Medical Information Only. Consult your healthcare provider before considering clinical trial enrollment.
Glycogen Storage Disease Type Ia clinical trials
4 of 6 indexed Glycogen Storage Disease Type Ia trials are currently recruiting.
6 US clinical trials · 4 currently recruiting
Glycogen Storage Disease Type Ia is the subject of 6 registered US clinical trials on ClinicalTrials.gov, 4 of them currently open to new participants. 1 are in Phase 3-4 (later-stage) and 1 in Phase 1-2 (earlier-stage). The most active sponsor is Ultragenyx Pharmaceutical, running 3 of these trials.
The research picture
Glycogen Storage Disease Type Ia has 6 registered US clinical trials, 4 of them open to new participants right now, about 67% of the total.
- 4
- recruiting participants now
- 67%
- of trials open to enrollment
- 1
- in Phase 3–4 (later-stage)
- 3
- top sponsor: Ultragenyx Pharmaceutical
Active & Recent Trials
6 total, page 1 of 1
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Ultragenyx Pharmaceutical
NCT06636383
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
NCT06735755
Accuracy of Lactate Meter in GSDIa
Connecticut Children's Medical Center
NCT06843330
Accuracy of Home Lactate Meter and Accu-chek Glucometer in Patients With Glycogen Storage Disease
Connecticut Children's Medical Center
NCT07459582
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
Ultragenyx Pharmaceutical
NCT05139316
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
Ultragenyx Pharmaceutical
NCT03970278
Phase Distribution
| Phase | Trial count |
|---|---|
| Phase 1 | 1 |
| Phase 3 | 1 |
Top Sponsors
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Reading the Glycogen Storage Disease Type Ia Trial Landscape
6 indexed Glycogen Storage Disease Type Ia studies · 4 recruiting (67%).
Phase mix: 1 late (III/IV) vs 1 early (I/II).
Top sponsor: Ultragenyx Pharmaceutical (3 trials), 2 others listed. 6 shown below, recruiting first.
How to read this condition's trial landscape
That ratio is a useful proxy for activity level: a high share of recruiting studies often signals that research interest is current and that new enrollment opportunities are appearing, while a low share typically means the field is dominated by completed or follow-up work where most participant spots have already been filled. These counts reflect the public registry only and include studies at every stage of design, so they should be read as an index of research attention rather than as a measure of treatment availability.
Phase 1 and Phase 2 studies focus on early safety signals, dosing, and preliminary effect, while Phase 3 studies are typically the larger efficacy and safety trials submitted toward regulatory review, and Phase 4 studies follow approved interventions in real-world use. A condition weighted toward later phases often reflects a mature research pipeline with several interventions already close to or past approval, whereas a heavier early-phase tilt suggests the field is still exploring new mechanisms and candidate approaches.
All figures are derived from the public ClinicalTrials.gov dataset maintained by the National Library of Medicine and are reproduced here for reference. Inclusion of a trial, sponsor, or intervention on this page is neither an endorsement nor a recommendation, eligibility, protocol changes, and site-level status can shift frequently, so always verify current details on ClinicalTrials.gov and consult a qualified healthcare provider before acting on anything you see here.
Frequently Asked Questions
How many clinical trials are there for Glycogen Storage Disease Type Ia?
PlainTrial tracks 6 US clinical trials for Glycogen Storage Disease Type Ia, of which 4 are currently recruiting participants. Data sourced from ClinicalTrials.gov.
How do I find a recruiting trial for Glycogen Storage Disease Type Ia?
Use the trial list above filtered by "Recruiting" status, or visit our trial finder at /recruiting to search by condition and state. Always discuss trial participation with your healthcare provider before enrolling.
Is this data current?
Data is sourced from ClinicalTrials.gov and reflects our most recent data pull. Trial status may have changed since then. Always verify current information at ClinicalTrials.gov before making decisions about participation.
Related
Disclaimer: This information is provided for informational purposes only and does not constitute professional advice. Data is sourced from ClinicalTrials.gov (National Library of Medicine). Consult a qualified professional before making decisions based on this data.
Read our methodology - how this data is sourced, computed, and verified.
Source: ClinicalTrials.gov (NIH/NLM) ClinicalTrials.gov AACT registry · 2026 Trial counts and statuses sourced from ClinicalTrials.gov. Sponsor counts include both industry and federal/academic sponsors.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page aggregates every registered trial for this condition, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.