Clinical Trial: First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: 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-Administe
Brief 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.
Detailed Summary:
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 a chaperone (AT2221).
The study aims to evaluate safety, tolerability, pharmacodynamics (PD), and immunogenicity of ATB200 co-administered with AT2221. The study will be conducted in 3 stages.
In Stage 1, safety, tolerability, and PK will be evaluated following sequential single ascending doses of intravenously infused ATB200.
In Stage 2, safety, tolerability, and PK will be evaluated following single- and multiple-ascending dose combinations of ATB200 and AT2221.
In Stage 3, long term safety and efficacy will be assessed following 24 month treatment of ATB200 co-administered with AT2221 (Miglustat)
No Muscle biopsies will be performed in this study.
Sponsor: Amicus Therapeutics
Current Primary Outcome:
- Plasma GAA activity levels as measured by maximum observed plasma concentration (Cmax). [ Time Frame: 18 Weeks ]
- Plasma GAA activity levels as measured by time to reach the maximum observed plasma concentration (tmax). [ Time Frame: 18 Weeks ]
- Plasma GAA activity levels as measured by area under the plasma-drug concentration time curve. [ Time Frame: 18 Weeks ]
- Safety and tolerability as measured by counts of Treatment Emergent Adverse Events (TEAEs), including Infusion Associated Reactions (IARs). [ Time Frame: 18 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Amicus Therapeutics
Dates:
Date Received: January 26, 2016
Date Started: January 2016
Date Completion: June 2019
Last Updated: March 15, 2017
Last Verified: February 2017