Pompe Disease Clinical Trial
Official title:
Developmental Outcomes of School-aged Children With Infantile-onset Pompe Disease: A Telemedicine Approach to Assessment and Cognitive Training
The primary purpose of this study is to: - Document the developmental outcomes of individuals with Pompe disease treated with long-term enzyme-replacement therapy (ERT) through school-age (ages 6-18) using measures of cognitive functioning, academic skills, and speech and language abilities. - Investigate possible cognitive processing speed weaknesses using BrainBaseline neurocognitive assessment software. - Investigate the relationship between behavior and other developmental factors including speech and language ability and cognitive ability. - Explore if the use of selected iPad applications may help strengthen cognitive processing speed in children with Pompe disease
The study will last approximately 2-3 years. It includes 2-4 visits to Duke for developmental assessments. The baseline/year 1 visit may take place at Duke (may take 1-2 days) or remotely via iPad. The follow up 1/year 2 visit may take place at Duke (1-2 days) or remotely via an iPad, depending upon each child's particular situation. ;
| 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 |