Actively Recruiting
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Led by Duke University · Updated on 2026-04-08
400
Participants Needed
1
Research Sites
1017 weeks
Total Duration
On this page
Sponsors
D
Duke University
Lead Sponsor
G
Genzyme, a Sanofi Company
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme/Myozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have developed high and sustained antibodies to ERT. To follow the long-term outcomes, we will collect medical records including but not limited to the diagnosis, clinical parameters, assessments for clinical monitoring, and laboratory values including antibody testing results.
CONDITIONS
Official Title
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease
- Must provide a written informed consent
You will not qualify if you...
History of severe allergic reactions to study medication Currently pregnant or breastfeeding Recent participation in another clinical trial within the last 30 days Presence of uncontrolled medical conditions that could affect safety
AI-Screening
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Trial Site Locations
Total: 1 location
1
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
Research Team
A
Ankit K Desai, MBBS
CONTACT
E
Eleanor Rodriguez-Rassi, MPH
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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