Pompe Disease Registry Protocol

Status Recruiting

Clinical Trial Summary

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.

Clinical Trial Description

Study Design Time Perspective: Retrospective and Prospective ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00231400
Study type	Observational [Patient Registry]
Source	Sanofi
Contact	Trial Transparency email recommended (Toll free number for US &
Phone	800-633-1610
Email	Contact-Us@sanofi.com
Status	Recruiting
Phase
Start date	September 15, 2004
Completion date	January 31, 2034

View Details

See also
Status	Clinical Trial	Phase
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Completed	`NCT00976352` - Safety Study of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase to Treat Pompe Disease	Phase 1/Phase 2
Recruiting	`NCT01665326` - Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Completed	`NCT01758354` - Newborn Screening Assay of Pompe's Disease	N/A
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Recruiting	`NCT04476550` - Clinical Specimen Collection From Pompe Disease Patients
Completed	`NCT01380743` - Drug-drug Interaction Study	Phase 2
Recruiting	`NCT05734521` - Avalglucosidase Alfa Pregnancy Study
Completed	`NCT02742298` - Pompe Disease QMUS and EIM	N/A
Terminated	`NCT02185651` - A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction	Phase 1
Completed	`NCT02654886` - Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease.	N/A
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Completed	`NCT00701129` - An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease	Phase 4
Completed	`NCT01451879` - Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies	N/A
Completed	`NCT02240407` - Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease	Phase 1
Completed	`NCT05073783` - A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
Active, not recruiting	`NCT04093349` - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)	Phase 1/Phase 2
Completed	`NCT02363153` - Diet and Exercise in Pompe Disease	N/A
Completed	`NCT01410890` - Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease	Phase 4