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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

ConditionGlycogen Storage Disease Type Ia
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2+
SponsorUltragenyx Pharmaceutical Inc

About this trial

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or

Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2)

Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures.

Disqualifiers

Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

Trial population

All participants who received DTX401 in a qualifying clinical study will be expected and asked to participate in the DMP upon completion or early termination (ET) from the parent study, as required by regulatory agencies for all gene therapy studies.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • No Intervention

    Other intervention

    No investigational/study product will be administered in this DMP.

Treatment groups

140 Participants
are divided into 2 treatment groups
Group A: Group 1: DTX401 in Prior Clinical Study1 intervention
Group B: Group 2: DTX401 in Post-Marketing Setting1 intervention

Trial outcomes

Primary outcomes

1

Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator

Time frame
10 Years
2

Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies

Time frame
10 Years
3

Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner

Time frame
10 Years
4

Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner

Time frame
10 Years

Secondary outcomes

1

Percent Change Over Time from Baseline in Total Cornstarch Intake

Time frame
Baseline, Up to 10 Years
2

Change from Baseline Over Time in Frequency of Cornstarch Intake

Time frame
Baseline, Up to 10 Years
3

Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch

Time frame
Baseline, Up to 10 Years
4

Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery

Time frame
Baseline, Up to 10 Years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Ultragenyx Pharmaceutical Inc

Lead sponsor

Source ClinicalTrials.gov
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