GBE Deficiency (GSD IV and APBD) Natural History Study

Part of paid clinical trials in Durham, North Carolina.

Sponsor
Duke University
Study ID
NCT02683512
Status
Recruiting
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Conditions

  • APBD
  • Adult Polyglucosan Body Disease
  • GSD IV
  • GSD4
  • Glycogen Storage Disease Type IV

Eligibility Criteria

Sex
ALL
Age
0 Years - 90 Years
Healthy Volunteers
Not accepted

Interventions

  • No intervention — OTHER
    This is an observational study that consists of data abstraction from patient medical records.

Study Details

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.

Key Dates

Start date
Dec 31, 2015
Status verified
May 2026
Primary completion
Dec 31, 2035
Completion
Dec 31, 2035

Study Design

Enrollment
200 participants (estimated)

Primary Outcome Measure

Progression of disease [ Time Frame: Duration of study, approximately 10 years ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Duke University Medical CenterDurhamNorth Carolina27710
Rebecca L Koch, PhD, RDN
[email protected]
919-681-8823
Nisha Dalal, M.S. CCC-SLP
[email protected]
919-668-3107
Priya S Kishnani, MD (PRINCIPAL_INVESTIGATOR)

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Duke University Medical Center· Durham, NC