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Pompe Disease Registry

This study is currently recruiting participants. (see Contacts and Locations)
Verified August 2014 by Sanofi
Sponsor:
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
ClinicalTrials.gov Identifier:
NCT00231400
First received: September 30, 2005
Last updated: August 26, 2014
Last verified: August 2014

September 30, 2005
August 26, 2014
September 2004
September 2021   (final data collection date for primary outcome measure)
Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ] [ Designated as safety issue: No ]
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Complete list of historical versions of study NCT00231400 on ClinicalTrials.gov Archive Site
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Pompe Disease Registry
Pompe Disease Registry

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®.
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Observational
Observational Model: Cohort
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Non-Probability Sample

Patients diagnosed with Pompe disease

  • Glycogen Storage Disease Type II
  • 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.
 
Recruiting
2000
September 2021
September 2021   (final data collection date for primary outcome measure)

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
Both
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No
Contact: Medical Information 800-745-4447 medinfo@genzyme.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com
United States
 
NCT00231400
Pompe Registry, DIREGC07005
No
Sanofi ( Genzyme, a Sanofi Company )
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
August 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP