Phase 2 N=12 Treatment

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

GSD1

Bottom Line

View on ClinicalTrials.gov: NCT03517085 ↗

Enrolled (actual)

Serious AEs

33.3%

Results posted

Nov 2022

Primary outcome: Primary: Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs) — 1; 1; 2; 2 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: DTX401 (Genetic); steroid regimen (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Ultragenyx Pharmaceutical Inc
Primary completion: Nov 2021

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants With Adverse Events (AEs) Treatment-Emergent AEs (TEAEs) Serious TEAEs, Discontinuations Due to TEAEs, and Dose-Limiting Toxicities (DLTs)	1; 1; 2; 2; 3; 3	—
SECONDARY Change From Baseline in Time to First Hypoglycemic Event Over Time	3.3; 1.7; 1.4; 4.3; 0.5; 0.7	—

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.

Eligibility Criteria

Key Inclusion Criteria

Males and females ≥18 years of age
Documented GSDIa with confirmation by molecular testing
Documented history of ≥1 hypoglycemic event with blood glucose 5 cm in size
Presence of liver adenoma >3 cm and ≤5 cm in size that has a documented annual growth rate of ≥0.5 cm per year
Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN), total bilirubin > 1.5 x ULN, or alkaline phosphatase > 2.5 x ULN

Note additional inclusion/exclusion criteria may apply, per protocol.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03517085). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.