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
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Sponsor:
Ultragenyx Pharmaceutical Inc
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc
Tracking Information | |||||
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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 |
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 |
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).
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Original Secondary Outcome Measures ICMJE |
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).
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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 |
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Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | GSD1 | ||||
Intervention ICMJE | Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC
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Study Arms ICMJE |
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Publications * |
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* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
12 | ||||
Original Estimated Enrollment ICMJE |
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:
Key Exclusion Criteria:
Note additional inclusion/exclusion criteria may apply, per protocol. |
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Sex/Gender ICMJE |
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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 ) |
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Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | Ultragenyx Pharmaceutical Inc | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | Ultragenyx Pharmaceutical Inc | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Not Provided | ||||
Investigators ICMJE |
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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 |