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Pompe Disease Registry

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ClinicalTrials.gov Identifier: NCT00231400
Recruitment Status : Recruiting
First Posted : October 4, 2005
Last Update Posted : May 12, 2020
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )

Study Description
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Brief Summary:

The Pompe Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Pompe disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician.

The objectives of the Registry are:

  • To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease;
  • To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
  • To characterize and describe the Pompe disease population as a whole; and
  • To evaluate the long-term effectiveness and safety of available treatment options including ERT(Enzyme Replacement Therapy) with Myozyme®.

Condition or disease
Glycogen Storage Disease Type II Pompe Disease

Study Design
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Study Type : Observational
Estimated Enrollment : 2000 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Pompe Disease Registry
Actual Study Start Date : September 15, 2004
Estimated Primary Completion Date : January 31, 2034
Estimated Study Completion Date : January 31, 2034

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Outcome Measures
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Primary Outcome Measures :
  1. Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ]

Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients diagnosed with Pompe disease
Criteria

Inclusion Criteria:

  • Patient must have a confirmed diagnosis of Pompe disease, documented by GAA(Glucosidase Alpha Acid) enzyme deficiency or GAA gene mutation

Exclusion Criteria:

  • There are no exclusion criteria
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00231400


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 800-633-1610 ext 1 then # Contact-Us@sanofi.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com

Locations
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Sponsors and Collaborators
Genzyme, a Sanofi Company
Investigators
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Study Director: Medical Monitor Genzyme, a Sanofi Company
More Information
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Publications:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier: NCT00231400    
Other Study ID Numbers: Pompe Registry
DIREGC07005 ( Other Identifier: Sanofi )
First Posted: October 4, 2005    Key Record Dates
Last Update Posted: May 12, 2020
Last Verified: May 2020
Keywords provided by Sanofi ( Genzyme, a Sanofi Company ):
Glycogen Storage Disease Type II (GSD-II)
GSD-II
Pompe Disease
 Pompe Disease (late-onset)
Acid Maltase Deficiency Disease
Glycogenosis II
Additional relevant MeSH terms:
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Glycogen Storage Disease Type II
Glycogen Storage Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
 Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases