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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 : Recruiting
First Posted : May 7, 2018
Last Update Posted : June 4, 2019
Sponsor:
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Study Description
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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.

Condition or disease Intervention/treatment Phase
GSD1 Genetic: DTX401 Phase 1 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 9 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : July 24, 2018
Estimated Primary Completion Date : September 19, 2020
Estimated Study Completion Date : September 19, 2020

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Arms and Interventions
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Arm Intervention/treatment
Experimental: DTX401 Dose 1
DTX401 solution for intravenous (IV) infusion
 Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC
Experimental: DTX401 Dose 2
DTX401 solution for intravenous (IV) infusion
 Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC
Experimental: DTX401 Dose 3
DTX401 solution for intravenous (IV) infusion
 Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC


Outcome Measures
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Primary Outcome Measures :
  1. Adverse Events (AEs), Treatment-emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs) [ Time Frame: Up to 52 Weeks ]

Secondary Outcome Measures :
  1. 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).


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03517085


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

Locations
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United States, Connecticut
UCONN Health Recruiting
Farmington, Connecticut, United States, 06030-3213
United States, Michigan
Michigan Medicine University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
United States, Texas
UT Health - McGovern Medical School Recruiting
Houston, Texas, United States, 77030
Canada, Quebec
Montreal Children Hospital, McGill University Health Centre Recruiting
Montréal, Quebec, Canada, H4A3J1
Netherlands
University Medical Center Groningen Recruiting
Groningen, Netherlands, 9700RB
Spain
Complejo Hospitalario Universitario de Santiago Recruiting
Santiago De Compostela, A Coruna, Spain, 15706
Hospital Universitario Cruces Servicio Pediatria Recruiting
Barakaldo, Vizcaya, Spain, 48903
Hospital Universitario 12 de Octubre Recruiting
Madrid, Spain, 28041
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
Investigators
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Study Director: Medical Director Ultragenyx Pharmaceutical Inc
More Information
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Responsible Party: Ultragenyx Pharmaceutical Inc
ClinicalTrials.gov Identifier: NCT03517085     History of Changes
Other Study ID Numbers: 401GSDIA01
1706-1617 ( Other Identifier: NIH Protocol Registration Number )
First Posted: May 7, 2018    Key Record Dates
Last Update Posted: June 4, 2019
Last Verified: June 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Ultragenyx Pharmaceutical Inc:
glycogen storage disorder Ia
AAV
gene therapy
von Gierke disease
glucose metabolism disorder

Additional relevant MeSH terms:
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Glycogen Storage Disease
Glycogen Storage Disease Type I
Carbohydrate Metabolism, Inborn Errors
 Metabolism, Inborn Errors
Genetic Diseases, Inborn
Metabolic Diseases