Pompe Disease Registry
-
- STATUS
- Recruiting
-
- End date
- Jan 31, 2034
-
- participants needed
- 2000
-
- sponsor
- Genzyme, a Sanofi Company
Updated on 19 February 2024
deficiency
replacement therapy
myozyme
gaa gene
glycogen storage disease type ii
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.
Description
Details
| Condition | pompe disease |
|---|---|
| Age | 100years or below |
| Clinical Study Identifier | NCT00231400 |
| Sponsor | Genzyme, a Sanofi Company |
| Last Modified on | 19 February 2024 |
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How to participate?
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2
Get screened
Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.
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Step
3
Enroll in the clinical study
If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.
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Step
4
Get your study results
Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.
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