Clinical Trial: Pompe Disease Registry
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational
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 Summary:
Sponsor: Genzyme, a Sanofi Company
Current Primary Outcome: Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: 15 Years ]
Original Primary Outcome:
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Sanofi
Dates:
Date Received: September 30, 2005
Date Started: September 2004
Date Completion: September 2022
Last Updated: April 29, 2016
Last Verified: April 2016
