NCT02675465

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

An Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: Amicus Therapeutics · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.

Detailed description

This is an open-label, fixed-sequence, ascending-dose, first-in-human study to evaluate the effect of a highly targeted rhGAA (ATB200) co-administered with an enzyme stabilizer (AT2221). The study aims to evaluate safety, tolerability, pharmacokinetics (PK), efficacy, pharmacodynamics (PD), and immunogenicity of ATB200 co-administered with AT2221. Stage 1: evaluation of safety, tolerability, and PK following sequential single ascending doses of intravenously infused ATB200 Stage 2: evaluation of safety, tolerability, and PK following single- and multiple-ascending dose combinations of ATB200 and AT2221 Stage 3: evaluation of long term safety, tolerability, and efficacy following 24 month treatment of ATB200 co-administered with AT2221 Stage 4: open-label extension period with functional assessments every 6 months

Conditions

• Pompe Disease

Interventions

Type	Name	Description
DRUG	ATB200
DRUG	AT2221

Timeline

• Start date: 2016-04-01
• Primary completion: 2024-08-22
• Completion: 2024-08-22
• First posted: 2016-02-05
• Last updated: 2025-10-23
• Results posted: 2025-10-23

Locations

19 sites across 6 countries: United States, Australia, Germany, Netherlands, New Zealand, United Kingdom