Trial Review

Did you know?

The ANZCTR now automatically displays published trial results and simplifies the addition of trial documents such as unpublished protocols and statistical analysis plans.

These enhancements will offer a more comprehensive view of trials, regardless of whether their results are positive, negative, or inconclusive.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
Stopped early
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/study/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/study/NCT02221362