Pompe Disease Registry

This study is currently recruiting participants.
Verified June 2013 by Genzyme, a Sanofi Company
Sponsor:
Information provided by (Responsible Party):
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00231400
First received: September 30, 2005
Last updated: July 17, 2013
Last verified: June 2013

September 30, 2005
July 17, 2013
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 ]
Not Provided
Complete list of historical versions of study NCT00231400 on ClinicalTrials.gov Archive Site
Not Provided
Not Provided
Not Provided
Not Provided
 
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 with Myozyme®.
Not Provided
Observational
Observational Model: Cohort
Not Provided
Not Provided
Non-Probability Sample

Patients diagnosed with Pompe disease

  • Glycogen Storage Disease Type II
  • Pompe Disease
Not Provided
Not Provided
Not Provided

*   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 enzyme deficiency or GAA gene mutation

Exclusion Criteria:

  • There are no exclusion criteria
Both
Not Provided
No
Contact: Medical Information 800-745-4447 medinfo@genzyme.com
Contact: Medical Information 617-252-7832 medinfo@genzyme.com
United States
 
NCT00231400
Pompe Registry
No
Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Genzyme, a Sanofi Company
June 2013

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