A Prospective, Noninterventional, Observational Study of Late-Onset Pompe Disease

Terminated

• Conditions: Late-onset Pompe Patients Untreated or Treated With rhGAA

• Registration Number: NCT02221362
• Lead Sponsor: BioMarin Pharmaceutical

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2014-08-06
• Study First Submitted QC: 2014-08-19
• Study First Posted: 2014-08-20
• Study Start: 2014-12
• Status Verified: 2015-09
• Primary Completion: 2016-09
• Study Completion: 2016-09
• Last Update Submitted: 2016-10-27
• Last Update Posted: 2016-10-31

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 30

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

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

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease	264 Weeks

Trial Locations

Locations (15)

Westmead Hospital, Dept. of Genetic Medicine; 🇦🇺 Westmead, New South Wales, Australia

Women's and Children's Hospital; 🇦🇺 Adelaide, South Australia, Australia

Monash Medical Centre; 🇦🇺 Clayton, Victoria, Australia

Alberta Children's Hospital; 🇨🇦 Calgary, Alberta, Canada

St. Vincent's University Hospital; 🇮🇪 Dublin, Ireland

Seoul National University Hospital; 🇰🇷 Seoul, Korea, Republic of

Copernicus, St. Adalbert Hospital; 🇵🇱 Gdansk, Poland

Diamond Clinic; 🇵🇱 Krakow, Poland

Insitute of Psychiatry and Neurology; 🇵🇱 Warsaw, Poland

Spitalul Clinic Judetean de Urgenta "Sf. Apostol Andrei"; 🇷🇴 Constanta, Romania

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