Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Purpose
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.
Condition
- GSD1
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Genders
- All
- Accepts Healthy Volunteers
- No
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 <60 mg/dL (<3.33 mmol/L)
- Patient's GSDIa disease is stable as evidenced by no hospitalization for severe hypoglycemia during the 4-week period preceding the screening visit
Exclusion Criteria
- Anti-AAV8 neutralizing antibody titer ≥1:5
- Screening or Baseline (Day 0) blood glucose level <60 mg/dL (<3.33 mmol/L)
- Liver transplant, including hepatocyte cell therapy/transplant
- Presence of liver adenoma >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 × ULN, alkaline phosphatase >2.5 × ULN
Note additional inclusion/exclusion criteria may apply, per protocol.
Study Design
- Phase
- Phase 1/Phase 2
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental DTX401 Dose 1 |
DTX401 solution for intravenous (IV) infusion |
|
Experimental DTX401 Dose 2 |
DTX401 solution for intravenous (IV) infusion |
|
Experimental DTX401 Dose 3 |
DTX401 solution for intravenous (IV) infusion |
|
Recruiting Locations
UT Health - McGovern Medical School
Houston, Texas 77030
Houston, Texas 77030
More Details
- NCT ID
- NCT03517085
- Status
- Recruiting
- Sponsor
- Ultragenyx Pharmaceutical Inc