GBE Deficiency (GSD IV and APBD) Natural History Study
NCT02683512 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 200
Last updated 2026-05-05
Summary
Collection and review of clinical information related to glycogen branching enzyme (GBE) deficiency, diagnosed as Glycogen Storage Disease Type IV (GSD IV) or Adult Polyglucosan Body Disease (APBD generated during clinic visits.
Conditions
- Glycogen Storage Disease Type IV
- Adult Polyglucosan Body Disease
- GSD4
- GSD IV
- APBD
Interventions
- OTHER
-
No intervention
This is an observational study that consists of data abstraction from patient medical records.
Sponsors & Collaborators
- lead OTHER
Principal Investigators
-
Priya Kishnani, MD · Duke University
Eligibility
- Min Age
- 0 Years
- Max Age
- 90 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2015-12-31
- Primary Completion
- 2035-12-31
- Completion
- 2035-12-31
Countries
- United States
Study Locations
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