Contact SCGE




Gene Therapy Trial Report

Summary

A Study of Adeno-Associated Virus Serotype 8-Mediated Gene Transfer of Glucose-6-Phosphatase in Patients With Glycogen Storage Disease Type Ia (GSDIa)


NCTID NCT05139316 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Glycogen Storage Disease Type Ia
Disease Ontology Term DOID:2749
Compound Name DTX401
Compound Alias Pariglasgene brecaparvovec
Sponsor Ultragenyx Pharmaceutical Inc
Funder Type Industry
Recruitment Status 🔄
Completed
Enrollment Count 49 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant G6PC
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 Phase 1/2: 2E12 GC/kg
Dose 2 Phase 1/2: 6E12 GC/kg
Dose 3 Phase 3: 1.0E13 GC/kg

Study Record Dates


Current Stage Phase3
Submit Date 2021-07-14
Completion Date 🔄 2026-02-20
Last Update 🔄 2026-03-25

Participation Criteria


Eligible Age >=8 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Key Inclusion Criteria: * Documented GSDIa with confirmation by molecular testing or enzymatic activity on liver biopsy * Currently receiving a therapeutic regimen of cornstarch (or equivalent), following international guidance/recommendations with stable nutrition, glycemic, and clinical status. * Willing and able to complete the informed consent process and to comply with study procedures and visit schedule * Females of childbearing potential and fertile males must consent to use highly effective contraception from the period following informed consent through the duration of the 144-week study and in cases of early withdrawal at least 48 weeks after the last dose of investigational product (IP). Female subjects must agree not to become pregnant. Male subjects must agree not to father a child or donate sperm Key Exclusion Criteria: * Detectable pre-existing antibodies to the AAV8 capsid * History of liver transplant, including hepatocyte cell therapy/ transplant * History of liver disease * 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 * Non-fasting triglycerides ≥1000 mg/dL * Pregnant, breastfeeding, or planning to become pregnant (self or partner) at any time during the study. * Current or previous participation in another gene transfer study * History of illicit drug use within 60 days prior to screening or positive results from an 8-panel urine drug screen during the Screening Period completed at 2 time points at least 4 weeks apart Note: additional inclusion/exclusion criteria may apply, per protocol
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 20
Locations Canada,Netherlands,United States,Japan,Brazil,Denmark,Italy,Germany,Spain

Regulatory Information


Has US IND True
FDA Designations 🔄 Fast Track, Orphan Drug Designation, Priority Review, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates BLA Submission completed 12/30/25, PDUFA Action Date set for 8/23/26

Resources/Links