Trial · NCT05139316
A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
NCT05139316, Phase 3, Completed, Ultragenyx Pharmaceutical Inc
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa). Trials, record NCT05139316. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05139316
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)
- NCT number
- NCT05139316
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 52 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- Glycogen Storage Disease Type IA
- Interventions
- DTX401, Placebo, Oral prednisolone, Placebo for oral prednisolone
- First posted
- 2021-12-01
- Last updated
- 2026-10-14
- Other registries' IDs
- eudract:2020-004184-12
Source ClinicalTrials.gov
Registered outcomes20
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Main Study: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams) | Baseline, Weeks 24, 30, 36, 42 and 48 | The least square (LS) mean and standard error (SE) are based on mixed-model for repeated measures (MMRM) analysis. |
| Primary | Japan Cohort: Percent Change From Baseline to Week 48 in Daily Cornstarch Intake (Grams) | Baseline, Weeks 24, 30, 36, 42 and 48 | |
| Secondary | Main Study: Change From Baseline to Week 48 in Number of Total Daily Doses of Cornstarch | Baseline, Weeks 24, 30, 36, 42 and 48 | The LS mean and SE are based on MMRM analysis. |
| Secondary | Japan Cohort: Change From Baseline to Week 48 in Number of Total Daily Doses of Cornstarch | Baseline, Weeks 24, 30, 36, 42 and 48 | |
| Secondary | Main Study: Change From Baseline to Week 48 in Percentage of Glucose Values in Hypoglycemic Range (Less Than (<) 70 Milligrams Per Deciliter (mg/dL) [3.9 Millimoles Per Liter (mmol/L)]) | Baseline, Weeks 24, 30, 36, 42 and 48 | The LS mean and SE are based on MMRM analysis. |
| Secondary | Japan Cohort: Change From Baseline to Week 48 in Percentage of Glucose Values in Hypoglycemic Range (< 70 mg/dL [3.9 mmol/L]) | Baseline, Weeks 24, 30, 36, 42 and 48 | |
| Secondary | Main Study: Patient Global Impression of Change (PGIC) Assessment Score at Week 48 | Week 48 | The PGIC captures the participants' perspective on changes in their glycogen storage disease type Ia (GSDIa) since the start of the study using a 7-point response scale ranging from much improved (+ 3) to much worse (- 3). The total score ranges from + 3 to - 3, with a higher score indicating best outcome. |
| Secondary | Japan Cohort: PGIC Assessment Score at Week 48 | Week 48 | The PGIC captures the participants' perspective on changes in their GSDIa since the start of the study using a 7-point response scale ranging from much improved (+ 3) to much worse (- 3). The total score ranges from + 3 to - 3, with a higher score indicating best outcome. |
| Secondary | Main Study: Change From Baseline to Week 48 in Time to Hypoglycemia (< 54 mg/dL [3.0 mmol/L]) During the Controlled Fasting Challenge (CFC) | Baseline, Weeks 36 and 48 | The LS mean and SE are based on MMRM analysis. |
| Secondary | Japan Cohort: Change From Baseline to Week 48 in Time to Hypoglycemia (< 54 mg/dL [3.0 mmol/L]) During the CFC | Baseline, Weeks 36 and 48 |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT05139316
GET https://conductscience.org/api/trials/v1/NCT05139316/registered