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
Grand Rapids, Michigan 49525
Principal Investigator: Khan Nedd
Phone: 616-954-0600
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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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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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Phoenix, Arizona 85028
Principal Investigator: Kumaraswamy Sivakumar
Phone: 480-314-1007
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