Pompe Disease Clinical Trial
Official title:
Pompe Disease Registry
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.
Study Design Time Perspective: Retrospective and Prospective ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT01942590 -
Safety and Efficacy of Clenbuterol in Individuals With Late-onset Pompe Disease and Receiving Enzyme Replacement Therapy
|
Phase 1/Phase 2 | |
Not yet recruiting |
NCT01409486 -
Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry
|
N/A | |
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 | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
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 | |
Completed |
NCT02405598 -
Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease
|
Phase 4 | |
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 |