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



Status:Recruiting
Conditions:Diabetes
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:18 - 75
Updated:2/9/2019
Start Date:January 2016
End Date:September 2021
Contact:For Site
Email:PompeSiteInfo@amicusrx.com
Phone:609-662-2000

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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

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.

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.

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
We found this trial at
11
sites
Phoenix, Arizona 85028
Principal Investigator: Kumaraswamy Sivakumar
Phone: 480-314-1007
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Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: Barry Byrne
Phone: 352-273-6563
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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4200 Fifth Ave
Pittsburgh, Pennsylvania 15260
(412) 624-4141
Principal Investigator: Paula Clemens
Phone: 412-648-9762
University of Pittsburgh The University of Pittsburgh is a state-related research university, founded as the...
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Decatur, Georgia 30033
Principal Investigator: William Wilcox
Phone: 404-727-3201
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2301 Erwin Rd
Durham, North Carolina 27710
919-684-8111
Principal Investigator: Priya Kishnani
Phone: 919-684-2036
Duke Univ Med Ctr As a world-class academic and health care system, Duke Medicine strives...
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Fairfax, Virginia 22030
Principal Investigator: Ozlem Goker-Alpan
Phone: 571-308-1925
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Grand Rapids, Michigan 49525
Principal Investigator: Khan Nedd
Phone: 616-954-0600
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Great Falls, Montana 59405
Principal Investigator: Karl Guter
Phone: 406-771-3311
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Newark, New Jersey 07103
Principal Investigator: Xue Ming
Phone: 973-972-2922
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North Adelaide, South Australia 5006
Principal Investigator: Drago Bratkovic
Phone: 61 8 81617295
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Orange, California 92868
Principal Investigator: Tahseen Mozaffar
Phone: 714-456-2332
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