Prospective pilot trial to address the feasibility and safety of treatment with oral Zinc in GNAO1 associated disorders (ZINCGNAO1)
This research study, called ZINCGNAO1, is exploring a new way to help people with GNAO1 associated disorders. It's a 'Phase II' trial, meaning doctors are testing if a treatment is safe and practical to use, and if it shows any promising signs of helping. Participants will take zinc capsules (either 25mg or 50mg) each day. The main goal is to see if taking the zinc regularly is 'doable' for people and if it causes any harmful side effects. Doctors will also keep a close eye on things like motor skills, quality of life, and any changes in symptoms like dystonia or seizures. This is an early study, but the findings could help decide if more research into zinc for GNAO1 disorders is worthwhile.
At a glance
What is this study about?
This study is looking into a condition called GNAO1 associated disorders. People with this condition can experience various challenges, often including problems with movement. Doctors are investigating whether a common mineral, zinc, given in capsule form, might be a helpful treatment. This isn't a final answer yet, but an exploratory step to see if it's worth more detailed research.
The main things the researchers want to find out are two-fold: firstly, how practical it is for people with GNAO1 disorders to take the zinc capsules every day as prescribed. They'll be looking to see if at least 80% of the planned doses are taken. Secondly, and very importantly, they want to make sure the treatment is safe. They'll be carefully monitoring for any unwanted side effects that might occur while taking the zinc.
Beyond safety and practicality, the study will also gently explore other areas. This includes looking at how zinc might affect movement (like how well someone can use their muscles), general well-being and quality of life, and specific symptoms such as involuntary movements (dystonia) or seizures. Parents will also keep daily diaries to record things like alertness, sleep, and seizure activity. They'll also check zinc levels in the blood to make sure the treatment is reaching the body, and other minerals to ensure balance. This careful approach helps the research team gather lots of information to guide future studies.
Key takeaways
- This study evaluates oral zinc for GNAO1 associated disorders.
- The main goals are to check how practical and safe zinc treatment is.
- It's a 'Phase II' study, an early step in researching new treatments.
- Patients must be 18 or older and have a GNAO1 disorder.
- Assessments include movement, quality of life, and blood tests.
- Parents/caregivers will record daily observations.
Who may be eligible?
This study is open to both men and women. To take part, you need to be at least 18 years old. There isn't an upper age limit mentioned, so adults of varied ages could potentially be included.
Having a diagnosis of a GNAO1 associated disorder is a key requirement for joining this study. If you or someone you care for has this specific condition and meets the age requirement, you might be able to take part.
Before enrolling, the study team will review your specific health situation to make sure the study is a good fit and safe for you. This will involve checking other health conditions or medications you might be taking.
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?
- Do you have a GNAO1 associated disorder?
- Are you able to take medication by mouth?
- Are you willing to attend clinic visits and complete diaries?
- Are you able to understand and provide informed consent?
What does participation involve?
If you decide to take part in this study, you'll be given zinc capsules (either 25mg or 50mg) to take regularly. During the study, you'll have specific clinic visits where doctors and nurses will check on your health. These visits will be at the start of the study, at a middle point (Visit 2), and again at the end (Visit 3).
At these visits, various assessments will take place. This includes checking motor skills using a special scale (GMFM-66), and assessing your quality of life through questionnaires. If you're a caregiver, you'll also complete a questionnaire about the person in your care. Your movement will be assessed using another scale (BFMDRS and AIMS). Blood samples will be taken at these visits to check zinc levels and other minerals. Stool samples might also be collected to look at gut health.
Parents or caregivers will play an important role by keeping daily diaries. In these diaries, you'll record details about general behaviour, alertness, sleep patterns, and any seizures. The exact total duration of the study isn't specified, but it involves several visits and daily logging over a period.
Potential risks and benefits
Locations (1)
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Common questions
What is a 'Phase II' trial?
It's an early stage of research to see if a new treatment is safe, practical to use, and shows any hopeful signs of helping a condition.
What are GNAO1 associated disorders?
These are conditions linked to a specific gene called GNAO1, often affecting movement and development.
What does taking zinc involve?
Participants will take oral zinc capsules (either 25mg or 50mg) as prescribed during the study period.
Why will parents keep a logbook?
The logbook helps researchers understand daily changes in behaviour, sleep, alertness, and any seizures firsthand from those providing care.
Will I know if the zinc is helping?
The study will gather information on movement, quality of life, and other symptoms to look for potential changes, but this is an exploratory study, and individual results may vary.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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