In short
A groundbreaking clinical trial is underway to evaluate the safety and effectiveness of using autologous oral mucosa-derived adult fibroblasts, along with keratinocytes, in treating cleft palate. This advanced therapy product, known as BIOCLEFT, is a tissue-engineered nanostructured fibrin-agarose oral mucosa substitute that could potentially revolutionize cleft palate treatment in pediatric patients.
At a glance
- Trial Type
- Phase I-IIa, randomized, controlled, open-label, single-center clinical trial
- Treatment
- Autologous tissue-engineered nanostructured fibrin-agarose oral mucosa substitute (BIOCLEFT)
- Condition
- Cleft lip and palate
- Primary Objectives
- Evaluate safety and feasibility of BIOCLEFT in cleft palate treatment
- Secondary Objectives
- Assess regeneration, healing, aesthetic appearance, craniofacial growth, hearing, speech, and quality of life
- Inclusion Criteria
- Pediatric patients with non-syndromic total unilateral cleft lip and palate, previous oral mucosa donation
- Exclusion Criteria
- Active infections, allergies to product components, severe medical conditions, other congenital malformations
- Primary Endpoints
- Feasibility of surgical implantation, occurrence of adverse events, indications of efficacy
What is BIOCLEFT?
BIOCLEFT is an innovative medical treatment being developed for patients with cleft palate. It is a type of advanced therapy product that uses the patient's own cells to create a living tissue substitute for repairing the palate. The full name of this treatment is "Autologous oral mucosa differentiated adult keratinocytes and fibroblasts expanded in biological fibrin-agarose matrix".
How Does BIOCLEFT Work?
BIOCLEFT works by using two types of cells from the patient's own mouth:
- Keratinocytes: These are the main cells that make up the outer layer of your skin and the lining of your mouth.
- Fibroblasts: These cells help in producing connective tissue and are important for wound healing.
These cells are taken from a small sample of the patient's oral mucosa (the lining inside the mouth) during an earlier surgery to repair the cleft lip. The cells are then grown and expanded in a laboratory. They are placed into a special structure made of fibrin and agarose, which are natural materials that provide a framework for the cells to grow on.
This living tissue is then implanted into the patient's palate during surgery. The goal is for this tissue to integrate with the patient's own tissue and help close the cleft in the palate.
Medical Conditions Treated
BIOCLEFT is specifically being developed to treat cleft lip and palate. This is a condition where a baby is born with an opening in the upper lip and/or the roof of the mouth (palate). It occurs when facial structures don't close completely during early fetal development.
Clinical Trial Details
BIOCLEFT is currently being studied in a Phase I-IIa clinical trial. This means it's in the early stages of testing in humans. The main goals of this trial are to:
- Evaluate the safety of using BIOCLEFT in patients with cleft palate
- Determine if it's feasible to use this treatment
- Look for early signs that the treatment might be effective
The trial is comparing patients who receive BIOCLEFT to a control group who receive standard treatment. They will look at several factors, including:
- How well the palate heals and regenerates
- The aesthetic appearance of the repaired palate
- How the treatment affects craniofacial growth (growth of the skull and face)
- Effects on hearing
- Effects on speech
- Overall quality of life
Eligibility Criteria
Not all patients with cleft palate will be eligible for this trial. The main criteria for inclusion are:
- Pediatric patients (children) of either sex
- Diagnosis of non-syndromic total unilateral cleft lip and palate (This means the cleft affects one side of the lip and palate and is not part of a larger syndrome)
- Children who have previously donated an oral mucosa sample during their cleft lip repair surgery
- Parents or guardians must provide informed consent
There are also several conditions that would exclude a patient from participating, including active infections, certain allergies, blood disorders, and other medical conditions.
Potential Benefits
While it's important to remember that BIOCLEFT is still in the experimental stage, researchers hope it could offer several potential benefits:
- Improved healing and regeneration of the palate
- Better aesthetic results
- Positive effects on facial growth
- Improvements in hearing and speech
- Enhanced overall quality of life for patients with cleft palate
Safety Considerations
As with any new medical treatment, safety is a primary concern. The clinical trial is carefully designed to monitor for any adverse events (side effects) that might occur. Because BIOCLEFT uses the patient's own cells, the risk of rejection is lower than with treatments using donor cells or tissues.
However, it's important to note that as an experimental treatment, there may be unknown risks. The researchers will be closely monitoring all participants for any unexpected effects.