Overview

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

Status:
Active, not recruiting

Trial end date:
2023-12-01

Target enrollment:

Participant gender:

Summary

This study is an international, multi-center, study of Pompe disease patients that are currently receiving enzyme-replacement therapy (ERT). The purpose of this study is to find out if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.

Phase:

Phase 1/Phase 2

Details

Lead Sponsor:

Amicus Therapeutics

Treatments:

Miglustat