Extension Study for Patients Who Have Participated in a BMN 701 Study

See also
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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
Recruiting	`NCT00231400` - Pompe Disease Registry Protocol
Recruiting	`NCT04476550` - Clinical Specimen Collection From Pompe Disease Patients
Recruiting	`NCT05687474` - Baby Detect : Genomic Newborn Screening
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	NCT01435772
Study type	Interventional
Source	BioMarin Pharmaceutical
Contact
Status	Terminated
Phase	Phase 2
Start date	August 15, 2011
Completion date	September 9, 2016

Extension Study for Patients Who Have Participated in a BMN 701 Study

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms