Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease (NCT01665326)
Determination of Cross-Reactive Immunological Material (CRIM) Status and Longitudinal Follow-up of Individuals With Pompe Disease
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Duke University Medical Center — Durham, North Carolina
🧪 Interventions in This Study
- Observational (OTHER)
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (1 Sites)
- Duke University Medical Center — Durham, North Carolina
📞 Contact the Study Coordinator
Phone: (919) 613-6310
Email: ankit.desai@duke.edu
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Pompe Disease
- Developing a Management Approach for Patients With "Late-Onset" Pompe Disease — Durham, North Carolina
- A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) — Phoenix, Arizona
- A Global Prospective Observational Registry of Patients With Pompe Disease — Little Rock, Arkansas
- Cognitive and Neurological Pathologies in Pompe Disease — Durham, North Carolina
- Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) — Irvine, California
Explore by condition: All Pompe Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.