Summary

for people ages 18-75 (full criteria)
at Orange, California and other locations
study started
estimated completion

Description

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.

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-Administered With Oral AT2221 in Adult Subjects With Pompe Disease

Details

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)

In Stage 4, treatment period will begin at the end of Stage 3 and will continue as open label extension until commercialization, study discontinuation or subject withdrawal, with functional assessments every 6 months

No Muscle biopsies will be performed in this study.

Keywords

Pompe DiseasePompe, rhGAAGlycogen Storage Disease Type IIMiglustatATB200AT2221ATB200 + AT2221

Eligibility

For people ages 18-75

Key Inclusion Criteria:

  • Male and female subjects between 18 and 75years of age, inclusive
  • Diagnosis of Pompe disease

Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):

  • Has received ERT with alglucosidase alfa for the previous 2-6 years, inclusive
  • Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week
  • Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
  • Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

ERT-experienced subjects (non-ambulatory):

  • Has received ERT with alglucosidase alfa (Myozyme/Lumizyme) for ≥2 years
  • Is wheelchair-bound

ERT-naïve subjects (ambulatory):

  • Must be able to walk 200-500 meters on the 6MWT
  • Has upright FVC must be 30% to 80% of predicted normal value
  • Subject has never received alglucosidase alfa

Enzyme Replacement Therapy (ERT)-experienced subject (ambulatory):

  • Has received ERT with alglucosidase alfa for >7years, inclusive
  • Subject is currently receiving alglucosidase alfa (Myozyme/Lumizyme), at a frequency of once every other week
  • Must be able to walk 200-500 meters on the 6-Minute Walk Test (6MWT )
  • Has upright Forced Vial Capacity (FVC) 30% to 80% of predicted normal value

Exclusion Criteria:

  • Subject has received treatment with prohibited medications within 30 days of Baseline Visit
  • Subject, if female, is pregnant or breastfeeding at screening
  • Subject, whether male or female, planning to conceive a child during the study
  • Subject has a medical or any other extenuating condition or circumstance that may, in opinion of investigator, pose an undue safety risk to the subject or compromise his/her ability to comply with protocol requirements
  • Subject has a history of allergy or sensitivity to miglustat or other iminosugars
  • Subjects with active systemic autoimmune disease such as lupus, scleroderma, or rheumatoid arthritis. All subjects with autoimmune disease must be discussed with the Amicus Medical Monitor
  • Subjects with active bronchial asthma. All subjects with bronchial asthma must be discussed with the Amicus Medical Monitor

Locations

  • University of California Irvineaccepting new patients
    OrangeCalifornia92868United States
  • Neuromuscular Research Centreaccepting new patients
    PhoenixArizona85028United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Amicus Therapeutics
ID
NCT02675465
Phase
Phase 1/2
Study Type
Interventional
Last Updated