Pompe Disease Registry

Tracking Information
First Submitted Date	September 30, 2005
First Posted Date	October 4, 2005
Last Update Posted Date	August 14, 2017
Actual Study Start Date	August 21, 2004
Estimated Primary Completion Date	September 30, 2022 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures; (submitted: January 30, 2013)	Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ]
Original Primary Outcome Measures	Not Provided
Change History	Complete list of historical versions of study NCT00231400 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures	Not Provided
Original Secondary Outcome Measures	Not Provided
Current Other Outcome Measures	Not Provided
Original Other Outcome Measures	Not Provided
Descriptive Information
Brief Title	Pompe Disease Registry
Official Title	Pompe Disease Registry
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®.
Detailed Description	Not Provided
Study Type	Observational
Study Design	Observational Model: Cohort; Time Perspective: Other
Target Follow-Up Duration	Not Provided
Biospecimen	Not Provided
Sampling Method	Non-Probability Sample
Study Population	Patients diagnosed with Pompe disease
Condition	Glycogen Storage Disease Type II; Pompe Disease
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	Recruiting
Estimated Enrollment; (submitted: January 30, 2013)	2000
Original Enrollment	Not Provided
Estimated Study Completion Date	September 30, 2022
Estimated Primary Completion Date	September 30, 2022 (Final data collection date for primary outcome measure)
Eligibility 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
Sex/Gender	Sexes Eligible for Study: All
Ages	Child, Adult, Older Adult
Accepts Healthy Volunteers	No
Contacts	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
Listed Location Countries	Brazil, Canada, China, Hong Kong, Italy, Korea, Republic of, Malaysia, Philippines, Singapore, Slovakia, Taiwan, Thailand, United States
Removed Location Countries
Administrative Information
NCT Number	NCT00231400
Other Study ID Numbers	Pompe Registry; DIREGC07005 ( Other Identifier: Sanofi )
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	August 2017