Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study)

• Register: ClinicalTrials.gov
• Last refreshed on: 16 February 2021
• Main ID: NCT02824068
• Date of registration: 20/06/2016
• Prospective Registration: No
• Primary sponsor: Klinikum der Universitaet Muenchen
• Public title: Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months (STIG-Pompe-Study) STIG
• Scientific title: Long-term Outcome in Late-onset Pompe Disease Treated Beyond 36 Months
• Date of first enrolment: June 1, 2016
• Target sample size: 112
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02824068
• Study type: Observational

Countries of recruitment

Argentina, Brazil, Germany, Italy, Taiwan

Contacts

• Name: Benedikt Schoser, MD
• Affiliation: Friedrich-Baur-Institute, Dep. of Neurology Klinikum der Universitaet Muenchen Munich, Germany

Key inclusion & exclusion criteria

Inclusion Criteria:

- Late-onset Pompe patients, aged over 8 years.

- The patient is willing and able to provide signed informed consent.

- The patient (and patient's legal guardian if patient is under 18 years of age) must
have the ability to comply with the clinical protocol.

- Long-term Myozyme treatment beyond 36 months.

- Known GAA genotype.

- GAA activity (Dried blood spot testing, or other methods).

Exclusion Criteria:

- - The patient is concurrently participating in another clinical study using Myozyme or
other treatment.

- The patient, in the opinion of the Investigator, is unable to adhere to the
requirements of the study.

- The patient has clinically significant organic disease (with the exception of symptoms
relating to Pompe disease), including clinically significant cardiovascular, hepatic,
pulmonary, neurologic, or renal disease, or other medical condition, serious
intercurrent illness, or extenuating circumstance that, in the opinion of the
Investigator, precludes participation in the study or potentially decreases survival.



Age minimum: 8 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Pompe Disease

Intervention(s)

Drug: glucosidase alfa

Primary Outcome(s)

change in muscle function [Time Frame: 12 months]

Secondary Outcome(s)

changes in maximal expiratory pressure (MEP) [Time Frame: 12 months]

changes in Medical Research Council (MRC) Scale for Muscle Strength [Time Frame: 12 months]

changes in minimal inspiratory pressure (MIP) [Time Frame: 12 months]

changes in loss of ambulation [Time Frame: 12 months]

changes in forced vital capacity (FVC) [Time Frame: 12 months]

data collection on survival, death and reason of deaths [Time Frame: 12 months]

Secondary ID(s)

Po001-STIG

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Genzyme, a Sanofi Company