Glycogen Storage Disease Type Ia (GSDIa). MedDRA version: 20.1 Level: LLT Classification code 10056911 Term: Glycogen storage disease type IA System Organ Class: 100000004850
Conditions
Interventions
Product Name: DTX401
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Not yet assigned.
Current Sponsor code: DTX401
Concentration unit: Other
Concentration type: not le
Sponsors
Ultragenyx Pharmaceutical, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Willing and able to provide written informed consent. 2. Males and females >=18 years of age. 3. Documented GSDIa with confirmation by molecular testing. 4. Documented history of >=1 hypoglycemic event with glucose =65 years) yes F.1.3.1 Number of subjects for this age range 2
Exclusion criteria
Exclusion criteria: 1. Screening or Baseline (Day 0) glucose level 5 cm in size. 4. Presence of liver adenoma >3 cm and =0.5 cm per year. 5. Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase > the upper limit of normal (ULN), total bilirubin >1.5 × ULN, or alkaline phosphatase >2.5 × ULN. Liver function tests may be repeated during the screening period at the investigator’s discretion. 6. Serum creatinine >2.0 mg/dL. 7. Triglycerides >=1000 mg/dL at the time of the screening visit. 8. Presence of active, or history of treatment for, hepatitis B virus or hepatitis C virus infection. 9. History of human immunodeficiency virus infection AND any of the following: CD4+ cell count 200 copies/mL, on 2 separate occasions, as measured by polymerase chain reaction. 10. History of a malignancy for which the subject has received treatment in the past 2 years except for prostate cancer treated with watchful waiting or surgically removed nonmelanoma skin cancer. 11. Active infection (viral or bacterial). 12. Anti-AAV8 neutralizing antibody titer >=1:5. 13. Participation (current or previous) in another gene transfer study. 14. Participation in another investigational product study within 3 months of Screening. 15. Has a positive serum pregnancy test at Screening (females of childbearing potential only), a positive urine pregnancy test at Baseline (Day 0; females of childbearing potential only), or is nursing. 16. Has any other significant medical condition that the investigator feels would be a risk to the subject or would impede the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To determine the safety of single IV doses of DTX401 in adults with GSDIa, including the incidence of dose-limiting toxicities (DLTs).;Secondary Objective: To establish a dose of DTX401 that achieves symptom-free euglycemia (glucose >=60 mg/dL [>=3.33 mmol/L]) in a setting of a controlled fasting challenge to allow further clinical development.;Primary end point(s): The incidence of AEs, including the incidence of DLTs at each dose level, TEAEs, and SAEs for each dosing cohort, assessed by severity and relationship to study product.;Timepoint(s) of evaluation of this end point: From the time the subject signs the Informed Consent Form (ICF) and up to 30 days after the end of the study / early withdrawal visit. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The change from baseline in time (in minutes) to first hypoglycemic event (defined as glucose <60 mg/dL [<3.33 mmol/L]) during a controlled fasting challenge at 6, 12, 24, and 52 weeks after IV administration of DTX401.;Timepoint(s) of evaluation of this end point: Day 0 and weeks 6, 12, 24 and 52. | — |
Countries
Canada, Netherlands, Spain, United States
Contacts
Public ContactClinical Development
Ultragenyx Pharmaceutical, Inc.
Outcome results
None listed