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A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

Primary Purpose

Pompe Disease (Late-onset)

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
AT2221
ATB200
Sponsored by
Amicus Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Pompe Disease (Late-onset) focused on measuring Pompe, rhGAA

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

1. Subject must have completed Study ATB200-03.

Exclusion Criteria

  1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
  2. Subject, if female, is pregnant or breastfeeding.
  3. Subject, whether male or female, is planning to conceive a child during the study.

Sites / Locations

  • University of Arkansas for Medical Sciences
  • University of California, Irvine
  • University of Florida Clinical Research Center
  • Emory Clinic
  • IU Health Neuroscience Center
  • Washington University School of Medicine
  • Billings Clinic
  • Hackensack University Medical Center
  • Northwell Health
  • NYU School of Medicine
  • Duke University Medical Center
  • University of Cincinnati Gardner Neuroscience Institute
  • Cincinnati Children's Hospital
  • The Ohio State University Wexner Medical Center
  • University of Pennsylvania
  • UPMC Montefiore Clinical and Translational Research Center
  • University of Utah
  • Lysosomal and Rare Disorders Research and Treatment Center, Inc.
  • Hospital Universitario Austral
  • Westmead Hospital
  • Royal Brisbane & Women's Hospital
  • Royal Adelaide Hospital
  • Monash Medical Centre
  • Medizinische Universität Innsbruck
  • UZ Leuven
  • University Clinical Centre of the Republic of Srpska
  • Heritage Medical Research Clinic
  • McMaster University Medical Centre
  • Aarhus Universitetshospital
  • Hôpital Pierre Wertheimer
  • Hôpital Raymond Poincaré
  • Hôpital Salengro
  • Hôpital de la Timone
  • Hôpital Pasteur 2
  • Universitätsklinikum Bonn
  • Friedrich-Baur Institut
  • Universitätsklinikum Münster
  • Eginition Hospital
  • Semmelweis University
  • University of Pécs
  • University of Szeged
  • UOC di Neurologia e Malattie Neuromuscolari
  • Universitaria Federico II
  • Fukuoka University Hospital
  • Kagoshima University Hospital
  • Izumi City General Hospital
  • Hokkaido University Hospital
  • The Jikei University Hospital
  • Pusan National University
  • Erasmus MC
  • University of Auckland
  • Centrum Medyczne Medyk
  • University Medical Centre Ljubljana
  • Hospital de la Santa Creu I Sant Pau
  • Sahlgrenska University Hospital
  • National Taiwan University Hospital
  • Queen Elizabeth Hospital Birmingham
  • Cambridge University Hospitals NHS Foundation Trust
  • Royal Free Hospital NHS Foundation Trust
  • Salford Royal NHS Foundation Trust

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ATB200/AT2221

Arm Description

Participants received ATB200 co-administered with AT2221 capsule (Miglustat)

Outcomes

Primary Outcome Measures

Proportion of participants with Treatment Emergent Adverse Events (TEAE)

Secondary Outcome Measures

6-Minute Walk Test
Change in 6MWD from baseline to assess the efficacy of ATB200/AT2221 co-administration
Pulmonary Function - Forced vital capacity (FVC)
Change from baseline in FVC (sitting) to assess the efficacy of ATB200/AT2221 co-administration
Change from baseline in muscle strength measured by Quantitative Muscle Strength testing
Change from baseline in muscle strength measured by Manual Muscle Strength testing
The Rasch-built Pompe-specific activity (R-PAct) questionnaires
Change in R-Pact from baseline to assess the efficacy of ATB200/AT2221 co-administration
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires
Change from baseline in scores of EQ-5D-5L questionnaire to assess the efficacy of ATB200/AT2221 co-administration.
Change from baseline in scores of PROMIS - physical function questionnaire
Change from baseline in scores of PROMIS - fatigue questionnaire
Change from baseline in scores of PROMIS - dyspnea questionnaire
Change from baseline in scores of PROMIS - upper extremity questionnaire
Motor Function - Gait, Stairs, Gower, Chair (GSGC) test
Change from baseline in GSGC score to assess the efficacy of ATB200/AT2221 co-administration.
Physician's Global Impression of Change
Change in the Physician's Global Impression of Change (PGIC) evaluation to assess the efficacy of ATB200/AT2221 co-administration.
Subject's Global Impression of Change
Change from baseline in scores of Subject's Global Impression of Change (SGIC) questionnaire to assess the efficacy of ATB200/AT2221 co-administration.
Change from baseline Biomarker -CK
Change from baseline Biomarker -uHex4
Immunogenicity: Incidence of neutralizing
Immunogenicity: anti-drug antibodies

Full Information

First Posted
October 18, 2019
Last Updated
August 25, 2023
Sponsor
Amicus Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT04138277
Brief Title
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
Official Title
A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 18, 2019 (Actual)
Primary Completion Date
December 2023 (Anticipated)
Study Completion Date
December 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amicus Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. Study Description

Brief Summary
This is a multicenter, international open-label extension study of ATB200/AT2221 in adult subjects with late-onset Pompe disease (LOPD) who completed Study ATB200-03.
Detailed Description
This is an open label extension study for subjects who completed the ATB200-03 study. The subjects will stay in this study until regulatory approval or marketing authorization and/or commercialization in the participating subject's country.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pompe Disease (Late-onset)
Keywords
Pompe, rhGAA

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
110 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
ATB200/AT2221
Arm Type
Experimental
Arm Description
Participants received ATB200 co-administered with AT2221 capsule (Miglustat)
Intervention Type
Drug
Intervention Name(s)
AT2221
Other Intervention Name(s)
Miglustat
Intervention Description
Participants received ATB200 co-administered with AT2221 (Miglustat)
Intervention Type
Biological
Intervention Name(s)
ATB200
Intervention Description
Enzyme Replacement Therapy via intravenous infusion
Primary Outcome Measure Information:
Title
Proportion of participants with Treatment Emergent Adverse Events (TEAE)
Time Frame
baseline, up to approximately 4 years
Secondary Outcome Measure Information:
Title
6-Minute Walk Test
Description
Change in 6MWD from baseline to assess the efficacy of ATB200/AT2221 co-administration
Time Frame
baseline, up to approximately 4 years
Title
Pulmonary Function - Forced vital capacity (FVC)
Description
Change from baseline in FVC (sitting) to assess the efficacy of ATB200/AT2221 co-administration
Time Frame
baseline, up to approximately 4 years
Title
Change from baseline in muscle strength measured by Quantitative Muscle Strength testing
Time Frame
baseline, up to approximately 4 years
Title
Change from baseline in muscle strength measured by Manual Muscle Strength testing
Time Frame
baseline, up to approximately 4 years
Title
The Rasch-built Pompe-specific activity (R-PAct) questionnaires
Description
Change in R-Pact from baseline to assess the efficacy of ATB200/AT2221 co-administration
Time Frame
baseline, up to approximately 4 years
Title
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires
Description
Change from baseline in scores of EQ-5D-5L questionnaire to assess the efficacy of ATB200/AT2221 co-administration.
Time Frame
baseline, up to approximately 4 years
Title
Change from baseline in scores of PROMIS - physical function questionnaire
Time Frame
baseline, up to approximately 4 years
Title
Change from baseline in scores of PROMIS - fatigue questionnaire
Time Frame
baseline, up to approximately 4 years
Title
Change from baseline in scores of PROMIS - dyspnea questionnaire
Time Frame
baseline, up to approximately 4 years
Title
Change from baseline in scores of PROMIS - upper extremity questionnaire
Time Frame
baseline, up to approximately 4 years
Title
Motor Function - Gait, Stairs, Gower, Chair (GSGC) test
Description
Change from baseline in GSGC score to assess the efficacy of ATB200/AT2221 co-administration.
Time Frame
baseline, up to approximately 4 years
Title
Physician's Global Impression of Change
Description
Change in the Physician's Global Impression of Change (PGIC) evaluation to assess the efficacy of ATB200/AT2221 co-administration.
Time Frame
baseline, up to approximately 4 years
Title
Subject's Global Impression of Change
Description
Change from baseline in scores of Subject's Global Impression of Change (SGIC) questionnaire to assess the efficacy of ATB200/AT2221 co-administration.
Time Frame
baseline, up to approximately 4 years
Title
Change from baseline Biomarker -CK
Time Frame
baseline, up to approximately 4 years
Title
Change from baseline Biomarker -uHex4
Time Frame
baseline, up to approximately 4 years
Title
Immunogenicity: Incidence of neutralizing
Time Frame
baseline, up to approximately 4 years
Title
Immunogenicity: anti-drug antibodies
Time Frame
baseline, up to approximately 4 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 1. Subject must have completed Study ATB200-03. Exclusion Criteria Subject plans to receive gene therapy or participate in another interventional study for Pompe disease. Subject, if female, is pregnant or breastfeeding. Subject, whether male or female, is planning to conceive a child during the study.
Facility Information:
Facility Name
University of Arkansas for Medical Sciences
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72205
Country
United States
Facility Name
University of California, Irvine
City
Irvine
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
University of Florida Clinical Research Center
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Emory Clinic
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
IU Health Neuroscience Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Billings Clinic
City
Billings
State/Province
Montana
ZIP/Postal Code
59101
Country
United States
Facility Name
Hackensack University Medical Center
City
Hackensack
State/Province
New Jersey
ZIP/Postal Code
07061
Country
United States
Facility Name
Northwell Health
City
Great Neck
State/Province
New York
ZIP/Postal Code
11021
Country
United States
Facility Name
NYU School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10017
Country
United States
Facility Name
Duke University Medical Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
University of Cincinnati Gardner Neuroscience Institute
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45219
Country
United States
Facility Name
Cincinnati Children's Hospital
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
The Ohio State University Wexner Medical Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
UPMC Montefiore Clinical and Translational Research Center
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
City
Fairfax
State/Province
Virginia
ZIP/Postal Code
22030
Country
United States
Facility Name
Hospital Universitario Austral
City
Buenos Aires
ZIP/Postal Code
B1629ODT
Country
Argentina
Facility Name
Westmead Hospital
City
Westmead
State/Province
New South Wales
Country
Australia
Facility Name
Royal Brisbane & Women's Hospital
City
Brisbane
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
Royal Adelaide Hospital
City
Adelaide
State/Province
South Australia
ZIP/Postal Code
5000
Country
Australia
Facility Name
Monash Medical Centre
City
Clayton
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Medizinische Universität Innsbruck
City
Innsbruck
ZIP/Postal Code
A-6020
Country
Austria
Facility Name
UZ Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
University Clinical Centre of the Republic of Srpska
City
Banja Luka
State/Province
The Republic Of Srpska
ZIP/Postal Code
78000
Country
Bosnia and Herzegovina
Facility Name
Heritage Medical Research Clinic
City
Calgary
State/Province
Alberta
ZIP/Postal Code
T3B 6A8
Country
Canada
Facility Name
McMaster University Medical Centre
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Aarhus Universitetshospital
City
Aarhus
Country
Denmark
Facility Name
Hôpital Pierre Wertheimer
City
Bron
ZIP/Postal Code
69577
Country
France
Facility Name
Hôpital Raymond Poincaré
City
Garches
ZIP/Postal Code
92380
Country
France
Facility Name
Hôpital Salengro
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
Hôpital de la Timone
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Hôpital Pasteur 2
City
Nice
ZIP/Postal Code
06001
Country
France
Facility Name
Universitätsklinikum Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Friedrich-Baur Institut
City
München
ZIP/Postal Code
80336
Country
Germany
Facility Name
Universitätsklinikum Münster
City
Münster
ZIP/Postal Code
48149
Country
Germany
Facility Name
Eginition Hospital
City
Athens
ZIP/Postal Code
11528
Country
Greece
Facility Name
Semmelweis University
City
Budapest
Country
Hungary
Facility Name
University of Pécs
City
Pécs
Country
Hungary
Facility Name
University of Szeged
City
Szeged
Country
Hungary
Facility Name
UOC di Neurologia e Malattie Neuromuscolari
City
Messina
ZIP/Postal Code
98124
Country
Italy
Facility Name
Universitaria Federico II
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Facility Name
Fukuoka University Hospital
City
Fukuoka
ZIP/Postal Code
814-0180
Country
Japan
Facility Name
Kagoshima University Hospital
City
Kagashima
ZIP/Postal Code
890-8520
Country
Japan
Facility Name
Izumi City General Hospital
City
Osaka
ZIP/Postal Code
594 0073
Country
Japan
Facility Name
Hokkaido University Hospital
City
Sapporo
ZIP/Postal Code
060 8648
Country
Japan
Facility Name
The Jikei University Hospital
City
Tokyo
ZIP/Postal Code
105-8471
Country
Japan
Facility Name
Pusan National University
City
Yangsan
State/Province
Gyeongsangnam-do
ZIP/Postal Code
50612
Country
Korea, Republic of
Facility Name
Erasmus MC
City
Rotterdam
ZIP/Postal Code
3015 GD
Country
Netherlands
Facility Name
University of Auckland
City
Auckland
ZIP/Postal Code
1142
Country
New Zealand
Facility Name
Centrum Medyczne Medyk
City
Rzeszów
State/Province
Podkarpackie
ZIP/Postal Code
35-326
Country
Poland
Facility Name
University Medical Centre Ljubljana
City
Ljubljana
ZIP/Postal Code
1000
Country
Slovenia
Facility Name
Hospital de la Santa Creu I Sant Pau
City
Barcelona
Country
Spain
Facility Name
Sahlgrenska University Hospital
City
Gothenburg
ZIP/Postal Code
41345
Country
Sweden
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
Queen Elizabeth Hospital Birmingham
City
Birmingham
ZIP/Postal Code
B15 2TH
Country
United Kingdom
Facility Name
Cambridge University Hospitals NHS Foundation Trust
City
Cambridge
ZIP/Postal Code
CB2 0QQ
Country
United Kingdom
Facility Name
Royal Free Hospital NHS Foundation Trust
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Salford Royal NHS Foundation Trust
City
Salford
ZIP/Postal Code
M6 8HD
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

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A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD

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