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 : Recruiting

First Posted : May 7, 2018

Last Update Posted : July 3, 2019

See Contacts and Locations

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

July 3, 2019

Actual Study Start Date ^ICMJE

July 24, 2018

Estimated Primary Completion Date

September 19, 2020 (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

Complete list of historical versions of study NCT03517085 on ClinicalTrials.gov Archive Site

Current 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).

Original Secondary Outcome Measures ^ICMJE

Same as current

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

Not Provided

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

Publications *

Not Provided

* 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

Recruiting

Estimated Enrollment ^ICMJE

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date ^ICMJE

September 19, 2020

Estimated Primary Completion Date

September 19, 2020 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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.

Sex/Gender ^ICMJE

Sexes Eligible for Study:

All

Ages ^ICMJE

18 Years and older (Adult, Older Adult)

Accepts Healthy Volunteers ^ICMJE

Contacts ^ICMJE

Contact: Patients Contact: Patient Advocacy	1-415-483-8800	patientadvocacy@ultragenyx.com
Contact: HCPs Contact: Medical Information	1-888-756-8657	medinfo@ultragenyx.com

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

July 2019

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

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