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Pompe Disease Registry

This study is currently recruiting participants.
Verified August 2017 by Sanofi ( Genzyme, a Sanofi Company )
Sponsor:
ClinicalTrials.gov Identifier:
NCT00231400
First Posted: October 4, 2005
Last Update Posted: August 14, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Sanofi ( Genzyme, a Sanofi Company )
September 30, 2005
October 4, 2005
August 14, 2017
August 21, 2004
September 30, 2022   (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 ]
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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
Time Perspective: Other
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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 30, 2022
September 30, 2022   (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
Sexes Eligible for Study: All
Child, Adult, Senior
No
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
Brazil,   Canada,   China,   Hong Kong,   Italy,   Korea, Republic of,   Malaysia,   Philippines,   Singapore,   Slovakia,   Taiwan,   Thailand,   United States
 
 
NCT00231400
Pompe Registry
DIREGC07005 ( Other Identifier: Sanofi )
No
Not Provided
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Sanofi ( Genzyme, a Sanofi Company )
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
August 2017
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