Pompe Disease Registry



Status:Recruiting
Conditions:Endocrine, Diabetes
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:Any
Updated:3/15/2019
Start Date:August 21, 2004
End Date:September 30, 2022
Contact:Trial Transparency email recommended (Toll free number for US & Canada)
Email:Contact-Us@sanofi.com
Phone:800-633-1610

Use our guide to learn which trials are right for you!

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®.


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
We found this trial at
9
sites
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Dublin, OH
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Boston, MA
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Cleveland, OH
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from
Helena, MT
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from
Morristown, NJ
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from
Paterson, NJ
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from
Sacramento, CA
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from
São Paulo,
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from
Tampa, FL
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