ALE1 for Hypophosphatasia

Inclusion criteria

• {"criterion_text":"- Aged ≥ 18 to ≤ 65 years (inclusive) at the time of signing the ICF."}
• {"criterion_text":"- Part 1: 1) Participants are overtly healthy as determined by a medical evaluation."}
• {"criterion_text":"- Part 1: 2) No concurrent medical conditions or significant medical history, in the opinion of the investigator."}
• {"criterion_text":"- Part 2: 1) Documented ALPL gene variant."}

Exclusion criteria

• {"criterion_text":"- Part 1: History of conditions affecting bone or mineral metabolism."}
• {"criterion_text":"- Part 2: 1) Previous treatment with an enzyme replacement therapy (ERT) or any advanced therapeutic agent (e.g., gene therapy) for the treatment of HPP or any treatment for osteoporotic diseases."}
• {"criterion_text":"- Part 2: 2) Previous exposure to any medication or investigational agent potentially affecting bone structure, muscle volume, muscle strength, or muscle or nerve function."}
• {"criterion_text":"- Part 2: 3) Diagnosis of hyperparathyroidism."}
• {"criterion_text":"- Part 2: 4) Diagnosis of hypoparathyroidism, unless secondary to HPP."}
• {"criterion_text":"- Part 2: 5) New fracture within 12 weeks before first dosing."}

Primary endpoints

• {"endpoint_text":"- Number and severity of treatment emergent adverse events (TEAEs) in healthy participants and patients with hypophosphatasia (HPP) with increasing oral doses of ALE1","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Pharmacokinetic parameters of single and multiple ascending doses of ALE1.","definition_or_measurement_approach":""}
• {"endpoint_text":"- Pharmacodynamic biomarker levels in blood samples of single and multiple ascending doses of ALE1.","definition_or_measurement_approach":""}
• {"endpoint_text":"- Dose proportionality in pharmacokinetic parameters in single and multiple ascending doses of ALE1.","definition_or_measurement_approach":""}
• {"endpoint_text":"- Effect of food on ALE1 pharmacokinetic parameters.","definition_or_measurement_approach":""}

Germany

Earliest CTIS Part Ii Submission Date

30-10-2025

Latest Decision Or Authorization Date

06-05-2026

Processing Time Days

188

Number Of Sites

2

Number Of Participants

24

Primary sponsor

Full Name

Alesta Therapeutics B.V.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Netherlands

Contract research organisations

Name

Novotech Clinical Research (Cyprus) Limited

Responsibilities

codes: 1,11,12,2,5,8

Name

QPS Netherlands B.V.

Responsibilities

PD analysis

Name

4g Clinical LLC

Responsibilities

code 3

Third parties

• {"country":"United Kingdom","full_name":"Congenica Ltd","duties_or_roles":"ALPL genotyping","organisation_type":"Health care"}
• {"country":"United States","full_name":"ClinCapture","duties_or_roles":"code 7","organisation_type":"Industry"}
• {"country":"United States","full_name":"Clario","duties_or_roles":"ECG analysis","organisation_type":"Health care"}
• {"country":"Netherlands","full_name":"QPS Netherlands B.V.","duties_or_roles":"PD analysis","organisation_type":"Pharmaceutical company"}
• {"country":"Cyprus","full_name":"Novotech Clinical Research (Cyprus) Limited","duties_or_roles":"codes: 1,11,12,2,5,8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"4g Clinical LLC","duties_or_roles":"code 3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Australia","full_name":"360 Biolabs Pty Limited","duties_or_roles":"PK analysis","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Thomas Jefferson University","duties_or_roles":"code 13","organisation_type":"Educational Institution"}
• {"country":"United Kingdom","full_name":"Acm Global Central Laboratory Limited","duties_or_roles":"PD analysis; code 4","organisation_type":"Laboratory/Research/Testing facility"}