Trial · NCT03517085
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
NCT03517085, Phase 1, Phase 2, Completed, Ultragenyx Pharmaceutical Inc; results first posted 2022-11-18
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa). Trials, record NCT03517085. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03517085
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
- NCT number
- NCT03517085
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- Ultragenyx Pharmaceutical Inc
- Conditions
- GSD1
- Interventions
- DTX401, steroid regimen
- First posted
- 2018-05-07
- Last updated
- 2022-11-18
- Other registries' IDs
- eudract:2016-003023-30
- Results first posted
- 2022-11-18
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug through the End of Study (EOS)/Early Withdrawal visit (up to Week 52) plus 30 days. | An AE is defined as any untoward medical occurrence, regardless of its causal relationship to study product. An SAE is defined as any event that: results in death; is immediately life threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; results in a congenital anomaly/birth defect; or an important medical event, in the opinion of the investigator. The relationship to study drug was categorized as unrelated, possible, probable or definite. A DLT is defined as any AE/SAE ≥ Grade 3 that is considered by the Investigator and/or Sponsor to be related to DTX401, based on the Nation Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version 5.0 or later version. Per protocol, SAEs that occurred \> 30 days after EOS or Early withdrawal visit, did not need to be reported unless Investigator considered them related to study product. |
| Secondary | Change From Baseline in Time to First Hypoglycemic Event Over Time | Baseline, Weeks 12, 24, 52 | The change from baseline in time (in hours) to first hypoglycemic event (defined as glucose \< 54 mg/dL \[\< 3.0 mmol/L\]) during a controlled fasting challenge at 12, 24, and 52 weeks after IV administration of DTX401. A positive change from baseline is favorable. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | AEs Prior to Dosing: From signing the informed consent form (ICF) to first dose of study drug. TEAEs: From first dose of study drug through the End of Study (EOS)/Early Withdrawal visit (up to Week 52) plus 30 days. | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Change From Baseline in Time to First Hypoglycemic Event Over Time | Baseline, Weeks 12, 24, 52 | Participants with an assessment at given time point | hours | MEAN |
Source ClinicalTrials.gov
Results by group47
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 1 | Any AE prior to dosing | 1 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 2 | Any AE prior to dosing | 1 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 3 | Any AE prior to dosing | 2 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 4 | Any AE prior to dosing | 2 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 1 | Any TEAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 2 | Any TEAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 3 | Any TEAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 4 | Any TEAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 1 | Related TEAE | 3 | Participants | |
| Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) | DTX401 Cohort 2 | Related TEAE | 3 | Participants |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | DTX401 Cohort 1 | 3 | |
| Overall Study | STARTED | DTX401 Cohort 2 | 3 | |
| Overall Study | STARTED | DTX401 Cohort 3 | 3 | |
| Overall Study | STARTED | DTX401 Cohort 4 | 3 | |
| Overall Study | COMPLETED | DTX401 Cohort 1 | 3 | |
| Overall Study | COMPLETED | DTX401 Cohort 2 | 3 | |
| Overall Study | COMPLETED | DTX401 Cohort 3 | 3 | |
| Overall Study | COMPLETED | DTX401 Cohort 4 | 3 | |
| Overall Study | NOT COMPLETED | DTX401 Cohort 1 | 0 | |
| Overall Study | NOT COMPLETED | DTX401 Cohort 2 | 0 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events525
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | DTX401 Cohort 1 | 0 | 3 | |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | DTX401 Cohort 2 | 0 | 3 | |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | DTX401 Cohort 3 | 1 | 3 | |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | DTX401 Cohort 4 | 0 | 3 | |
| other | Iron Deficiency Anaemia | Blood and lymphatic system disorders | Total | 1 | 12 | |
| other | Polycythaemia | Blood and lymphatic system disorders | DTX401 Cohort 1 | 0 | 3 | |
| other | Polycythaemia | Blood and lymphatic system disorders | DTX401 Cohort 2 | 1 | 3 | |
| other | Polycythaemia | Blood and lymphatic system disorders | DTX401 Cohort 3 | 0 | 3 | |
| other | Polycythaemia | Blood and lymphatic system disorders | DTX401 Cohort 4 | 0 | 3 | |
| other | Polycythaemia | Blood and lymphatic system disorders | Total | 1 | 12 |
1 to 10 of 525Source ClinicalTrials.gov
See all 525 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | DTX401 Cohort 1 | 0 | 3 |
| Total, all-cause mortality | DTX401 Cohort 2 | 0 | 3 |
| Total, all-cause mortality | DTX401 Cohort 3 | 0 | 3 |
| Total, all-cause mortality | DTX401 Cohort 4 | 0 | 3 |
| Total, all-cause mortality | Total | 0 | 12 |
| Total, other adverse events | DTX401 Cohort 1 | 3 | 3 |
| Total, other adverse events | DTX401 Cohort 2 | 3 | 3 |
| Total, other adverse events | DTX401 Cohort 3 | 3 | 3 |
| Total, other adverse events | DTX401 Cohort 4 | 3 | 3 |
| Total, other adverse events | Total | 12 | 12 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers3
The papers the trial lists on ClinicalTrials.gov.
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/NCT03517085
GET https://conductscience.org/api/trials/v1/NCT03517085/results?page=2