Interested in participating? Learn about which studies might be right for you.
GlucoGene™ Gene Therapy Phase 3 Study
Have been diagnosed with GSDIa
Are 8 years of age or older
Are currently receiving cornstarch regularly as part of your dietary regimen
Ultragenyx is sponsoring this study to find out if DTX401 is a safe and effective treatment for adults and children with GSDIa. The goal of this study is to evaluate if DTX401 can help people with GSDIa maintain or improve blood sugar levels with less or no cornstarch and reduce the risk of hypoglycemia.
Ultragenyx recognizes that your participation in clinical research has an impact on your (and your loved ones’) time. We may provide compensation and travel support for participation in our clinical trials—the details may vary program to program. You will learn more during the consent process.
All participants will receive DTX401, either initially or after ~1 year (if still eligible).
All patients who receive DTX401 will be offered enrollment in an optional long-term follow-up study.
Duration
Approximately 2 years
Treatment
An investigational gene therapy called DTX401 is being tested for possible use in the treatment of glycogen storage disease type Ia (GSDIa).
Study visits
Infusion of study and/or placebo medicine into your arm
Medical questionnaires
Continuous glucose monitoring*
Diet and cornstarch assessment
Controlled fasting challenge**
Diet diary
Lab tests
*Study participants will not see these results
**At the hospital
Gene therapy is the introduction, removal, or change of genetic material in the cells of a person. For certain rare genetic diseases, gene therapy offers the potential for treatment that could have a significant impact on the lives of patients. Find out more details, including how gene therapies are designed to work.
GSDIa Odyssey Study
Have been diagnosed (or are the caregiver for someone) with GSDIa
Live in the United States and have received medical care in the US in the past 7 years
Did you know you and your child’s unique medical history can have a positive impact on the future care for the GSDIa community? Anonymized, real-world data from patient medical records can be used to help advance GSDIa understanding and potentially improve the quality of care.
Sign up online. It only takes a few minutes.
For more information, see the PicnicHealth study website or connect with a Patient Enrollment Liaison (PEL) team member.
This study allows participants to get their medical records collected, encrypted, and digitized for free—and they’re yours to keep, use, and share as you wish.
Participate in nonclinical research from your home, and help make a difference in GSDIa care.
Step 1
Sign up: Create a free PicnicHealth account (learn about PicnicHealth below), and sign the consent forms (it only takes a few minutes). Provide the names of your doctors, and we’ll take it from there.
Step 2
Wait for your records: Your medical records are collected and digitized into your own PicnicHealth Timeline.
GSDIa research is hard to conduct. By collecting and analyzing anonymized patient data, researchers may gain a greater understanding of GSDIa, with the potential to advance future care for patients.
Ultragenyx Pharmaceutical is working in partnership with PicnicHealth, a company that collects, digitizes, and encrypts medical records. If you sign up to participate in this study, you’re agreeing to share your anonymized medical records with researchers and organizations working with PicnicHealth on the study.
Researchers only receive records stripped of any information that could be used to identify you personally. Outside of that, no one outside of PicnicHealth will be able to see your medical record timeline unless you choose to share them.
To learn about current and future studies, you only need to fill out the form once.
*All fields are required.
By submitting this form, you confirm that a diagnosis of GSDIa has been made by a medical professional.
If you would like to know more about your privacy and the data you submit and data we collect while you are on this website, please see below or visit our privacy policy.
Our Patient Enrollment Liaison (PEL) team will contact you by email or phone within one business day to discuss your study eligibility and answer questions.
If you choose to be contacted by email, be sure to check your spam folder for an email from [email protected], if it’s not in your regular inbox.
Email our PEL team about current and future medical research opportunities.
Patient portrayal.
GSDIa is the most severe genetically inherited glycogen storage disease. It is caused by a defective gene for the enzyme glucose-6-phosphatase-α, resulting in the inability to regulate glucose. Hypoglycemia in patients with GSDIa can be life threatening, while the accumulation of glycogen in certain organs and tissues can impair their ability to function normally.
If you are thinking about joining a study, our Patient Enrollment Liaison (PEL) team is here to help. Every member of our PEL team has a robust professional background and clinical expertise and experience to help answer your questions. Your PEL team member will assist in exploring clinical and nonclinical studies and can determine if you or someone you care for may be eligible to take part. Your PEL is here to make the process as easy as possible.
To speak with a PEL about current and future medical research opportunities, please email our PEL team at [email protected].
When you voluntarily submit your personal data, Ultragenyx stores and processes this information in an electronic database consistent with our Privacy Policy. Ultragenyx will use this information to contact you to fulfill your request regarding Ultragenyx research opportunities.
Clinical and nonclinical studies provide invaluable information to treat, diagnose, or discover new breakthroughs in treating rare and ultra-rare diseases. By choosing to participate in research, you may help advance treatments, provide crucial insights, and ultimately improve the quality of life for others living with a rare or ultra-rare disease. You might also benefit from learning more about your condition, gain access to specific care, or achieve faster access to treatment. All efforts of participation, big and small, can make a difference.
Ultragenyx is leading the future of rare disease medicine. We leverage our experience, insight, and commitment to help move the rare disease community forward. Our primary goal is to provide medicines to those with limited options, and to help patients face rare disease head on with courage and confidence.
Ultragenyx is not responsible for the content or privacy policies of third-party websites outside of its control. Content from Ultragenyx Patient Advocacy (ultrarareadvocacy.com) is intended for a U.S. patient and caregiver audience. Content from the Ultragenyx corporate website (ultragenyx.com) is intended for investors and the general public within the U.S. The information provided on these Ultragenyx sites may not apply to your country. Please contact your physician to learn more about these topics. Thank you for visiting our Clinical Trial site. We hope your visit was informative and enjoyable.
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