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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT04138277
Ethics application status
Date submitted
18/10/2019
Date registered
24/10/2019

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
Treatment: Other - ATB200

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


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

Treatment: Other: ATB200
Enzyme Replacement Therapy via intravenous infusion
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Treatment: Other
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
Timepoint [1] 0 0
baseline, up to approximately 4 years
Secondary outcome [2] 0 0
Pulmonary Function - Forced vital capacity (FVC)
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
Timepoint [5] 0 0
baseline, up to approximately 4 years
Secondary outcome [6] 0 0
EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) questionnaires
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
Timepoint [11] 0 0
baseline, up to approximately 4 years
Secondary outcome [12] 0 0
Physician's Global Impression of Change
Timepoint [12] 0 0
baseline, up to approximately 4 years
Secondary outcome [13] 0 0
Subject's Global Impression of Change
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
Sex
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
Not applicable
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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/12/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2024
Actual
Sample size
Target
110
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
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
Montana
Country [8] 0 0
United States of America
State/province [8] 0 0
New Jersey
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
Utah
Country [14] 0 0
United States of America
State/province [14] 0 0
Virginia
Country [15] 0 0
Argentina
State/province [15] 0 0
Buenos Aires
Country [16] 0 0
Austria
State/province [16] 0 0
Innsbruck
Country [17] 0 0
Belgium
State/province [17] 0 0
Leuven
Country [18] 0 0
Bosnia and Herzegovina
State/province [18] 0 0
The Republic Of Srpska
Country [19] 0 0
Canada
State/province [19] 0 0
Alberta
Country [20] 0 0
Canada
State/province [20] 0 0
Ontario
Country [21] 0 0
Denmark
State/province [21] 0 0
Aarhus
Country [22] 0 0
France
State/province [22] 0 0
Bron
Country [23] 0 0
France
State/province [23] 0 0
Garches
Country [24] 0 0
France
State/province [24] 0 0
Lille
Country [25] 0 0
France
State/province [25] 0 0
Marseille
Country [26] 0 0
France
State/province [26] 0 0
Nice
Country [27] 0 0
Germany
State/province [27] 0 0
Bonn
Country [28] 0 0
Germany
State/province [28] 0 0
München
Country [29] 0 0
Germany
State/province [29] 0 0
Münster
Country [30] 0 0
Greece
State/province [30] 0 0
Athens
Country [31] 0 0
Hungary
State/province [31] 0 0
Budapest
Country [32] 0 0
Hungary
State/province [32] 0 0
Pécs
Country [33] 0 0
Hungary
State/province [33] 0 0
Szeged
Country [34] 0 0
Italy
State/province [34] 0 0
Messina
Country [35] 0 0
Italy
State/province [35] 0 0
Napoli
Country [36] 0 0
Japan
State/province [36] 0 0
Fukuoka
Country [37] 0 0
Japan
State/province [37] 0 0
Kagashima
Country [38] 0 0
Japan
State/province [38] 0 0
Osaka
Country [39] 0 0
Japan
State/province [39] 0 0
Sapporo
Country [40] 0 0
Japan
State/province [40] 0 0
Tokyo
Country [41] 0 0
Korea, Republic of
State/province [41] 0 0
Gyeongsangnam-do
Country [42] 0 0
Netherlands
State/province [42] 0 0
Rotterdam
Country [43] 0 0
New Zealand
State/province [43] 0 0
Auckland
Country [44] 0 0
Poland
State/province [44] 0 0
Podkarpackie
Country [45] 0 0
Slovenia
State/province [45] 0 0
Ljubljana
Country [46] 0 0
Spain
State/province [46] 0 0
Barcelona
Country [47] 0 0
Sweden
State/province [47] 0 0
Gothenburg
Country [48] 0 0
Taiwan
State/province [48] 0 0
Taipei
Country [49] 0 0
United Kingdom
State/province [49] 0 0
Birmingham
Country [50] 0 0
United Kingdom
State/province [50] 0 0
Cambridge
Country [51] 0 0
United Kingdom
State/province [51] 0 0
London
Country [52] 0 0
United Kingdom
State/province [52] 0 0
Salford

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04138277