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Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®

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ClinicalTrials.gov Identifier: NCT00731081
Recruitment Status : Completed
First Posted : August 8, 2008
Last Update Posted : February 6, 2014
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Tracking Information
First Submitted Date August 6, 2008
First Posted Date August 8, 2008
Last Update Posted Date February 6, 2014
Study Start Date March 2007
Actual Primary Completion Date May 2009   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: August 7, 2008)
To describe severe late onset patients with Pompe disease receiving Myozyme and follow-up according to the CETP recommendations [ Time Frame: 12 to 18 months ]
Original Primary Outcome Measures Same as current
Change History Complete list of historical versions of study NCT00731081 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures Not Provided
Original Secondary Outcome Measures Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme®
Official Title Observational Study About the Evolution of Severe Late Onset Pompe Disease Patient With Pulmonary Dysfunction and Receiving Myozyme
Brief Summary To describe severe late onset patients with pompe disease receiving Myozyme®
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Retention:   Samples Without DNA
Description:
urine and plasma oligosaccharides
Sampling Method Non-Probability Sample
Study Population Patients with severe late onset of Pompe disease with respiratory deficiency and treated by Myozyme
Condition
  • Pompe Disease (Late-Onset)
  • Glycogen Storage Disease Type II (GSD II)
  • Glycogenesis 2 Acid Maltase Deficiency
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: August 7, 2008)
8
Original Estimated Enrollment Same as current
Actual Study Completion Date May 2009
Actual Primary Completion Date May 2009   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Male or Female ≥ 18 years of age;
  • The patient and/or patient's legal representative has given their informed consent in writing before any study procedure is initiated;
  • Pompe disease confirmed by documented deficit in endogenous acid alpha-glucosidase (GAA) activity;
  • A severe form of the disease as defined as follows: a. Moderate to severe limb girdle muscle weakness requiring help for walking around (sticks, crutches, walking frame or wheelchair); and b. Symptoms of diaphragmatic dysfunction defined by at least 2 out of the 3 following criteria: orthopnea, vital capacity < 50%, paradoxical respiration detected in measurement of transdiaphragmatic pressure; and c. Use of invasive ventilation (defined by need for tracheotomy) or noninvasive ventilation (defined by utilization of assisted ventilation using a nasal or facial mask)day and night prescribed ≥ 12 hours/day;
  • Treated for ≥6 months with Myozyme;
  • Followed-up in a reference center according to the CETP recommendations.

Exclusion Criteria:

  • The patient presents with a major congenital anomaly;
  • The patient presents with a clinically important organic disease (except for symptoms related to Pompe disease) such as cardiovascular, hepatic, pulmonary, neurological or renal disease or any other medical condition, serious disease or particular circumstances that in the investigator's opinion, should preclude the patient's participation.
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries France
Removed Location Countries  
 
Administrative Information
NCT Number NCT00731081
Other Study ID Numbers AGLU04107
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Sanofi ( Genzyme, a Sanofi Company )
Study Sponsor Genzyme, a Sanofi Company
Collaborators Not Provided
Investigators
Study Director: Medical Monitor Genzyme, a Sanofi Company
PRS Account Sanofi
Verification Date February 2014