- 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
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2021 – 2026
- U.S. locations
- 9
- States / cities
- Orange, California • Aurora, Colorado • Farmington, Connecticut + 6 more
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
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.
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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