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

Pompe Disease (Late-onset) Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04138277
• Other study ID #: ATB200-07
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: December 18, 2019
• Est. completion date: December 2024

Study information

• Verified date: August 2023
• Source: Amicus Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 110
• Est. completion date: December 2024
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Pompe Disease (Late-onset)

Intervention

Drug:
• AT2221
Participants received ATB200 co-administered with AT2221 (Miglustat)

Biological:
• ATB200
Enzyme Replacement Therapy via intravenous infusion

Locations

Country	Name	City	State
Argentina	Hospital Universitario Austral	Buenos Aires
Australia	Royal Adelaide Hospital	Adelaide	South Australia
Australia	Royal Brisbane & Women's Hospital	Brisbane	Queensland
Australia	Monash Medical Centre	Clayton	Victoria
Australia	Westmead Hospital	Westmead	New South Wales
Austria	Medizinische Universität Innsbruck	Innsbruck
Belgium	UZ Leuven	Leuven
Bosnia and Herzegovina	University Clinical Centre of the Republic of Srpska	Banja Luka	The Republic Of Srpska
Canada	Heritage Medical Research Clinic	Calgary	Alberta
Canada	McMaster University Medical Centre	Hamilton	Ontario
Denmark	Aarhus Universitetshospital	Aarhus
France	Hôpital Pierre Wertheimer	Bron
France	Hôpital Raymond Poincaré	Garches
France	Hôpital Salengro	Lille
France	Hôpital de la Timone	Marseille
France	Hôpital Pasteur 2	Nice
Germany	Universitätsklinikum Bonn	Bonn
Germany	Friedrich-Baur Institut	München
Germany	Universitätsklinikum Münster	Münster
Greece	Eginition Hospital	Athens
Hungary	Semmelweis University	Budapest
Hungary	University of Pécs	Pécs
Hungary	University of Szeged	Szeged
Italy	UOC di Neurologia e Malattie Neuromuscolari	Messina
Italy	Universitaria Federico II	Napoli
Japan	Fukuoka University Hospital	Fukuoka
Japan	Kagoshima University Hospital	Kagashima
Japan	Izumi City General Hospital	Osaka
Japan	Hokkaido University Hospital	Sapporo
Japan	The Jikei University Hospital	Tokyo
Korea, Republic of	Pusan National University	Yangsan	Gyeongsangnam-do
Netherlands	Erasmus MC	Rotterdam
New Zealand	University of Auckland	Auckland
Poland	Centrum Medyczne Medyk	Rzeszów	Podkarpackie
Slovenia	University Medical Centre Ljubljana	Ljubljana
Spain	Hospital de la Santa Creu I Sant Pau	Barcelona
Sweden	Sahlgrenska University Hospital	Gothenburg
Taiwan	National Taiwan University Hospital	Taipei
United Kingdom	Queen Elizabeth Hospital Birmingham	Birmingham
United Kingdom	Cambridge University Hospitals NHS Foundation Trust	Cambridge
United Kingdom	Royal Free Hospital NHS Foundation Trust	London
United Kingdom	Salford Royal NHS Foundation Trust	Salford
United States	Emory Clinic	Atlanta	Georgia
United States	Billings Clinic	Billings	Montana
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	University of Cincinnati Gardner Neuroscience Institute	Cincinnati	Ohio
United States	The Ohio State University Wexner Medical Center	Columbus	Ohio
United States	Duke University Medical Center	Durham	North Carolina
United States	Lysosomal and Rare Disorders Research and Treatment Center, Inc.	Fairfax	Virginia
United States	University of Florida Clinical Research Center	Gainesville	Florida
United States	Northwell Health	Great Neck	New York
United States	Hackensack University Medical Center	Hackensack	New Jersey
United States	IU Health Neuroscience Center	Indianapolis	Indiana
United States	University of California, Irvine	Irvine	California
United States	University of Arkansas for Medical Sciences	Little Rock	Arkansas
United States	NYU School of Medicine	New York	New York
United States	University of Pennsylvania	Philadelphia	Pennsylvania
United States	UPMC Montefiore Clinical and Translational Research Center	Pittsburgh	Pennsylvania
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Amicus Therapeutics

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Austria,  Belgium,  Bosnia and Herzegovina,  Canada,  Denmark,  France,  Germany,  Greece,  Hungary,  Italy,  Japan,  Korea, Republic of,  Netherlands,  New Zealand,  Poland,  Slovenia,  Spain,  Sweden,  Taiwan,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of participants with Treatment Emergent Adverse Events (TEAE)		baseline, up to approximately 4 years
Secondary	6-Minute Walk Test	Change in 6MWD from baseline to assess the efficacy of ATB200/AT2221 co-administration	baseline, up to approximately 4 years
Secondary	Pulmonary Function - Forced vital capacity (FVC)	Change from baseline in FVC (sitting) to assess the efficacy of ATB200/AT2221 co-administration	baseline, up to approximately 4 years
Secondary	Change from baseline in muscle strength measured by Quantitative Muscle Strength testing		baseline, up to approximately 4 years
Secondary	Change from baseline in muscle strength measured by Manual Muscle Strength testing		baseline, up to approximately 4 years
Secondary	The Rasch-built Pompe-specific activity (R-PAct) questionnaires	Change in R-Pact from baseline to assess the efficacy of ATB200/AT2221 co-administration	baseline, up to approximately 4 years
Secondary	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.	baseline, up to approximately 4 years
Secondary	Change from baseline in scores of PROMIS - physical function questionnaire		baseline, up to approximately 4 years
Secondary	Change from baseline in scores of PROMIS - fatigue questionnaire		baseline, up to approximately 4 years
Secondary	Change from baseline in scores of PROMIS - dyspnea questionnaire		baseline, up to approximately 4 years
Secondary	Change from baseline in scores of PROMIS - upper extremity questionnaire		baseline, up to approximately 4 years
Secondary	Motor Function - Gait, Stairs, Gower, Chair (GSGC) test	Change from baseline in GSGC score to assess the efficacy of ATB200/AT2221 co-administration.	baseline, up to approximately 4 years
Secondary	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.	baseline, up to approximately 4 years
Secondary	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.	baseline, up to approximately 4 years
Secondary	Change from baseline Biomarker -CK		baseline, up to approximately 4 years
Secondary	Change from baseline Biomarker -uHex4		baseline, up to approximately 4 years
Secondary	Immunogenicity: Incidence of neutralizing		baseline, up to approximately 4 years
Secondary	Immunogenicity: anti-drug antibodies		baseline, up to approximately 4 years