Glycogen Storage Disease Type IA
2
Pipeline Programs
1
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
0
0
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Ultragenyx PharmaceuticalNOVATO, CA
2 programs1
1
DTX401Phase 31 trial
[6,6-2H2]glucosePhase 1/21 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Ultragenyx PharmaceuticalDTX401
Ultragenyx Pharmaceutical[6,6-2H2]glucose
Clinical Trials (2)
Total enrollment: 69 patients across 2 trials
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Start: Nov 2021Est. completion: Feb 202649 patients
Phase 3Completed
Endogenous Glucose Production in Patients With Glycogen Storage Disease Type Ia
Start: Jan 2021Est. completion: Feb 202220 patients
Phase 1/2Completed
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 late-stage (Phase 3) programs — potential near-term approvals
1 companies competing in this space