RecruitingNot Applicable

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

United States, Brazil, Canada140 participantsStarted 2024-11-04

Plain-language summary

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.

Who can participate

Age range

2 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Patient who had: * 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. Exclusion Criteria: * Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

Timeframe: 10 Years

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

Timeframe: 10 Years

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

Timeframe: 10 Years

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

Timeframe: 10 Years

Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)

Timeframe: 1 Year

Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)

Timeframe: 1 Year

Trial details

NCT IDNCT06636383

SponsorUltragenyx Pharmaceutical Inc

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2036-12

Contact for this trial

Patients Contact: Trial Recruitment

1-888-756-8657 trialrecruitment@ultragenyx.com

View on ClinicalTrials.gov More Glycogen Storage Disease Type Ia trials

Who can participate

Age range

2 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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

Timeframe: 10 Years

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

Timeframe: 10 Years

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

Timeframe: 10 Years

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

Timeframe: 10 Years

Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)

Timeframe: 1 Year

Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)

Timeframe: 1 Year