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

Late-onset Pompe Patients Untreated or Treated With rhGAA Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT02221362
• Other study ID #: 701-901
• Status: Terminated
• Phase: N/A
• First received: August 6, 2014
• Last updated: October 27, 2016
• Start date: December 2014
• Est. completion date: September 2016

Study information

• Verified date: September 2015
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 30
• Est. completion date: September 2016
• Est. primary completion date: September 2016
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Observational Model: Case Control, Time Perspective: Prospective

Related Conditions & MeSH terms

• Glycogen Storage Disease Type II
• Late-onset Pompe Patients Untreated or Treated With rhGAA

Locations

Country	Name	City	State
Australia	Women's and Children's Hospital	Adelaide	South Australia
Australia	Monash Medical Centre	Clayton	Victoria
Australia	Westmead Hospital, Dept. of Genetic Medicine	Westmead	New South Wales
Canada	Alberta Children's Hospital	Calgary	Alberta
Ireland	St. Vincent's University Hospital	Dublin
Korea, Republic of	Seoul National University Hospital	Seoul
Poland	Copernicus, St. Adalbert Hospital	Gdansk
Poland	Diamond Clinic	Krakow
Poland	Insitute of Psychiatry and Neurology	Warsaw
Romania	Spitalul Clinic Judetean de Urgenta "Sf. Apostol Andrei"	Constanta
Singapore	National University Hospital	Singapore
Spain	Hospital de la Santa Creu I Sant Pau	Barcelona
Spain	Hospital General Universitario Gregorio Maranon	Madrid
Spain	Hospital Universitari i Politecnic La Fe	Valencia
Taiwan	Mackay Memorial Hospital	Taipei

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Countries where clinical trial is conducted

Australia,  Brazil,  Canada,  Greece,  Ireland,  Korea, Republic of,  Poland,  Romania,  Serbia,  Singapore,  Slovenia,  Spain,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To evaluate the degree of change in respiratory and endurance endpoints over time in patients with Pompe disease		264 Weeks	No