A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adenoassociated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia.
This research is a major study (Phase 3) for adults living with Glycogen Storage Disease Type Ia (GSDIa). GSDIa is a rare genetic condition where the body can't properly process sugar, leading to dangerously low blood sugar levels. People with GSDIa often need to eat cornstarch regularly to keep their blood sugar stable. This study is testing a new gene therapy, called Pariglasgene brecaparvovec, to see if it can help reduce how much cornstarch is needed every day. Some participants will receive the new treatment, while others will get a 'dummy' treatment (placebo), alongside a steroid to prevent reactions. Researchers want to compare the two groups to understand if the new gene therapy is effective and safe.
At a glance
What is this study about?
This study is a Phase 3 clinical trial, which means it's one of the final steps in checking if a new treatment works and is safe before it can become widely available. The condition it's focusing on is Glycogen Storage Disease Type Ia (GSDIa). GSDIa is a rare inherited condition where the body has trouble breaking down stored sugar (glycogen) in the liver. This can lead to very low blood sugar levels, especially between meals. To manage this, people with GSDIa often need to eat uncooked cornstarch regularly, even through the night, to provide a slow release of sugar and keep their blood sugar stable. This day-to-day management can be very challenging and can significantly impact quality of life.
The main goal of this study is to see if a new gene therapy, called Pariglasgene brecaparvovec, can help people with GSDIa reduce the amount of cornstarch they need to take each day. Gene therapy aims to correct the underlying problem by delivering a working copy of the gene that is faulty in GSDIa. Researchers will compare how much cornstarch people take after 48 weeks (about a year) between those who received the new gene therapy and those who received a placebo (a treatment that looks the same but has no active medicine).
Beyond just cornstarch intake, the study will also look at other important things. This includes how often people have dangerously low blood sugar, how stable their blood sugar levels are, and how participants feel their condition is affecting them. Safety is very important, so the study will also carefully track any side effects or problems that might happen during the trial.
Key takeaways
- This is a major study (Phase 3) for a new gene therapy for adults with GSDIa.
- The main goal is to see if the new treatment can reduce the daily cornstarch needed.
- Some participants will receive the active gene therapy, others a placebo.
- The study also tracks blood sugar control, how people feel, and safety.
- You must be 18 or older to take part, and suitability will be carefully checked.
- Participation involves regular checks and medication for about a year.
Who may be eligible?
To take part in this study, you must be 18 years old or older. The study is open to both men and women.
More specific details about your current health, other medicines you might be taking, and your history with GSDIa would be checked by the study doctors to make sure the trial is right and safe for you. This often means running some tests and going over your medical records to ensure you meet all the necessary requirements.
This study aims to find out if this new treatment works for adults with GSDIa. If you or someone you know has GSDIa and meets the age requirement, and is interested in potential new treatment options, it's a good idea to discuss this trial with your doctor.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you 18 years old or older?
- Have you been diagnosed with Glycogen Storage Disease Type Ia (GSDIa)?
- Are you able to attend regular clinic visits and follow study instructions?
- Are you willing to potentially receive either the active gene therapy or a placebo?
- Are you generally in good health apart from your GSDIa, as determined by the study doctors?
What does participation involve?
If you decide to join this study, you will be assigned to one of two groups by chance (like flipping a coin). One group will receive the new gene therapy (Pariglasgene brecaparvovec), and the other group will receive a placebo, which looks like the active treatment but contains no medicine. You, your family, and your doctors involved in the study will not know which treatment you are receiving (this is called 'double-blind').
Before you receive the main study treatment, you will be given special tablets of Prednisolone to help prevent your body from having a strong reaction to the gene therapy. Regular check-ups, blood tests, and assessments of your cornstarch intake will be part of the study from the start and over the course of 48 weeks (just under a year). Your blood sugar levels will be closely monitored, and you will be asked to report how you feel your condition is affecting you. There will also be a detailed follow-up to check for any side effects.
Potential risks and benefits
Locations (5)
- —UnverifiedDenmark
- —UnverifiedNetherlands
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedGermany
Common questions
What is GSDIa?
GSDIa is a rare genetic condition where your body can't properly use sugar stored in your liver, leading to low blood sugar levels.
What is gene therapy?
Gene therapy is a new type of treatment that aims to fix the root cause of a disease by adding a working copy of a faulty gene to your body.
What does 'placebo-controlled' mean?
It means some people get the new treatment, and others get a 'dummy' treatment (placebo) that looks the same but has no active medicine. This helps researchers compare the real treatment's effects.
Why is cornstarch mentioned?
People with GSDIa often need to eat cornstarch regularly to slowly release sugar and keep their blood sugar levels stable throughout the day and night.
How long will the study last?
The main part of the study will involve monitoring your daily cornstarch intake and other health measures for 48 weeks (just under a year).
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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