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

Alesta Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Hypophosphatasia (HPP)

Treatments: Drug: ALE1; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT07179640

ALE1-101

Details and patient eligibility

About

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).

Enrollment: 120 estimated patients

Sex: All

Ages: 18 to 50 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

120 participants in 5 patient groups

Part 1 Cohort A

Experimental group

Treatment:

Drug: Placebo

Drug: ALE1

Part 1 Cohort B

Experimental group

Treatment:

Drug: Placebo

Drug: ALE1

Part 2 Cohort A

Experimental group

Treatment:

Drug: Placebo

Drug: ALE1

Part 2 Cohort B

Experimental group

Treatment:

Drug: Placebo

Drug: ALE1

Part 1 Cohort C

Experimental group

Treatment:

Drug: Placebo

Drug: ALE1