NCT06114329

Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease

A Phase II, Open-label Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, and Efficacy in Males With Classic Fabry Disease Who Have Never Received Any Treatment

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 16 (estimated)
• Sponsor: AceLink Therapeutics, Inc. · Industry
• Sex: Male
• Age: 18 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase II, open-label study designed to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of AL01211 in male subjects with classic Fabry disease who have never received any treatment (eg. ERT or chaperone therapy). Eligible subjects will receive 182 days (26 weeks) of study treatment as the primary study period followed by an extension period. The total study duration for a subject is up to at most 2 years including the primary period of 26 weeks.

Conditions

• Fabry Disease

Interventions

Type	Name	Description
DRUG	AL01211	AL01211 is a novel, proprietary, selective GCS inhibitor with high potency against GCS with limited off target activity.

Timeline

• Start date: 2023-10-25
• Primary completion: 2024-12-01
• Completion: 2026-06-01
• First posted: 2023-11-02
• Last updated: 2024-07-30

Locations

6 sites across 1 country: China