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Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00113035
First Posted: June 6, 2005
Last Update Posted: May 5, 2015
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 evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by:
Sanofi
June 3, 2005
June 6, 2005
May 5, 2015
May 2005
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Complete list of historical versions of study NCT00113035 on ClinicalTrials.gov Archive Site
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Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Screening Protocol to Evaluate Acid Alpha Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease for Potential Inclusion in Future Clinical Studies With Myozyme (Alglucosidase Alfa)
Pompe disease (also known as glycogen storage disease type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The primary objective of this study is to identify potential candidates for future clinical studies in Pompe disease.
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Observational
Time Perspective: Prospective
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Pompe Disease
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Patients with late onset Pompe Disease
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
60
October 2005
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Inclusion Criteria:

  • Must provide written informed consent prior to any study-related procedures being performed
  • Clinical presentation consistent with late-onset Pompe disease, or a current clinical diagnosis of late-onset Pompe disease
  • Must be greater than or equal to 8 years of age
  • Must be able to ambulate (use of assistive devices, such as a walker, cane or crutches, is permitted)

Exclusion Criteria:

  • Requires the use of invasive ventilatory support
  • Requires the use of noninvasive ventilatory support while awake and in an upright position
  • Use of any investigational product within 30 days prior to study enrollment
  • Unwillingness to comply with protocol requirements
  • Has clinically significant organic disease, unstable medical condition, serious or intercurrent illness
  • Is pregnant or lactating
  • Has participated in the Prospective Observational Study in Patients with Late-Onset Pompe Disease (AGLU02303, "LOPOS")
Sexes Eligible for Study: All
8 Years and older   (Child, Adult, Senior)
No
Contact information is only displayed when the study is recruiting subjects
United States
 
 
NCT00113035
AGLU02905
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Genzyme, a Sanofi Company
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Sanofi
May 2015