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

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ClinicalTrials.gov Identifier: NCT03517085
Recruitment Status : Completed
First Posted : May 7, 2018
Last Update Posted : November 23, 2021
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Tracking Information
First Submitted Date  ICMJE April 24, 2018
First Posted Date  ICMJE May 7, 2018
Last Update Posted Date November 23, 2021
Actual Study Start Date  ICMJE July 24, 2018
Actual Primary Completion Date November 2, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 24, 2018)
Adverse Events (AEs), Treatment-emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs) [ Time Frame: Up to 52 Weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 18, 2021)
Change from Baseline in Time (Minutes) to First Hypoglycemic Event During a Controlled Fasting Challenge at Weeks 12, 24, and 52 [ Time Frame: Baseline and Weeks 12, 24, and 52 ]
The change from baseline in time (in minutes) to first hypoglycemic event (defined as glucose <54 mg/dL [<3.0 mmol/L]) during a controlled fasting challenge after IV administration of DTX401).
Original Secondary Outcome Measures  ICMJE
 (submitted: April 24, 2018)
Change from Baseline in Time (Minutes) to First Hypoglycemic Event During a Controlled Fasting Challenge at Weeks 6, 12, 24, and 52 [ Time Frame: Baseline and Weeks 6, 12, 24, and 52 ]
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 after IV administration of DTX401).
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Official Title  ICMJE A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6- Phosphatase (G6Pase) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
Brief 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.
Detailed Description Subjects enrolled in the 401GSDIA01 study will be monitored for 52 weeks following DTX401 administration. Subjects in Cohorts 1, 2, and 3 will receive reactive oral steroid treatment for possible vector-induced hepatitis following treatment with DTX401. Subjects in Cohort 4 will receive prophylactic oral steroid treatment to prevent possible vector-induced hepatitis. After completion of the Week 52 visit or early withdrawal, subjects will be offered enrollment into a 4-year extension study.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE GSD1
Intervention  ICMJE Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC
Study Arms  ICMJE
  • Experimental: DTX401 Dose 1
    DTX401 solution for intravenous (IV) infusion
    Intervention: Genetic: DTX401
  • Experimental: DTX401 Dose 2
    DTX401 solution for intravenous (IV) infusion
    Intervention: Genetic: DTX401
  • Experimental: DTX401 Dose 3
    DTX401 solution for intravenous (IV) infusion
    Intervention: Genetic: DTX401
  • Experimental: DTX401 Dose 4
    DTX401 solution for intravenous (IV) infusion
    Intervention: Genetic: DTX401
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: November 19, 2020)
12
Original Estimated Enrollment  ICMJE
 (submitted: April 24, 2018)
9
Actual Study Completion Date  ICMJE November 2, 2021
Actual Primary Completion Date November 2, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 <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

Key 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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   Netherlands,   Spain,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03517085
Other Study ID Numbers  ICMJE 401GSDIA01
1706-1617 ( Other Identifier: NIH Protocol Registration Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Ultragenyx Pharmaceutical Inc
Study Sponsor  ICMJE Ultragenyx Pharmaceutical Inc
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Ultragenyx Pharmaceutical Inc
PRS Account Ultragenyx Pharmaceutical Inc
Verification Date November 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP