DrugLib.com — Drug Information Portal

Rx drug information, pharmaceutical research, clinical trials, news, and more



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

Page last updated: August 23, 2015

-- advertisement -- The American Red Cross
 
Home | About Us | Contact Us | Site usage policy | Privacy policy

All Rights reserved - Copyright DrugLib.com, 2006-2017