Clinical trial NCT04138277: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Pompe Disease (Late-onset)
Keywords: Pompe
rhGAA
Lead sponsor: Amicus Therapeutics
Sponsor class: INDUSTRY
First posted: 2019-10-24
Enrollment: 119
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Proportion of participants with Treatment Emergent Adverse Events (TEAE)
timeFrame: baseline, up to approximately 4 years
Secondary outcomes:
measure: 6-Minute Walk Test
timeFrame: baseline, up to approximately 4 years
measure: Pulmonary Function - Forced vital capacity (FVC)
timeFrame: baseline, up to approximately 4 years
measure: Change from baseline in muscle strength measured by Quantitative Muscle Strength testing
timeFrame: baseline, up to approximately 4 years
measure: Change from baseline in muscle strength measured by Manual Muscle Strength testing
timeFrame: baseline, up to approximately 4 years
measure: The Rasch-built Pompe-specific activity (R-PAct) questionnaires
timeFrame: baseline, up to approximately 4 years
measure: EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires
timeFrame: baseline, up to approximately 4 years
measure: Change from baseline in scores of PROMIS - physical function questionnaire
timeFrame: baseline, up to approximately 4 years
measure: Change from baseline in scores of PROMIS - fatigue questionnaire
timeFrame: baseline, up to approximately 4 years
measure: Change from baseline in scores of PROMIS - dyspnea questionnaire
timeFrame: baseline, up to approximately 4 years
measure: Change from baseline in scores of PROMIS - upper extremity questionnaire
timeFrame: baseline, up to approximately 4 years
measure: Motor Function - Gait, Stairs, Gower, Chair (GSGC) test
timeFrame: baseline, up to approximately 4 years
measure: Physician's Global Impression of Change
timeFrame: baseline, up to approximately 4 years
measure: Subject's Global Impression of Change
timeFrame: baseline, up to approximately 4 years
measure: Change from baseline Biomarker -CK
timeFrame: baseline, up to approximately 4 years
measure: Change from baseline Biomarker -uHex4
timeFrame: baseline, up to approximately 4 years
measure: Immunogenicity: Incidence of neutralizing
timeFrame: baseline, up to approximately 4 years
measure: Immunogenicity: anti-drug antibodies
timeFrame: baseline, up to approximately 4 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: University of Arkansas for Medical Sciences
state: Arkansas
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Gainesville
country: United States
facility: University of Florida Clinical Research Center
state: Florida
city: Atlanta
country: United States
facility: Emory Clinic
state: Georgia
city: Indianapolis
country: United States
facility: IU Health Neuroscience Center
state: Indiana
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Billings
country: United States
facility: Billings Clinic
state: Montana
city: Hackensack
country: United States
facility: Hackensack University Medical Center
state: New Jersey
city: Great Neck
country: United States
facility: Northwell Health
state: New York
city: New York
country: United States
facility: NYU School of Medicine
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cincinnati
country: United States
facility: University of Cincinnati Gardner Neuroscience Institute
state: Ohio
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Columbus
country: United States
facility: The Ohio State University Wexner Medical Center
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Montefiore Clinical and Translational Research Center
state: Pennsylvania
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Fairfax
country: United States
facility: Lysosomal and Rare Disorders Research and Treatment Center, Inc.
state: Virginia
city: Buenos Aires
country: Argentina
facility: Hospital Universitario Austral
city: Westmead
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Brisbane
country: Australia
facility: Royal Brisbane & Women's Hospital
state: Queensland
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Clayton
country: Australia
facility: Monash Medical Centre
state: Victoria
city: Innsbruck
country: Austria
facility: Medizinische Universität Innsbruck
city: Leuven
country: Belgium
facility: UZ Leuven
city: Banja Luka
country: Bosnia and Herzegovina
facility: University Clinical Centre of the Republic of Srpska
state: The Republic Of Srpska
city: Calgary
country: Canada
facility: Alberta Children's Hospital, Heritage Medical Research Clinic
state: Alberta
city: Hamilton
country: Canada
facility: McMaster University Medical Centre
state: Ontario
city: Aarhus
country: Denmark
facility: Aarhus Universitetshospital
city: Bron
country: France
facility: Hôpital Pierre Wertheimer
city: Garches
country: France
facility: Hôpital Raymond Poincaré
city: Lille
country: France
facility: Hôpital Salengro
city: Marseille
country: France
facility: Hôpital de la Timone
city: Nice
country: France
facility: Hôpital Pasteur 2
city: Bonn
country: Germany
facility: Universitätsklinikum Bonn
city: München
country: Germany
facility: Friedrich-Baur Institut
city: Münster
country: Germany
facility: Universitätsklinikum Münster
city: Athens
country: Greece
facility: Eginition Hospital
city: Budapest
country: Hungary
facility: Semmelweis University
city: Pécs
country: Hungary
facility: University of Pécs
city: Szeged
country: Hungary
facility: University of Szeged
city: Messina
country: Italy
facility: UOC di Neurologia e Malattie Neuromuscolari
city: Napoli
country: Italy
facility: Universitaria Federico II
city: Fukuoka
country: Japan
facility: Fukuoka University Hospital
city: Kagashima
country: Japan
facility: Kagoshima University Hospital
city: Osaka
country: Japan
facility: Izumi City General Hospital
city: Sapporo
country: Japan
facility: Hokkaido University Hospital
city: Tokyo
country: Japan
facility: The Jikei University Hospital
city: Yangsan
country: Korea, Republic of
facility: Pusan National University
state: Gyeongsangnam-do
city: Rotterdam
country: Netherlands
facility: Erasmus MC
city: Auckland
country: New Zealand
facility: University of Auckland
city: Rzeszów
country: Poland
facility: Centrum Medyczne Medyk
state: Podkarpackie
city: Ljubljana
country: Slovenia
facility: University Medical Centre Ljubljana
city: Barcelona
country: Spain
facility: Hospital de la Santa Creu I Sant Pau
city: Gothenburg
country: Sweden
facility: Sahlgrenska University Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Birmingham
country: United Kingdom
facility: Queen Elizabeth Hospital Birmingham
city: Cambridge
country: United Kingdom
facility: Cambridge University Hospitals NHS Foundation Trust
city: London
country: United Kingdom
facility: Royal Free Hospital NHS Foundation Trust
city: Salford
country: United Kingdom
facility: Salford Royal NHS Foundation Trust
Study officials:
References: