Pompe Disease Clinical Trial
Official title:
Efficacy of Continuous Positive Airway Pressure of Treatment of Hypernasality of Children With Infantile Pompe Disease
Patients with infantile onset Pompe disease will be trained with continuous positive airway pressure to see if hypernasality can be improved.
n/a
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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 |
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 |