Mesenchymal stromal cell therapy for children with recessive dystrophic epidermolysis bullosa
Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genetic condition that makes skin extremely fragile. Even gentle friction can lead to painful blisters and long-lasting wounds. At present, there is no cure, and treatment focuses on wound care and pain relief. This UK study looks at whether a cell therapy called CORDStrom can help. The treatment uses special donor cells, known as mesenchymal stromal cells, given through a drip into a vein. Doctors hope these cells will calm inflammation, help skin heal faster, and soothe chronic itching and pain. The trial is open to children aged 6 months to 16 years. Over 15 months, every child will receive both the real cell treatment and a harmless dummy drip at different times. Specialist teams at children's hospitals in London and Birmingham will closely monitor each child's health and comfort throughout.
במבט חטוף
על מה המחקר הזה?
Recessive dystrophic epidermolysis bullosa (RDEB) is a severe, lifelong condition caused by a missing protein that normally holds skin layers together. Without it, the skin tears and blisters very easily. Everyday activities can cause painful sores that take a long time to heal, leading to constant itching, scarring, and distress for children and their families.
This trial is testing an experimental treatment called CORDStrom. It uses mesenchymal stromal cells, which are healthy cells taken from donated umbilical cord tissue. Earlier research suggests these cells can send helpful signals to the body to calm inflammation, soothe pain and itch, and encourage damaged skin to repair itself more effectively.
Because there is currently no cure for RDEB, discovering treatments that provide meaningful relief is vital. This study is designed to check both the safety and effectiveness of CORDStrom. Researchers also want to find out whether the therapy improves daily quality of life, such as helping children sleep better, and whether it represents good value for the NHS.
Care is provided by expert teams at Great Ormond Street Hospital in London and Birmingham Children's Hospital. By comparing the cell therapy against an inactive dummy treatment, researchers hope to gather the strong evidence needed to guide future NHS care for children with RDEB.
נקודות עיקריות
- Tests a stem cell drip (CORDStrom) for children aged 6 months to 16 years with RDEB.
- Aims to reduce pain, soothe itching, and help fragile skin heal faster.
- Every child in the study receives the real treatment at some stage.
- Takes place at specialist children's hospitals in London and Birmingham.
- Participation lasts 15 months, with an optional 12-month extension.
מי עשוי להיות זכאי?
This study is open to children aged between 6 months and 16 years who have a confirmed diagnosis of recessive dystrophic epidermolysis bullosa (generalised severe or generalised intermediate types). A parent or legal guardian must give written permission, and older children will be asked for their agreement as well.
Children cannot take part if they have a different form of epidermolysis bullosa, such as EB simplex or junctional EB. They will also be unable to join if they have had an active infection needing antibiotics within the last week, have taken high-dose oral or topical steroids for more than a week recently, or have had other stem cell treatments in the past 9 months.
Doctors will run routine screening checks, including blood tests, to make sure the trial is safe for each child. Families should speak to their hospital specialist to check if their child meets all the medical requirements.
האם מחקר זה יכול להתאים לך?
ענה על השאלות המהירות הללו כדי לבדוק אם אתה עשוי להיות זכאי. זהו מדריך בלבד — צוות המחקר מקבל את ההחלטה הסופית.
- My child is aged between 6 months and 16 years.
- My child has a confirmed diagnosis of recessive dystrophic epidermolysis bullosa (RDEB).
- My child has not had stem cell therapy within the last 9 months.
- My child does not currently have an active infection needing antibiotics.
- We can travel to London or Birmingham for hospital appointments.
מה כרוך בהשתתפות?
Taking part in the trial lasts 15 months. It uses a 'crossover' design, meaning each child is placed by chance into one of two groups. One group receives the cell therapy drip first and the dummy drip second. The other group receives them in reverse order. Neither you nor the doctors will know which drip is given first, but every child is guaranteed to receive the real treatment during the study.
The treatments are given as a slow liquid drip into a vein during hospital visits at Great Ormond Street Hospital or Birmingham Children's Hospital. Before each drip, children receive an antihistamine medicine to prevent allergic reactions. Throughout the 15 months, doctors will check wound healing, take small blood samples, and ask questions about pain, itchiness, and sleep quality. After the main study ends, families may have the choice to continue receiving the cell therapy for another 12 months.
סיכונים ותועלות פוטנציאליים
מיקומים (2)
- Great Ormond Street HospitalמשוערLondon, England
- Birmingham Children's HospitalמשוערBirmingham, England
שאלות נפוצות
Will my child definitely receive the active cell therapy?
Yes. Because this is a crossover study, every child receives both the active cell drip and the dummy drip at different points during the 15 months.
How is the treatment given?
It is given as a liquid through a small tube into a vein (an intravenous drip) in a specialist hospital unit.
Where does the trial take place?
Visits take place at either Great Ormond Street Hospital in London or Birmingham Children's Hospital.
What are the most common side effects?
Most children tolerate the drip well, but mild side effects can include a slight temperature, headache, mild tummy ache, or a temporary garlic taste or smell.
Can we stop taking part if we change our minds?
Yes. You can withdraw your child from the trial at any point, and their standard NHS treatment will continue as normal.
כיצד לגלות עוד
Anna Martinez
תמיד התייעץ עם רופא המשפחה או המומחה שלך לפני שתחליט להשתתף במחקר.
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