Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Key facts

Sponsor

Ultragenyx Pharmaceutical Inc

Principal Investigator

Medical Director

Ultragenyx Pharmaceuticals Inc.

Intervention

No Intervention(other)

Enrollment

140 enrolled

Eligibility

2 years · All sexes

Timeline

2024 – 2036

Study locations (19)

• Children's Hospital of Orange County, Orange, California, United States
• Children's Hospital Colorado, Denver, Colorado, United States
• University of Connecticut Health Center, Hartford, Connecticut, United States
• University of Michigan, Ann Arbor, Michigan, United States
• Duke University Medical Center, Durham, North Carolina, United States
• The Cleveland Clinic Foundation, Cleveland, Ohio, United States
• Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
• University of Texas Health Science Center at Houston, Houston, Texas, United States
• Primary Children's Hospital, Salt Lake City, Utah, United States
• Hospital de Clinicas de Porto Alegre (HCPA) - PPDS, Porto Alegre, Rio Grande do Sul, Brazil
• McGill University Health Center, Montreal, Quebec, Canada
• Rigshospitalet, Copenhagen, Denmark
• Universitätsklinikum Hamburg Eppendorf, Hamburg, Germany
• University of Naples, Naples, Campania, Italy
• Istituto G Gaslini Ospedale Pediatrico IRCCS, Genoa, Italy
• +4 more locations on ClinicalTrials.gov

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

Other trials for Glycogen storage disease due to glucose-6-phosphatase deficiency

Additional recruiting or active studies for the same condition.

• RECRUITINGPHASE1, PHASE2NCT06735755
  A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa)
  Beam Therapeutics Inc.

See all trials for Glycogen storage disease due to glucose-6-phosphatase deficiency →