Actively Recruiting
Cognitive and Neurological Pathologies in Pompe Disease
Led by Duke University · Updated on 2026-03-04
45
Participants Needed
1
Research Sites
656 weeks
Total Duration
On this page
Sponsors
D
Duke University
Lead Sponsor
S
Sanofi
Collaborating Sponsor
AI-Summary
What this Trial Is About
The purpose of this study is to better understand the long-term health effects of Pompe disease and to determine if there are any abnormal changes in the brain and peripheral nerves. Additionally, the investigators will study the relationship between the abnormal changes in brain, nervous system findings, and developmental outcomes. The investigators will collect clinical information from clinic visits as well as assessments such as neuroimaging (magnetic resonance imaging (MRI), magnetic resonance spectroscopy (MRS), and diffusion tensor imaging (DTI)), cognition, academic skills, speech and language function, physical therapy and quantitative muscle ultrasound. Subjects will be in this study for at least 3 years and up to 6 years.
CONDITIONS
Official Title
Cognitive and Neurological Pathologies in Pompe Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Subject has a confirmed and documented diagnosis of infantile Pompe Disease (IPD) or Late onset Pompe Disease (LOPD)
You will not qualify if you...
- Refusal to give informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
Research Team
G
Gretchen Nichting
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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