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Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

NCT03517085 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2022-11-18

Study results available
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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.

Conditions

  • GSD1

Interventions

GENETIC

DTX401

DTX401 administered as a single peripheral intravenous (IV) infusion

DRUG

steroid regimen

prednisone or prednisolone to manage alanine aminotransferase (ALT) elevation

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceutical Inc

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-05-18
Primary Completion
2021-11-02
Completion
2021-11-02
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Netherlands
  • Spain

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03517085 on ClinicalTrials.gov