Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03517085 · ClinicalTrials.gov registry record · Phase 1
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
A Phase 1 study of GSD1, sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 6
- Study locations
NCT03517085: Completed Phase 1 study of GSD1, sponsored by Ultragenyx Pharmaceutical.
NCT03517085 is a Phase 1 study of GSD1 that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 12 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower). The trial reports 6 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03517085, a Phase 1 study of GSD1, has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 6
- Study locations
Study Summary
The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.
Primary Outcome
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
Conditions Studied
Interventions
- GENETIC DTX401
- DRUG steroid regimen
Study Locations (6)
Connecticut
- UCONN Health - Farmington
Michigan
- Michigan Medicine University of Michigan - Ann Arbor
Texas
- UT Health - McGovern Medical School - Houston
Quebec
- Montreal Children Hospital, McGill University Health Centre - Montreal
Other
- University Medical Center Groningen - Groningen
A Coruna
- Complejo Hospitalario Universitario de Santiago - Santiago de Compostela
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2018-05-18 |
| Est. Completion | 2021-11-02 |
| Phase | Phase 1 |
What the finished NCT03517085 record still lists
NCT03517085 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower).
The record links to 1 condition, with GSD1 appearing as the primary indexed condition, and to 2 interventions - of which DTX401 is the first listed.
NCT03517085 lists 6 locations in 6 states (Connecticut, Michigan, Texas).
Frequently Asked Questions
What is clinical trial NCT03517085 about?
NCT03517085 is a clinical study titled "Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)". The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each dose level.
What is the current status of trial NCT03517085?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2018-05-18. Estimated completion is 2021-11-02.
What conditions does trial NCT03517085 study?
This clinical trial studies the following conditions: GSD1.
What interventions are being tested in trial NCT03517085?
The interventions under investigation include: DTX401 (GENETIC), steroid regimen (DRUG).
Who is sponsoring clinical trial NCT03517085?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03517085 being conducted?
This trial has 6 study locations across Connecticut, Michigan, Texas, Quebec, A Coruna. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03517085's enrollment target sits among peer trials
12 1852nd of 2000 higher than 90 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT02602769 · 12 participants
Whole Transcriptome Profiling and Metabolic Phenotyping in Children With ROHHAD Syndrome
- NCT02605421 · 12 participants · Phase 2
Myeloablative Consolidation Therapy and Tandem Autologous Stem Cell Rescue in Patients With High-Risk Neuroblastoma
- NCT02619448 · 12 participants · Early Phase 1
Concurrent Chemotherapy Plus HFR Radiation Therapy in Inoperable NSCLC
- NCT03311503 · 12 participants · Phase 1
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI