Pompe Disease Registry Protocol
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00231400|
Recruitment Status : Recruiting
First Posted : October 4, 2005
Last Update Posted : January 13, 2023
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, and for other research and non-research related purposes.
The objectives of the Registry are:
- To enhance understanding of the variability, progression, identification, and natural history of Pompe disease, with the ultimate goal of better guiding and assessing therapeutic intervention.
- To assist the Pompe medical community with the development of recommendations for monitoring patients, and to provide reports on patient outcomes, to optimize patient care.
- To characterize the Pompe disease population.
- To evaluate the long-term effectiveness of alglucosidase alfa.
|Condition or disease|
|Glycogen Storage Disease Type II Pompe Disease|
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||2000 participants|
|Target Follow-Up Duration:||5 Years|
|Official Title:||Pompe Disease Registry|
|Actual Study Start Date :||September 15, 2004|
|Estimated Primary Completion Date :||January 31, 2034|
|Estimated Study Completion Date :||January 31, 2034|
- Understanding of the variability, progression , identification and natural history of the manifestations of Pompe disease [ Time Frame: maximum 30 years ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00231400
|Contact: Trial Transparency email recommended (Toll free number for US & Canada)||800-633-1610 ext 1 then #||Contact-Us@sanofi.com|
|Contact: Pompe Registry HelpLine||617-591-5500|
|Study Director:||Study Director||Genzyme, a Sanofi Company|