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ClinicalTrials.gov public records Last synced May 22, 2026, 3:48 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–11 of 11 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Glycogen Storage Disease Type IA Clear all
Completed No phase listed Observational
View directory record Official record
Conditions
Glycogen Storage Disease Type IA
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
8 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 28, 2024 · Synced May 22, 2026, 3:48 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease
Interventions
mRNA-3745
Drug
Lead sponsor
ModernaTX, Inc.
Industry
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
8
States / cities
Farmington, Connecticut • Boston, Massachusetts • Durham, North Carolina + 4 more
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced May 22, 2026, 3:48 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Glycogen Storage Disease Type Ia
Interventions
No Intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
2 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2036
U.S. locations
9
States / cities
Orange, California • Denver, Colorado • Hartford, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced May 22, 2026, 3:48 AM EDT
Completed No phase listed Observational Results available
View directory record Official record
Conditions
Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
Interventions
No intervention
Other
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced May 22, 2026, 3:48 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Glycogen Storage Disease Type IA, Glycogen Storage Disease Type I, Glycogen Storage Disease Type IB, Glycogen Storage Disease Xi
Interventions
Lactate Meter, Accu Chek
Other · Device
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
Up to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced May 22, 2026, 3:48 AM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
GSD1
Interventions
DTX401, steroid regimen
Genetic · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
3
States / cities
Farmington, Connecticut • Ann Arbor, Michigan • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 17, 2022 · Synced May 22, 2026, 3:48 AM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease Type Ia
Interventions
BEAM-301: Single dose of BEAM-301 administered by IV
Drug
Lead sponsor
Beam Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
3
States / cities
Orange, California • Farmington, Connecticut • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced May 22, 2026, 3:48 AM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Ornithine Transcarbamylase Deficiency, Wilson Disease, Glycogen Storage Disease Type IA
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
18 Years and older
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Akron, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 11, 2023 · Synced May 22, 2026, 3:48 AM EDT
Completed Phase 3 Interventional
View directory record Official record
Conditions
Glycogen Storage Disease Type IA
Interventions
DTX401, Placebo, Oral prednisolone, Placebo for oral prednisolone
Genetic · Other · Drug
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
8 Years and older
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
9
States / cities
Orange, California • Aurora, Colorado • Farmington, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced May 22, 2026, 3:48 AM EDT
Completed No phase listed Observational Results available
View directory record Official record
Conditions
Inflammatory Bowel Disease, Glycogen Storage Disease Type Ia
Interventions
Glycogen Storage Disease type Ia patients
Other
Lead sponsor
University of Florida
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 27, 2020 · Synced May 22, 2026, 3:48 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Glycogen Storage Disease Type Ia
Interventions
blood glucose meter; blood lactate meter
Device
Lead sponsor
Connecticut Children's Medical Center
Other
Eligibility
0 Years to 60 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Hartford, Connecticut
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced May 22, 2026, 3:48 AM EDT