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

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Trial Status Terminated

Trial Identifier

NCT02221362

Condition

Late-onset Pompe Patients Untreated or Treated With rhGAA

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

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.

Eligibility Criteria

Birth Sex

All

Age

From 18 Years

Healthy Volunteers

No

Study Type:

Observational

Number of Participants:

30

Study Started:

2014-12

Study Updated:

2016-10-31

Trial Locations

  • 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

  • Porto Alegre, Brazil

  • Recife, Brazil

  • Ribeirão Preto, Brazil

  • Alberta Children's Hospital

    Calgary, Alberta, Canada

  • Alexandroupoli, Greece

  • Athens, Greece

  • Larissa, Greece

  • Thessaloniki, Greece

  • St. Vincent's University Hospital

    Dublin, Ireland

  • 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"

    Constanța, Romania

  • Belgrade, Serbia

  • National University Hospital

    Singapore, Singapore

  • Ljubljana, Slovenia

  • Seoul National University Hospital

    Seoul, South Korea

  • Hospital de la Santa Creu I Sant Pau

    Barcelona, Spain

  • Hospital General Universitario Gregorio Maranon

    Madrid, Spain

  • Hospital Universitari i Politecnic La Fe

    Valencia, Spain

  • Mackay Memorial Hospital

    Taipei, Taiwan

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

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