Open-Label Extension Study to Assess GLM101 in PMM2-CDG Patients
This is an ongoing study for individuals in the UK with a rare genetic condition called PMM2-CDG. It provides continued access to a medicine named GLM101 for those who have already received it in a previous clinical trial. The main goals are to understand the long-term safety of GLM101 and to observe its effects over an extended period. Participants will receive weekly infusions of GLM101 at the same dose they previously received. Researchers will also look at whether the medicine helps with symptoms like problems with movement and balance, and how it impacts their overall quality of life through regular check-ups and questionnaires. This helps gather more information on this important potential treatment.
At a glance
What is this study about?
This research study is for people in the UK who have a rare genetic condition called PMM2-CDG. In this condition, the body has trouble making a crucial sugar and protein, which can affect many body parts, especially the brain, muscles, and nervous system.
The study offers continued access to a medicine called GLM101. It's for those who have previously taken GLM101 in another clinical trial. The main reasons for this study are to help patients continue receiving a treatment that might be beneficial, and to learn more about how GLM101 works over a longer period. This includes carefully checking its safety and whether it helps improve symptoms or overall well-being.
By continuing this research, doctors hope to gather valuable information about GLM101's long-term effects. This will help them understand if it can make a real difference in the lives of people with PMM2-CDG and ultimately work towards making it more widely available if it proves safe and effective.
Key takeaways
- This study offers continued treatment with GLM101 for PMM2-CDG patients who have taken it before.
- The main goals are to check long-term safety and how well GLM101 works over time.
- Participants will receive GLM101 weekly as an infusion.
- Regular check-ups and questionnaires will assess effects on movement and quality of life.
- The study includes individuals aged 2 and older with a confirmed PMM2-CDG diagnosis.
Who may be eligible?
To join this study, participants must be able to understand the study information and agree to take part, either by themselves or with help from a legal guardian. A key requirement is that they have already taken part in a previous study using GLM101 and successfully completed its treatment period.
Participants need to be at least 2 years old when they agree to join. They must also have a confirmed diagnosis of PMM2-CDG, which means doctors have identified the specific genetic changes causing the condition. This diagnosis can be from current tests or from previous medical records.
For female participants who could become pregnant, special precautions are needed to prevent pregnancy during the study and for a short time afterwards. This ensures safety for both the participant and any potential future pregnancy. There are no restrictions based on gender for participation.
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.
- Have you previously taken part in a clinical study for GLM101?
- Are you at least 2 years old?
- Do you have a confirmed diagnosis of PMM2-CDG?
- Are you able to agree to take part, either yourself or with a legal guardian's help?
- If you are a female and could become pregnant, are you willing to use an effective method of birth control during the study?
What does participation involve?
If you join this study, you will continue to receive GLM101 as a weekly infusion, just as you did in the previous trial. The dose will generally be the same, around 30 mg/kg, but doctors might adjust it if they believe a change would be safer or more helpful based on new information. This means you would visit a clinic regularly for these infusions.
Throughout the study, you'll have check-ups so the research team can monitor your health and any effects of the medicine. You will also be asked to complete questionnaires. These questions are designed to help researchers understand if there are any changes in your movement and balance (ataxia) and how the treatment might be affecting your overall quality of life. The study aims to follow participants for an extended period, providing continued care while gathering important long-term data.
Potential risks and benefits
Locations (2)
- Hospital Sant Joan de DéuVerified postcodeBarcelona, Spain
- Great Ormond Street HospitalVerified postcodeLondon, United Kingdom
Common questions
What is PMM2-CDG?
PMM2-CDG is a rare genetic condition where the body has trouble making a specific type of sugar molecule, which affects many body functions, especially the brain and nervous system.
What is GLM101?
GLM101 is the name of the medicine being studied for its potential to help people with PMM2-CDG.
Who can join this study?
This study is for people with PMM2-CDG who are at least 2 years old and have already received GLM101 in a previous clinical trial.
What will I have to do if I take part?
You'll receive weekly infusions of GLM101, have regular health check-ups, and fill out questionnaires about your symptoms and quality of life.
How long does the study last?
This is an open-label study, meaning it aims to provide continued access to GLM101, gathering long-term information on its safety and effects.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Interested in taking part?
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