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

Ultragenyx Pharmaceutical Inc12 enrolled

Overview

The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.

Only participants who received DTX401 in study 401GSDIA01 (NCT03517085) are eligible to participate in study 401GSDIA02. No investigational product will be administered during study 401GSDIA02. Participants will be followed in study 401GSDIA02 for at least 4 years, and up to 6 years after administration of DTX401.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Glycogen Storage Disease Type IA Von Gierke's Disease (GSD Type Ia)

Interventions

No interventionOTHER

No intervention

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Received DTX401 in study 401GSDIA01. 2. Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed. 3. Willing and able to comply with all scheduled study visits, procedures, and requirements. Exclusion Criteria: 1. Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study. 2. Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results.

Locations (6)

UCONN Health

Farmington, Connecticut, United States

Michigan Medicine University of Michigan

Ann Arbor, Michigan, United States

University of Texas Health Science Center at Houston

Houston, Texas, United States

Montreal Children Hospital, McGill University Health Centre

Montreal, Quebec, Canada

Outcomes

Primary Outcomes

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

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)

Data from ClinicalTrials.gov

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