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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06150820




Registration number
NCT06150820
Ethics application status
Date submitted
21/11/2023
Date registered
29/11/2023

Titles & IDs
Public title
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
Scientific title
A Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients With Late-Onset Pompe Disease
Secondary ID [1] 0 0
AT845-02
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
Other interventions - No Intervention

Other: Participants with Late-Onset Pompe Disease - Adolescent or adult participants with LOPD.


Other interventions: No Intervention
No investigational drug will be administered to participants in this study.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Occurrence of total antibodies to AAV8
Timepoint [1] 0 0
Up to 2 years
Primary outcome [2] 0 0
Occurrence of neutralizing antibodies to AAV8
Timepoint [2] 0 0
Up to 2 years
Secondary outcome [1] 0 0
Seroconversion of antibodies to AAV8 over time
Timepoint [1] 0 0
Up to 2 years
Secondary outcome [2] 0 0
Creatine kinase [CK] levels
Timepoint [2] 0 0
Up to 2 years
Secondary outcome [3] 0 0
Urine glucose tetrasaccharide [Glc4]/hexose tetrasaccharide [Hex4] over time
Timepoint [3] 0 0
Up to 2 years
Secondary outcome [4] 0 0
Occurrence of anti-GAA antibodies in participants on ERT
Timepoint [4] 0 0
Up to 2 years

Eligibility
Key inclusion criteria
* Participant has a documented clinical diagnosis of LOPD.
* Participant is enzyme replacement therapy (ERT) naïve (ERT-N) or has received any ERT for 6 months or more (ERT-E).
* Participant is willing and able to comply with study visits and procedures.
* Participant agrees to not start participating in any other clinical study involving an investigational study treatment, including ERT, while participating in this study.
Minimum age
16 Years
Maximum age
69 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participant previously received an AAV-related product (any serotype).
* Participant is currently participating in a Pompe-related interventional study (other than ERT-interventional studies) or has received gene or cell therapy.
* Participant requires any invasive or noninvasive ventilation support while awake and upright (non-invasive support while sleeping with either continuous positive airway pressure (CPAP) or bilevel positive airway pressure (BiPAP) is acceptable for eligibility).
* Participant is unable to ambulate (assistive devices [e.g., cane or walker] are acceptable for eligibility).
* Participants who have received any ERT for less than 6 months as of the Baseline visit are not eligible.

Study design
Purpose of the study
Other
Allocation to intervention
NA
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
NA
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
1/02/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/05/2027
Actual
Sample size
Target
100
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
AU61003 - Adelaide
Recruitment hospital [2] 0 0
AU61001 - Herston
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment postcode(s) [2] 0 0
- Herston
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Minnesota
Country [2] 0 0
United States of America
State/province [2] 0 0
Virginia
Country [3] 0 0
France
State/province [3] 0 0
Angers
Country [4] 0 0
France
State/province [4] 0 0
Garches
Country [5] 0 0
France
State/province [5] 0 0
Lille
Country [6] 0 0
France
State/province [6] 0 0
Limoges
Country [7] 0 0
France
State/province [7] 0 0
Marseille
Country [8] 0 0
France
State/province [8] 0 0
Nantes
Country [9] 0 0
France
State/province [9] 0 0
Nice Cedex 3
Country [10] 0 0
France
State/province [10] 0 0
Strasbourg
Country [11] 0 0
Germany
State/province [11] 0 0
Hochheim
Country [12] 0 0
Italy
State/province [12] 0 0
Roma
Country [13] 0 0
Japan
State/province [13] 0 0
Kodaira-Shi
Country [14] 0 0
Japan
State/province [14] 0 0
Shinjuku-Ku
Country [15] 0 0
Spain
State/province [15] 0 0
Albacete
Country [16] 0 0
Spain
State/province [16] 0 0
Barcelona
Country [17] 0 0
Spain
State/province [17] 0 0
L'hospitalet de Llobregat
Country [18] 0 0
Spain
State/province [18] 0 0
Madrid
Country [19] 0 0
Spain
State/province [19] 0 0
San Sebastian
Country [20] 0 0
Spain
State/province [20] 0 0
Valencia
Country [21] 0 0
Taiwan
State/province [21] 0 0
Taipei
Country [22] 0 0
Taiwan
State/province [22] 0 0
Taoyuan City
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Newcastle upon Tyne
Country [24] 0 0
United Kingdom
State/province [24] 0 0
Salford

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Astellas Gene Therapies
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Astellas Gene Therapies
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Astellas Gene Therapies
Address 0 0
Country 0 0
Phone 0 0
800-888-7704
Fax 0 0
Email 0 0
[email protected]
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
Access to anonymized individual participant level data will not be provided for this trial as it meets one or more of the exceptions described on www.clinicalstudydatarequest.com under "Sponsor Specific Details for Astellas."


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/NCT06150820