High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric

See also
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	`NCT00231400` - Pompe Disease Registry Protocol
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

NCT number	NCT04981210
Study type	Observational
Source	National Taiwan University Hospital
Contact
Status	Recruiting
Phase
Start date	August 1, 2021
Completion date	July 31, 2024

High Risk Screen of Childhood Late-onset Pompe Disease in Pediatric Outpatient Clinics

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms