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NCT03970278 · ClinicalTrials.gov registry record
Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia
A clinical trial of Glycogen Storage Disease Type Ia and Von Gierke's Disease (GSD Type Ia), sponsored by Ultragenyx Pharmaceutical.
- Completed
- Registry status
- 12
- Enrollment target
- 6
- Study locations
NCT03970278: Completed study of Glycogen Storage Disease Type Ia and Von Gierke's Disease (GSD Type Ia), sponsored by Ultragenyx Pharmaceutical.
NCT03970278 is a study of Glycogen Storage Disease Type Ia and Von Gierke's Disease (GSD Type Ia) that has completed, run by Ultragenyx Pharmaceutical. The registered enrollment target is 12 participants, below the 49-participant average among 5 other Glycogen Storage Disease Type Ia trials with a reported enrollment target (76% lower). The trial reports 6 study locations across 6 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03970278, a study of Glycogen Storage Disease Type Ia and Von Gierke's Disease (GSD Type Ia), has completed, sponsored by Ultragenyx Pharmaceutical.
- COMPLETED
- Registry status
- 12 participants
- Enrollment target
- 6
- Study locations
Study Summary
The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.
Primary Outcome
An adverse event (AE) is defined as any untoward medical occurrence, regardless of its causal relationship to study product. A TEAE is defined as any AE not present prior to the initiation of the drug treatment or any AE already present that worsens in either intensity or frequency following exposure to the drug treatment. A serious TEAE is an AE that meets any of the following criteria in the view of either the Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or pr
Conditions Studied
Interventions
- OTHER No intervention
Study Locations (6)
Connecticut
- UCONN Health - Farmington
Michigan
- Michigan Medicine University of Michigan - Ann Arbor
Texas
- University of Texas Health Science Center at Houston - 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 | 2019-07-15 |
| Est. Completion | 2025-02-25 |
What the finished NCT03970278 record still lists
NCT03970278 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 49-participant average among 5 other Glycogen Storage Disease Type Ia trials with a reported enrollment target (76% lower).
The record links to 2 conditions, with Glycogen Storage Disease Type Ia appearing as the primary indexed condition, and to 1 intervention - of which No intervention is the first listed.
NCT03970278 lists 6 locations in 6 states (Connecticut, Michigan, Texas).
Frequently Asked Questions
What is clinical trial NCT03970278 about?
NCT03970278 is a clinical study titled "Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia". The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.
What is the current status of trial NCT03970278?
This trial is currently completed. The enrollment target is 12 participants. The study started on 2019-07-15. Estimated completion is 2025-02-25.
What conditions does trial NCT03970278 study?
This clinical trial studies the following conditions: Glycogen Storage Disease Type Ia, Von Gierke's Disease (GSD Type Ia).
What interventions are being tested in trial NCT03970278?
The interventions under investigation include: No intervention (OTHER).
Who is sponsoring clinical trial NCT03970278?
This trial is sponsored by Ultragenyx Pharmaceutical, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03970278 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.
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