Glycogen Storage Disease Type IA, Von Gierke's Disease (GSD Type Ia)
Conditions
Keywords
AAV, gene therapy, glucose metabolism disorder, carbohydrate metabolism, inborn errors, genetic diseases, Inborn Metabolic Diseases
Brief summary
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.
Detailed description
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.
Interventions
OTHERNo intervention
No intervention
Sponsors
Ultragenyx Pharmaceutical Inc
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs | From Baseline (Week 52 of 401GSDIA01/Visit 1 of 401GSDIA02) Up to Week 329 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Time to First Hypoglycemic Event During a Controlled Fasting Challenge Over Time | 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) | 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. |
Countries
Canada, Netherlands, Spain, United States
Contacts
STUDY_DIRECTORMedical Director
Ultragenyx Pharmaceutical Inc
Participant flow
Recruitment details
All 12 participants were dosed with DTX401 in study 401GSDIA01 (NCT03517085); after completing 52 weeks of follow-up in study 401GSDIA01, they subsequently enrolled in study 401GSDIA02 for an overall mean study duration of 276.2 weeks.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 12 Participants |
| Age, Continuous | 31.5 years STANDARD_DEVIATION 13.2 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 12 Participants |
| Region of Enrollment Canada | 1 participants |
| Region of Enrollment Netherlands | 0 participants |
| Region of Enrollment Spain | 0 participants |
| Region of Enrollment United States | 0 participants |
| Sex: Female, Male Female | 4 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk |
|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 3 | 0 / 3 | 0 / 3 | 0 / 3 |
| other Total, other adverse events | 3 / 3 | 3 / 3 | 3 / 3 | 3 / 3 |
| serious Total, serious adverse events | 2 / 3 | 1 / 3 | 2 / 3 | 2 / 3 |
Outcome results
None listed