Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

Browse trials for

• Glucose Metabolism Disorders 256
• Genetic Diseases, Inborn 401

Keywords

• AAV
• gene therapy
• glucose metabolism disorder
• carbohydrate metabolism, inborn errors
• genetic diseases
• Inborn Metabolic Diseases

Study population

Groups and cohorts

• DTX401 Cohort 1

  Participants received a single intravenous (IV) dose of 2.0 × 10\^12 GC/kg DTX401(pariglasgene brecaparvovec) with a reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.

  Other: No intervention

• DTX401 Cohort 2

  Participants received a single IV dose of 6.0 × 10\^12 GC/kg DTX401(pariglasgene brecaparvovec) with a reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.

  Other: No intervention

• DTX401 Cohort 3

  Participants received a single IV dose of 6.0 × 10\^12 GC/kg DTX401(pariglasgene brecaparvovec) with an optimized reactive steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.

  Other: No intervention

• DTX401 Cohort 4

  Participants received a single IV dose of 6.0 × 10\^12 GC/kg DTX401(pariglasgene brecaparvovec) with a prophylactic steroid regimen during their participation in study 401GSDIA01 (NCT03517085). No intervention was provided during the 401GSDIA02 long term follow up study.

  Other: No intervention

Interventions

• OtherNo intervention

  No intervention

Primary outcomes

1. Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs

   An adverse event (AE) is defined as any untoward medical occurrence, regardless of its causal relationship to study product. A TEAE is defined as any AE not present prior to the initiation of the drug treatment or any AE already present that worsens in either intensity or frequency following exposure to the drug treatment. A serious TEAE is an AE that meets any of the following criteria in the view of either the Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; disability/Incapacity; congenital anomaly/birth defect not present at screening; other important medical events. Severity of events were graded as mild (grade 1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5). As DTX401 was administered as part of Study 401GSDIA01, all AEs in Study 401GSDIA02 were considered TEAEs.

   Time frame: From Baseline (Week 52 of 401GSDIA01/Visit 1 of 401GSDIA02) Up to Week 329

Secondary outcomes

1. Change From Baseline in Time to First Hypoglycemic Event During a Controlled Fasting Challenge Over Time

   The change from baseline in time (in hours) to first hypoglycemic event (defined as glucose \< 54 mg/dL \[\< 3.0 mmol/L\]) during a controlled fasting challenge at the participant's last visit. A positive change from baseline is favorable. Change from baseline is calculated from Baseline (Week 0) of the 401GSDIA01 study.

   Time frame: Baseline (Week 0 of 401GSDIA01), Weeks 52 (Visit 1 of 401GSDIA02), 78, 104, 130, 156, 182, 208, 234, 260, Last Visit (Up to Week 329)

Baseline characteristics

Related links

• Ultragenyx Transparency Commitment ↗

Study documents

• Study protocol · Apr 3, 2023
• Statistical analysis plan · Aug 26, 2024

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No — Due to the rarity of GSDIa and the small number of subjects in this trial, individual patient data will not be shared in order to safeguard patient privacy, consistent with the data sharing commitment statement listed on Ultragenyx.com.