Efficacy and safety of allogenic cultured Adipose-derived mesenchymal stromal cell injections on MoUth fibrosis and handicap in patients with Systemic sclEroderma
This study is investigating a new approach to help people with Systemic Sclerosis who experience mouth stiffness and difficulty with daily activities like eating and speaking. The treatment involves injecting special cells, called adipose-derived mesenchymal stromal cells, into the mouth. Researchers want to find out if these injections can reduce the 'handicap' or impact of mouth problems, making it easier for people to go about their daily lives. They will closely monitor how patients feel, measuring improvements in mouth movement and overall satisfaction. Crucially, they will also be carefully checking for any side effects or safety concerns. This is a Phase II study, meaning it's an early-stage trial focusing on both the effectiveness and safety of this potential new treatment.
At a glance
What is this study about?
This study is exploring a new way to help people who have a condition called Systemic Sclerosis, sometimes known as scleroderma. One of the challenges for people with this condition can be that the skin around their mouth becomes tight and stiff. This can make it difficult to open their mouth wide, eat, speak, or even smile comfortably. This study is testing if injecting special cells, called 'adipose-derived mesenchymal stromal cells,' can help make the mouth less stiff and improve how well people can use their mouth in their daily lives.
The main goal of the study is to see if these injections can make a real difference in reducing the mouth problems. To measure this, researchers will use a special questionnaire called the 'Mouth Handicap in Systemic Sclerosis scale' (MHISS). They want to see if patients' scores on this scale improve by at least 5 points after 12 weeks, as this would suggest a meaningful improvement for patients. Beyond that, they will also be very carefully watching to make sure the treatment is safe and doesn't cause any serious side effects.
The researchers will also look at many other aspects, such as how wide people can open their mouths, their satisfaction with the treatment, changes in their oral hygiene, and even how their posture or overall quality of life might be affected. This comprehensive approach helps them get a full picture of how the treatment might work and its potential benefits for people living with Systemic Sclerosis.
Key takeaways
- The study explores a cell-based injection for mouth stiffness in Systemic Sclerosis.
- It aims to improve mouth mobility and daily activities.
- Safety is a key focus, with careful monitoring for side effects.
- You will have regular check-ups and questionnaires during the study.
- It's a Phase II study, meaning it's an early look at effectiveness and safety.
Who may be eligible?
To be considered for this study, you need to be an adult, aged 18 years or older. The study is open to both men and women.
More specific details about your health condition, medications you might be taking, or other medical history will be discussed with the study team to decide if the treatment is suitable and safe for you. This will ensure that the study is right for your individual circumstances.
It's important to remember that not everyone who meets the initial age and sex criteria will be able to join the study. The study team will review each person carefully to make sure they fit all the study's requirements.
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 diagnosis of Systemic Sclerosis?
- Are you able to attend regular study appointments?
- Are you comfortable filling out questionnaires about your health?
What does participation involve?
If you decide to take part in this study, you will have several visits to the study centre, which will involve various assessments. Researchers will measure things like how wide you can open your mouth and ask you to fill out questionnaires about how your mouth problems affect your daily life and your overall satisfaction. You will receive either the study treatment (the cell injections) or a comparison treatment (a simple salt solution, like the solutions used in drips).
The study will last for at least 24 weeks (about six months) from when you start. Throughout this time, your health will be carefully monitored for any changes or side effects. Some specific assessments, like certain scans or detailed mouth checks, might only be available at particular study sites, such as the one in Toulouse. You will be told exactly what tests and visits are involved at the specific site you attend.
Potential risks and benefits
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Common questions
What is Systemic Sclerosis?
Systemic Sclerosis is a long-term condition that causes the immune system to attack healthy tissues, leading to a build-up of scar tissue, often affecting the skin and internal organs.
What are 'adipose-derived mesenchymal stromal cells'?
These are special cells collected from fatty tissue. Researchers are investigating if they can help repair damaged tissues and reduce inflammation.
What does a 'Phase II' study mean?
A Phase II study is an early-stage trial that looks at whether a new treatment works and if it's safe. It often involves a smaller group of people than later-stage studies.
Will I know if I'm getting the actual treatment or the salt solution?
Often in studies like this, participants don't know whether they are receiving the active treatment or a comparison treatment. This helps ensure the results are accurate.
How long will I be monitored after the injections?
You will be carefully monitored for at least 24 weeks (about six months) after you begin the study to track any changes or side effects.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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