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NCT01665326 · ClinicalTrials.gov registry record
Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
A clinical trial of Pompe Disease, sponsored by Duke University.
- Recruiting
- Registry status
- 400
- Enrollment target
- 1
- Study location
NCT01665326: Recruiting study of Pompe Disease, sponsored by Duke University.
NCT01665326 is a study of Pompe Disease that is actively recruiting participants, run by Duke University. The registered enrollment target is 400 participants, above the 204-participant average among 14 other Pompe Disease trials with a reported enrollment target (96% higher). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01665326, a study of Pompe Disease, is actively recruiting participants, sponsored by Duke University.
- RECRUITING
- Registry status
- 400 participants
- Enrollment target
- 1
- Study location
Study 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.
Primary Outcome
Medical records will be tracked until the patient reaches the age of 18 years to follow clinical response to ERT. This will allow us to gain an understanding of CRIM status in relation to clinical outcomes and development for these subjects.
Conditions Studied
Interventions
- OTHER Observational
Study Locations (1)
North Carolina
- Duke University Medical Center - Durham
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 400 participants |
| Start Date | 2009-09 |
| Est. Completion | 2029-03 |
What NCT01665326 shows while recruiting
NCT01665326 is an observational study that tracks outcomes without assigning an intervention. The registered 400 participants enrollment target is mid-sized for trials with a published cap, above the 204-participant average among 14 other Pompe Disease trials with a reported enrollment target (96% higher).
The record links to 1 condition, with Pompe Disease appearing as the primary indexed condition, and to 1 intervention - of which Observational is the first listed.
NCT01665326 reports a single indexed study location in North Carolina.
Frequently Asked Questions
What is clinical trial NCT01665326 about?
NCT01665326 is a clinical study titled "Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease". 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 immunosuppressiv...
What is the current status of trial NCT01665326?
This trial is currently recruiting. The enrollment target is 400 participants. The study started on 2009-09. Estimated completion is 2029-03.
What conditions does trial NCT01665326 study?
This clinical trial studies the following conditions: Pompe Disease.
What interventions are being tested in trial NCT01665326?
The interventions under investigation include: Observational (OTHER).
Who is sponsoring clinical trial NCT01665326?
This trial is sponsored by Duke University, which has 1,725 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01665326 being conducted?
This trial has 1 study location across North Carolina. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Pompe Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01665326's enrollment target sits among peer trials
400 3rd of 14 higher than 12 of 14 other Pompe Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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