Pompe Disease Clinical Trial
Official title:
Search for Serum/Plasma Biomarkers in Pompe's Disease
This study aims to analyze serum and plasma samples from patients with late onset Pompe disease treated and not treated with enzyme replacement therapy (ERT) to identify microRNA that could be specific of the disease. The investigators will correlate the concentration of these microRNA with several muscle function tests and quantitative muscle MRI to know whether they are good biomarkers of progression.
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Status | Clinical Trial | Phase | |
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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
|
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Completed |
NCT01758354 -
Newborn Screening Assay of Pompe's Disease
|
N/A | |
Recruiting |
NCT00231400 -
Pompe Disease Registry Protocol
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||
Recruiting |
NCT04476550 -
Clinical Specimen Collection From Pompe Disease Patients
|
||
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
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Completed |
NCT01380743 -
Drug-drug Interaction Study
|
Phase 2 | |
Recruiting |
NCT05734521 -
Avalglucosidase Alfa Pregnancy Study
|
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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
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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 |