DTX401 for von Gierke Disease
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to monitor the long-term safety and effectiveness of DTX401, a gene therapy for Glycogen Storage Disease Type Ia (GSDIa), a rare genetic disorder affecting sugar storage in the body. The study will observe patients who have already received DTX401, either during a previous clinical trial or through a prescription. Individuals treated with DTX401 and willing to participate are a good fit for this trial. The researchers aim to gather information over at least 10 years to better understand the treatment's safety and effectiveness in real-world settings. As a Phase 4 trial, this study focuses on a treatment already FDA-approved and proven effective, aiming to understand its benefits for more patients.
Do I need to stop my current medications for the DTX401 trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.
What is the safety track record for DTX401?
Research has shown that DTX401, also known as pariglasgene brecaparvovec, has been safe in previous studies. One study found that people generally tolerated the treatment well, which aligns with expectations for this type of gene therapy. Another source confirmed that the safety results from earlier trials met expected standards and matched initial studies. Participants were monitored for a year after receiving DTX401, and the treatment proved manageable. Overall, evidence suggests that DTX401 is safe to use, based on researchers' observations so far.12345
Why are researchers enthusiastic about this study treatment?
Researchers are excited about DTX401 (pariglasgene brecaparvovec) for Glycogen Storage Disease Type Ia (GSDIa) because it offers a novel gene therapy approach. Unlike the traditional management strategies, which mainly involve dietary modifications and cornstarch supplementation to manage symptoms, DTX401 targets the root cause of the disease by introducing a functional copy of the deficient gene. This innovative mechanism has the potential to provide a long-term solution rather than just symptom relief, making it a groundbreaking development in GSDIa treatment.
What is the effectiveness track record for DTX401?
This trial will evaluate DTX401, a gene therapy, in two different contexts. Group 1 includes patients who received DTX401 in a prior clinical study, while Group 2 involves patients receiving DTX401 in a post-marketing setting. Studies have shown that DTX401 can effectively help people with Glycogen Storage Disease Type Ia (GSDIa). Specifically, research indicates that after treatment with DTX401, many patients needed less daily cornstarch, which is important for managing this condition. By Week 96, most patients reported feeling better and experiencing fewer symptoms. This therapy works by providing a working gene to address the root cause of the disease. These findings suggest DTX401 could greatly improve life for those with GSDIa.13467
Who Is on the Research Team?
Medical Director
Principal Investigator
Ultragenyx Pharmaceuticals Inc.
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive DTX401 in a prior clinical study or in a post-marketing setting
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- DTX401
How Is the Trial Designed?
2
Treatment groups
Experimental Treatment
Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting
Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401
Find a Clinic Near You
Who Is Running the Clinical Trial?
Ultragenyx Pharmaceutical Inc
Lead Sponsor
Dr. Emil D. Kakkis
Ultragenyx Pharmaceutical Inc
Chief Executive Officer since 2010
MD/PhD in Biological Chemistry from UCLA
Dr. Eric Crombez
Ultragenyx Pharmaceutical Inc
Chief Medical Officer since 2023
MD from Wayne State University School of Medicine
Citations
Safety and Efficacy of DTX401, an AAV8‐Mediated Liver‐ ...
In most participants, ALT reached peak values between Weeks 6 and 12. Study maximum ALT concentration reached 323 U/L on day 46 post‐DTX401 ...
2.
ir.ultragenyx.com
ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-top-line-results-phase-3-studyRelease details
Treatment with DTX401 resulted in a statistically significant and clinically meaningful reduction in daily cornstarch intake compared with placebo at Week 48.
NCT03517085 | Safety and Dose-Finding Study of DTX401 ...
The primary objective of the study is to determine the safety of single doses of DTX401, including the incidence of dose-limiting toxicities (DLTs) at each ...
4.
ir.ultragenyx.com
ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-longer-term-data-phase-3-studyRelease details
At Week 96, 83% of the DTX401 group and 95% of the crossover group reported improvements in disease burden (+1 to +3 change) as measured by the ...
Pariglasgene Brecaparvovec – Application in Therapy and ...
Pariglasgene brecaparvovec is a gene therapy using a modified virus to deliver a functional gene to treat Glycogen Storage Disease Type Ia (GSDIa).
NCT03517085 | Safety and Dose-Finding Study of DTX401 ...
Participants enrolled in the 401GSDIA01 study will be monitored for 52 weeks following DTX401 administration. Participants in Cohorts 1, 2, ...
Health Technology Briefing April 2023
Pariglasgene brecaparvovec is in development for the treatment of patients with glycogen storage disease type 1a (GSD1a). GSD1a is an inherited ...
Unbiased Results
We believe in providing patients with all the options.
Your Data Stays Your Data
We only share your information with the clinical trials you're trying to access.
Verified Trials Only
All of our trials are run by licensed doctors, researchers, and healthcare companies.