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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT04138277




Registration number
NCT04138277
Ethics application status
Date submitted
18/10/2019
Date registered
24/10/2019
Date last updated
4/09/2020

Titles & IDs
Public title
A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD
Scientific 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
Secondary ID [1] 0 0
ATB200-07
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Pompe Disease (Late-onset) 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - AT2221
Other interventions - ATB200

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


Treatment: Drugs: AT2221
Participants received ATB200 co-administered with AT2221 (Miglustat)

Other interventions: ATB200
Enzyme Replacement Therapy via intravenous infusion

Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Proportion of participants with Treatment Emergent Adverse Events (TEAE)
Timepoint [1] 0 0
baseline, up to approximately 4 years
Secondary outcome [1] 0 0
6-Minute Walk Test - Change in 6MWD from baseline to assess the efficacy of ATB200/AT2221 co-administration
Timepoint [1] 0 0
baseline, up to approximately 4 years
Secondary outcome [2] 0 0
Pulmonary Function - Forced vital capacity (FVC) - Change from baseline in FVC (sitting) to assess the efficacy of ATB200/AT2221 co-administration
Timepoint [2] 0 0
baseline, up to approximately 4 years
Secondary outcome [3] 0 0
Change from baseline in muscle strength measured by Quantitative Muscle Strength testing
Timepoint [3] 0 0
baseline, up to approximately 4 years
Secondary outcome [4] 0 0
Change from baseline in muscle strength measured by Manual Muscle Strength testing
Timepoint [4] 0 0
baseline, up to approximately 4 years
Secondary outcome [5] 0 0
The Rasch-built Pompe-specific activity (R-PAct) questionnaires - Change in R-Pact from baseline to assess the efficacy of ATB200/AT2221 co-administration
Timepoint [5] 0 0
baseline, up to approximately 4 years
Secondary outcome [6] 0 0
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.
Timepoint [6] 0 0
baseline, up to approximately 4 years
Secondary outcome [7] 0 0
Change from baseline in scores of PROMIS - physical function questionnaire
Timepoint [7] 0 0
baseline, up to approximately 4 years
Secondary outcome [8] 0 0
Change from baseline in scores of PROMIS - fatigue questionnaire
Timepoint [8] 0 0
baseline, up to approximately 4 years
Secondary outcome [9] 0 0
Change from baseline in scores of PROMIS - dyspnea questionnaire
Timepoint [9] 0 0
baseline, up to approximately 4 years
Secondary outcome [10] 0 0
Change from baseline in scores of PROMIS - upper extremity questionnaire
Timepoint [10] 0 0
baseline, up to approximately 4 years
Secondary outcome [11] 0 0
Motor Function - Gait, Stairs, Gower, Chair (GSGC) test - Change from baseline in GSGC score to assess the efficacy of ATB200/AT2221 co-administration.
Timepoint [11] 0 0
baseline, up to approximately 4 years
Secondary outcome [12] 0 0
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.
Timepoint [12] 0 0
baseline, up to approximately 4 years
Secondary outcome [13] 0 0
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.
Timepoint [13] 0 0
baseline, up to approximately 4 years
Secondary outcome [14] 0 0
Change from baseline Biomarker -CK
Timepoint [14] 0 0
baseline, up to approximately 4 years
Secondary outcome [15] 0 0
Change from baseline Biomarker -uHex4
Timepoint [15] 0 0
baseline, up to approximately 4 years
Secondary outcome [16] 0 0
Immunogenicity: Incidence of neutralizing
Timepoint [16] 0 0
baseline, up to approximately 4 years
Secondary outcome [17] 0 0
Immunogenicity: anti-drug antibodies
Timepoint [17] 0 0
baseline, up to approximately 4 years

Eligibility
Key inclusion criteria
1. Subject must have completed Study ATB200-03.
Minimum age
18 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Westmead Hospital - Westmead
Recruitment hospital [2] 0 0
Royal Brisbane & Women's Hospital - Brisbane
Recruitment hospital [3] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [4] 0 0
Monash Medical Centre - Clayton
Recruitment postcode(s) [1] 0 0
- Westmead
Recruitment postcode(s) [2] 0 0
4029 - Brisbane
Recruitment postcode(s) [3] 0 0
5000 - Adelaide
Recruitment postcode(s) [4] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
Montana
Country [7] 0 0
United States of America
State/province [7] 0 0
New Jersey
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Utah
Country [13] 0 0
United States of America
State/province [13] 0 0
Virginia
Country [14] 0 0
Argentina
State/province [14] 0 0
Buenos Aires
Country [15] 0 0
Austria
State/province [15] 0 0
Innsbruck
Country [16] 0 0
Belgium
State/province [16] 0 0
Leuven
Country [17] 0 0
Bosnia and Herzegovina
State/province [17] 0 0
The Republic Of Srpska
Country [18] 0 0
Canada
State/province [18] 0 0
Alberta
Country [19] 0 0
Denmark
State/province [19] 0 0
Aarhus
Country [20] 0 0
France
State/province [20] 0 0
Bron
Country [21] 0 0
France
State/province [21] 0 0
Garches
Country [22] 0 0
France
State/province [22] 0 0
Lille
Country [23] 0 0
France
State/province [23] 0 0
Marseille
Country [24] 0 0
France
State/province [24] 0 0
Nice
Country [25] 0 0
Hungary
State/province [25] 0 0
Budapest
Country [26] 0 0
Hungary
State/province [26] 0 0
Pécs
Country [27] 0 0
Hungary
State/province [27] 0 0
Szeged
Country [28] 0 0
Poland
State/province [28] 0 0
Podkarpackie
Country [29] 0 0
Spain
State/province [29] 0 0
Barcelona
Country [30] 0 0
Taiwan
State/province [30] 0 0
Taipei
Country [31] 0 0
United Kingdom
State/province [31] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Amicus Therapeutics
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT04138277
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
For Site
Address 0 0
Country 0 0
Phone 0 0
609-662-2000
Fax 0 0
Email 0 0
PompeSiteInfo@amicusrx.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT04138277