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

Who is this study for? Adult patients with Pompe disease
Status: Active, not recruiting
Location: See all (19) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: No
View:

• 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

Locations
United States
Arizona
Neuromuscular Research Centre
Phoenix
California
University of California Irvine
Orange
Florida
University of Florida
Gainesville
Georgia
Emory University Division of Medical Genetics
Decatur
Michigan
Infusion Associates
Grand Rapids
Montana
Great Falls Clinic, LLP
Great Falls
North Carolina
Duke University Medical Center
Durham
New Jersey
Rutgers New Jersey Medical School
Newark
Pennsylvania
Perelman Center for Advanced Medicine
Philadelphia
University of Pittsburgh
Pittsburgh
Abramson Cancer Center Chester County Hospital
West Chester
Virginia
Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)
Fairfax
Other Locations
Australia
Womens & Childrens Hospital, Adelaide
North Adelaide
Germany
University Children's Hospital Department of Neuropediatrics and Inborn Metabolic Disorders, St. Josefs-Hospital
Bochum
Friedrich-Baur-Institure, Dep of Neurology - University Munich
Munich
Netherlands
Erasmus Medical Center
Rotterdam
New Zealand
School of Medicine, University of Auckland
Auckland
United Kingdom
University Hospital Birmingham NHS Foundation Trust, Queen Elizabeth Medical Center
Birmingham
Salford Royal NHS Foundation Trust
Salford
Time Frame
Start Date: January 2016
Estimated Completion Date: December 2023
Participants
Target number of participants: 32
Treatments
Experimental: ATB200
In Stage 1, safety, tolerability, and PK will be evaluated following sequential single ascending doses of intravenously infused ATB200 for 3 dosing periods.
Experimental: ATB200 + AT2221
In Stage 2, safety, tolerability, and PK will be evaluated following single- and multiple-ascending dose combinations of ATB200 co-administered with AT2221 (Miglustat) In Stage 3, long term safety and efficacy will be assessed following 24 month treatment of ATB200 co-administered with AT2221 (Miglustat)
Authors
William Wilcox, Paula Clemens, Mark Roberts, Barry Byrne, Priya Kishnani, Khan Nedd, Karl Guter, Ozlem Goker-Alpan, Tahseen Mozaffar, Benedikt Schoser, Nesrin Karabul, Schwenkreis Peter, Xue Ming, Tarekegn Hiwot, Kumaraswamy Sivakumar, Drago Bratkovic, A.T. van der Ploeg
Related Therapeutic Areas
Sponsors
Leads: Amicus Therapeutics

This content was sourced from clinicaltrials.gov

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