Pompe Disease Registry
Information source: Sanofi
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Glycogen Storage Disease Type II; Pompe Disease
Phase: N/A
Status: Recruiting
Sponsored by: Genzyme, a Sanofi Company Official(s) and/or principal investigator(s): Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company
Overall contact: Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
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®.
Clinical Details
Official title: Pompe Disease Registry
Study design: Observational Model: Cohort
Primary outcome: Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Both.
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
Locations and Contacts
Medical Information, Phone: 800-745-4447, Email: medinfo@genzyme.com
Registry participation is worldwide and not limited to this facility; facilities not yet active may enroll upon identification of a patient, Cambridge, Massachusetts 02142, United States; Recruiting
Additional Information
Starting date: September 2004
Last updated: August 14, 2015
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