- 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
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Showing 1–6
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- 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
- 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
- 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
- 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
- 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