MedTrace Logo

Rare Glycogen Storage Diseases Natural History Study

NCT06795152 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 200

Last updated 2026-01-07

No results posted yet for this study

Summary

The purpose of this study is to collect and study key medical data about several ultra-rare GSDs (Glycogen Storage Diseases) including, but not limited to, GSD types 0a, 0b, VII, X, XII, XIII, XV, PRKAG2 syndrome and Danon disease.

Conditions

  • GSD X
  • GSD XII
  • GSD XIII
  • GSD XV
  • PGBM2
  • PRKAG2
  • Danon Disease
  • Polyglucosan Body Myopathy Type 1
  • Glycogen Storage Disease
  • GSD Type 0A
  • GSD Type 0B
  • GSD VII
  • Tarui Disease
  • Polyglucosan Body Myopathy Type 2
  • RBCK1 Deficiency

Interventions

OTHER

No intervention

Observational. Natural history study.

Sponsors & Collaborators

Principal Investigators

  • Priya Kishnani, M.D. · Duke

Eligibility

Min Age
0 Years
Max Age
90 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-12-23
Primary Completion
2034-12-31
Completion
2034-12-31

Countries

  • United States

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06795152 on ClinicalTrials.gov