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