort BPart 1 Cohort CPart 2 Cohort APart 2 Cohort B
PlaceboDRUG

Specified dose on specified days

Part 1 Cohort APart 1 Cohort BPart 1 Cohort CPart 2 Cohort APart 2 Cohort B

Eligibility Criteria

Age18 Years - 50 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64)

You may qualify if:

  • Participants are overtly healthy as determined by a medical evaluation
  • No concurrent medical conditions or significant medical history, in the opinion of the investigator.
  • \. Documented ALPL gene variant

You may not qualify if:

  • \. 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

New Zealand Clinical Research

Grafton, Auckland, 1010, New Zealand

RECRUITING

Fortrea Clinical Research Unit

Leeds, United Kingdom

RECRUITING

MeSH Terms

Conditions

Hypophosphatasia

Condition Hierarchy (Ancestors)

Metal Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 4, 2025

First Posted

September 18, 2025

Study Start

September 30, 2025

Primary Completion (Estimated)

January 1, 2027

Study Completion (Estimated)

January 1, 2027

Last Updated

January 30, 2026

Record last verified: 2026-01

Data Sharing

IPD Sharing
Will not share

Locations

NZ(1)GB(1)