NCT01665326 Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
| NCT ID | NCT01665326 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Duke University |
| Condition | Pompe Disease |
| Study Type | OBSERVATIONAL |
| Enrollment | 400 participants |
| Start Date | 2009-09 |
| Primary Completion | 2028-03 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 400 participants in total. It began in 2009-09 with a primary completion date of 2028-03.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: * Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease * Must provide a written informed consent
Contact & Investigator
Priya S Kishnani, MD
PRINCIPAL INVESTIGATOR
Duke University
Frequently Asked Questions
Who can join the NCT01665326 clinical trial?
This trial is open to participants of all sexes, studying Pompe Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT01665326 currently recruiting?
Yes, NCT01665326 is actively recruiting participants. Contact the research team at ankit.desai@duke.edu for enrollment information.
Where is the NCT01665326 trial being conducted?
This trial is being conducted at Durham, United States.
Who is sponsoring the NCT01665326 clinical trial?
NCT01665326 is sponsored by Duke University. The principal investigator is Priya S Kishnani, MD at Duke University. The trial plans to enroll 400 participants.