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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02221362




Registration number
NCT02221362
Ethics application status
Date submitted
6/08/2014
Date registered
20/08/2014
Date last updated
31/10/2016

Titles & IDs
Public title
A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
Scientific title
A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease
Secondary ID [1] 0 0
701-901
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Late-onset Pompe Patients Untreated or Treated With rhGAA 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
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Observational - No interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease
Timepoint [1] 0 0
264 Weeks

Eligibility
Key inclusion criteria
- Willing and able to provide written informed consent after the nature of the study has
been explained and prior to any study-related procedure

- Diagnosed with late-onset Pompe disease based on current or previous genomic testing
and/or endogenous GAA activity

- At least 18 years of age at study entry

- Willing and able to comply with all study procedures
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Requires non-invasive ventilatory support while awake and in the upright position

- Concurrent disease, medical condition, or extenuating circumstance that, in the
opinion of the investigator, might compromise study completion or data collection

- Unable to perform baseline efficacy assessments

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/09/2016
Sample size
Target
Accrual to date
Final
30
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC
Recruitment hospital [1] 0 0
Westmead Hospital, Dept. of Genetic Medicine - Westmead
Recruitment hospital [2] 0 0
Women's and Children's Hospital - Adelaide
Recruitment hospital [3] 0 0
Monash Medical Centre - Clayton
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
5006 - Adelaide
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
Brazil
State/province [1] 0 0
Porto Alegre
Country [2] 0 0
Brazil
State/province [2] 0 0
Recife
Country [3] 0 0
Brazil
State/province [3] 0 0
Ribeirao Preto
Country [4] 0 0
Canada
State/province [4] 0 0
Alberta
Country [5] 0 0
Greece
State/province [5] 0 0
Alexandroupolis
Country [6] 0 0
Greece
State/province [6] 0 0
Athens
Country [7] 0 0
Greece
State/province [7] 0 0
Larissa
Country [8] 0 0
Greece
State/province [8] 0 0
Thessalonika
Country [9] 0 0
Ireland
State/province [9] 0 0
Dublin
Country [10] 0 0
Korea, Republic of
State/province [10] 0 0
Seoul
Country [11] 0 0
Poland
State/province [11] 0 0
Gdansk
Country [12] 0 0
Poland
State/province [12] 0 0
Krakow
Country [13] 0 0
Poland
State/province [13] 0 0
Warsaw
Country [14] 0 0
Romania
State/province [14] 0 0
Constanta
Country [15] 0 0
Serbia
State/province [15] 0 0
Belgrade
Country [16] 0 0
Singapore
State/province [16] 0 0
Singapore
Country [17] 0 0
Slovenia
State/province [17] 0 0
Ljubljana
Country [18] 0 0
Spain
State/province [18] 0 0
Barcelona
Country [19] 0 0
Spain
State/province [19] 0 0
Madrid
Country [20] 0 0
Spain
State/province [20] 0 0
Valencia
Country [21] 0 0
Taiwan
State/province [21] 0 0
Taipei

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
BioMarin Pharmaceutical
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Study 701-901, a multicenter, multinational, longitudinal, non-interventional observational
study in subjects, at least 18 years old, diagnosed with late-onset Pompe disease
prospectively collects data to understand clinical progression in terms of respiratory
function, symptomology, genotype, biochemistry, endurance and selected subject-reported
measures for 24 weeks followed by a 240 week additional observation period for up to 100
subjects.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02221362
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Monitor, MD
Address 0 0
BioMarin Pharmaceutical
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT02221362