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Glycogen Storage Disease Type Ia: full-pipeline trial registry
4 of 6 indexed Glycogen Storage Disease Type Ia trials are currently recruiting.
Corpus placement: #2,866 of 28,707 conditions by registered trial count.
6 US clinical trials · 4 currently recruiting · #2,866 of 28,707 conditions by registered trial count
Glycogen Storage Disease Type Ia: 6 US clinical trials tracked, 4 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.
Enrollment posture brief
Registry condition glycogen-storage-disease-type-ia
OPEN 4 | RECR 67% | LATE 1 | EARLY 1 | RANK #2866 | SPON 3
Volume-matched condition peers
Nearest open-share peer: Adenocarcinoma of Lung (67% recruiting · 6 trials)
Glycogen Storage Disease Type Ia lists 4 open slots (67% of 6 indexed trials). Peers are matched on registered volume, not therapeutic-area browse lists. How peer matching works →
Key findings
What ClinicalTrials.gov does not surface for Glycogen Storage Disease Type Ia on its own pages , computed from the registry mirror as of 2026-08-08. Each line carries its own denominator so it can be quoted as it stands.
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Glycogen Storage Disease Type Ia is recruiting above its size-band peers.
4 of 6 indexed trials (66.7%) are open to enrollment, against a 46.5% average across 27,806 conditions with fewer than 20 trials. The peer set uses the same ClinicalTrials.gov-derived counts as this page.
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One sponsor concentrates Glycogen Storage Disease Type Ia research.
Ultragenyx Pharmaceutical accounts for 3 of 6 trials (50%). 3 distinct sponsors appear on at least one study for this condition label.
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Most Glycogen Storage Disease Type Ia trials plan to enrol fewer than 100 people.
Median target enrolment is 36 among the 6 trials that report a genuine target (sentinel values excluded). 5 of those (83.3%) plan for fewer than 100 participants.
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Glycogen Storage Disease Type Ia research leans observational.
2 interventional and 4 observational studies make up this index (33.3% interventional). Study type is sponsor-reported on ClinicalTrials.gov.
Open and 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
Named interventions
Most-linked intervention names on Glycogen Storage Disease Type Ia records (sponsor-reported on ClinicalTrials.gov). Counts are trial links, not unique products.
US sites by state
Facility locations filed on Glycogen Storage Disease Type Ia trials. A trial with sites in several states counts in each.
Source: ClinicalTrials.gov, National Library of Medicine. Data is informational only.
Related
What to do with this Glycogen Storage Disease Type Ia page
6 registered trials, 4 currently recruiting, is a starting point for a conversation, not a diagnosis or a recommendation.
- 4 Glycogen Storage Disease Type Ia trials are currently recruiting, check eligibility criteria before contacting a site. Browse recruiting trials
- Ultragenyx Pharmaceutical sponsors the most Glycogen Storage Disease Type Ia trials on record, review their full trial history before evaluating a specific study. See Ultragenyx Pharmaceutical's trials
- Bring the specific trial ID (NCT number) to your own doctor before enrolling, this page is a directory, not medical guidance. Read the trial-finding guide
Trial and recruiting counts reflect ClinicalTrials.gov registry status as of the data vintage above; individual trial eligibility and enrollment status can change.
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This Glycogen Storage Disease Type Ia condition card totals 6 registered trials; most-linked intervention Placebo for oral prednisolone; top US site state Connecticut. See our editorial standards & corrections policy, the methodology behind these numbers, the data changelog, or report a data error.