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Autologous Oral Mucosa-Derived Adult Fibroblasts, Ex-Vivo Expanded

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.

Table of Contents

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[1]. The full name of this treatment is “Autologous oral mucosa differentiated adult keratinocytes and fibroblasts expanded in biological fibrin-agarose matrix”[1].

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[1].

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[1].

Medical Conditions Treated

BIOCLEFT is specifically being developed to treat cleft lip and palate[1]. 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[1]

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[1]

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[1]

There are also several conditions that would exclude a patient from participating, including active infections, certain allergies, blood disorders, and other medical conditions[1].

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[1]

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[1].

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.

Aspect Details
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

FAQ

  • What is the main purpose of this clinical trial? The main purpose of this clinical trial is to evaluate the safety, feasibility, and potential effectiveness of using an autologous tissue-engineered nanostructured fibrin-agarose human palatal mucosa substitute (BIOCLEFT) in the treatment of cleft palate in pediatric patients.
  • Who can participate in this clinical trial? The trial is open to pediatric patients of either sex who have been diagnosed with non-syndromic total unilateral cleft lip and palate (FLPNS) and are scheduled for corrective surgery. Participants must have previously donated an oral mucosa sample during their cleft lip repair procedure (cheiloplasty).
  • What is BIOCLEFT made of? BIOCLEFT is made of autologous oral mucosa-derived adult keratinocytes and fibroblasts that have been expanded ex-vivo and combined with a biological fibrin-agarose matrix. This creates a living tissue equivalent that can be used as a substitute for human palatal mucosa.
  • What are the potential benefits of this treatment? The potential benefits of this treatment include improved regeneration and healing of the cleft palate, better aesthetic appearance, improved craniofacial growth, and potentially enhanced hearing and speech outcomes. The trial also aims to evaluate the impact on the patient's quality of life.
  • How will the safety and effectiveness of BIOCLEFT be evaluated? The safety of BIOCLEFT will be assessed by monitoring for any adverse events, both serious and non-serious, particularly those related to the treatment. Effectiveness will be evaluated by comparing various clinical parameters between the treatment group and the control group, including regeneration and healing, aesthetic appearance, craniofacial growth, hearing evaluation, speech therapy outcomes, and quality of life measures.

Ongoing Clinical Trials on Autologous Oral Mucosa-Derived Adult Fibroblasts, Ex-Vivo Expanded

  • Study on the Safety and Feasibility of Using Autologous Oral Mucosa-Derived Keratinocytes and Fibroblasts for Treating Cleft Palate Patients

    Recruiting

    1 1
    Investigated drugs:
    Spain

Glossary

  • Autologous: Derived from the patient's own body. In this context, it refers to cells taken from the patient's own oral mucosa.
  • Fibroblasts: Cells that produce collagen and other proteins important for maintaining the structure of tissues.
  • Keratinocytes: The predominant cell type in the outer layer of the skin (epidermis), which forms a barrier against environmental damage.
  • Ex-vivo expanded: Cells that have been grown and multiplied outside the body in a laboratory setting.
  • Cleft palate: A birth defect where there is an opening in the roof of the mouth due to the failure of tissues to join during fetal development.
  • Fibrin-agarose matrix: A biocompatible scaffold made from fibrin (a protein involved in blood clotting) and agarose (a polysaccharide polymer) that provides structure for tissue growth.
  • Nanostructured: Having features or structures at the nanoscale level (extremely small, typically between 1 to 100 nanometers).
  • Cheiloplasty: A surgical procedure to repair a cleft lip.
  • Non-syndromic: Not associated with other developmental abnormalities or part of a larger syndrome.
  • FLPNS: Abbreviation for 'non-syndromic total unilateral cleft lip and palate', a specific type of cleft affecting one side of the lip and palate.

References

  1. http://clinicaltrials.eu/trial/study-on-the-safety-and-feasibility-of-using-autologous-oral-mucosa-derived-keratinocytes-and-fibroblasts-for-treating-cleft-palate-patients/