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

A groundbreaking clinical trial is underway to evaluate the safety and feasibility of using autologous tissue-engineered nanostructured fibrin-agarose oral mucosa substitute for treating patients with cleft palate. This Phase I-IIa study focuses on a novel approach using the patient’s own oral mucosa-derived adult keratinocytes and fibroblasts, which are expanded in a laboratory setting. The trial aims to assess the potential benefits of this advanced therapy in improving outcomes for children with cleft lip and palate.

Table of Contents

What is BIOCLEFT?

BIOCLEFT is a new medical treatment being developed for patients with cleft palate. It is considered an advanced therapy product, which means it uses innovative biological methods to treat diseases. BIOCLEFT is made from a patient’s own cells, specifically from their oral mucosa (the lining inside the mouth)[1].

The full name of this treatment is “Autologous oral mucosa differentiated adult keratinocytes and fibroblasts expanded in biological fibrin-agarose matrix.” Let’s break this down:

  • Autologous: This means the cells come from the patient’s own body.
  • Oral mucosa: This is the moist tissue that lines the inside of the mouth.
  • Keratinocytes and fibroblasts: These are two types of cells found in the oral mucosa. Keratinocytes form the outer layer of the mucosa, while fibroblasts help in tissue repair and wound healing.
  • Expanded: The cells are grown in a laboratory to increase their numbers.
  • Fibrin-agarose matrix: This is a special gel-like substance that holds the cells together, creating a living tissue equivalent.

How Does BIOCLEFT Work?

BIOCLEFT works by using the patient’s own cells to create a tissue substitute that can be used to repair the cleft palate. Here’s a simple breakdown of the process[1]:

  1. A small sample of oral mucosa is taken from the patient during an earlier surgery (usually during cleft lip repair).
  2. The sample is sent to a laboratory where the keratinocytes and fibroblasts are isolated and grown to increase their numbers.
  3. These cells are then combined with a special fibrin-agarose gel to create a living tissue equivalent.
  4. During cleft palate repair surgery, this tissue equivalent (BIOCLEFT) is implanted to help close the gap in the palate.

This approach is hoped to provide a more natural and effective repair of the cleft palate compared to traditional surgical methods.

What Conditions Does BIOCLEFT Treat?

BIOCLEFT is specifically being developed to treat cleft lip and palate[1]. This is a birth defect where there is an opening in the upper lip and the roof of the mouth (palate). It occurs when facial structures don’t close completely during fetal development. Cleft lip and palate can cause problems with feeding, speech, hearing, and dental development if not treated properly.

Clinical Trial Details

BIOCLEFT is currently being studied in a clinical trial. This trial is described as a “Phase I-IIa, randomized, controlled, open-label, single-center clinical trial”[1]. Here’s what this means:

  • Phase I-IIa: This is an early stage of testing in humans, focusing on safety and initial signs of effectiveness.
  • Randomized: Participants are randomly assigned to either receive BIOCLEFT or standard treatment.
  • Controlled: The study compares BIOCLEFT to a control group receiving standard treatment.
  • Open-label: Both the researchers and participants know which treatment is being given.
  • Single-center: The study is being conducted at one medical facility.

Who Can Participate in the Trial?

The trial has specific criteria for who can participate[1]:

  • Pediatric patients (children) of either sex
  • Diagnosed with non-syndromic total unilateral cleft lip and palate (FLPNS)
  • Children who have previously donated an oral mucosa sample during their cleft lip repair surgery
  • Parents or legal guardians must provide informed consent

There are also several conditions that would prevent a child from participating, such as active infections, allergies to components of BIOCLEFT, certain medical conditions, or other factors that the study doctor thinks might affect the results.

What Are the Goals of the Trial?

The main objectives of this clinical trial are[1]:

  1. To evaluate the safety of using BIOCLEFT in cleft palate treatment
  2. To assess the feasibility of the BIOCLEFT procedure
  3. To gather initial evidence of how well BIOCLEFT works

The researchers will be looking at several factors to determine if BIOCLEFT is successful, including:

  • How well the palate heals and regenerates
  • The aesthetic appearance after treatment
  • Effects on craniofacial (skull and face) growth
  • Impacts on hearing
  • Effects on speech
  • Overall quality of life for the patients

Safety and Effectiveness

As this is an early-stage clinical trial, the safety and effectiveness of BIOCLEFT are still being evaluated. The researchers will be closely monitoring all participants for any side effects or adverse events[1].

The trial will be considered feasible if the BIOCLEFT implantation can be successfully carried out in at least 3 out of 5 patients in the initial phase. The safety will be assessed by monitoring for any adverse events, whether serious or non-serious, especially those related to the treatment.

It’s important to note that as an experimental treatment, BIOCLEFT’s full benefits and risks are not yet known. The results of this clinical trial will help determine if BIOCLEFT could become a new treatment option for children with cleft palate in the future.

Aspect Details
Trial Type Phase I-IIa, randomized, controlled, open-label, single-center clinical trial
Condition Studied Cleft lip and palate
Investigational Product BIOCLEFT: Autologous tissue-engineered nanostructured fibrin-agarose oral mucosa substitute
Main Objective Evaluate safety and feasibility of using BIOCLEFT in cleft palate treatment
Secondary Objectives Assess regeneration, healing, aesthetics, craniofacial growth, hearing, speech, and quality of life
Key Inclusion Criteria Pediatric patients with non-syndromic total unilateral cleft lip and palate
Primary Endpoints Feasibility of surgical implantation, safety assessment, indicators of efficacy
Therapeutic Area Congenital, Hereditary, and Neonatal Diseases and Abnormalities

FAQ

  • What is the main purpose of this clinical trial? The main purpose of this clinical trial is to evaluate the safety and feasibility of using an autologous tissue-engineered nanostructured fibrin-agarose human palatal mucosa substitute in the treatment of cleft palate. It also aims to assess the potential benefits of this advanced therapy in improving outcomes for children with cleft lip and palate.
  • 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). Informed consent from parents or legal guardians is required.
  • What is BIOCLEFT? BIOCLEFT is the name given to the investigational product used in this trial. It is an autologous tissue-engineered nanostructured fibrin-agarose oral mucosa substitute made from the patient's own oral mucosa-derived adult keratinocytes and fibroblasts, which are expanded in a laboratory setting.
  • What are the main outcomes the researchers are looking for? The researchers are primarily assessing the feasibility and safety of the BIOCLEFT implantation. They will also evaluate indicators of efficacy, including regeneration and healing, aesthetic appearance, craniofacial growth, hearing, speech, and quality of life. The occurrence of any adverse events, both serious and non-serious, will be closely monitored.
  • How is this treatment different from traditional cleft palate treatments? This treatment uses advanced therapy techniques by creating a personalized tissue substitute from the patient's own cells. Traditional treatments typically involve surgical repair using existing tissue. The BIOCLEFT approach aims to potentially improve healing, aesthetics, and overall outcomes by providing a more natural and compatible tissue substitute for the repair.

Ongoing Clinical Trials on Autologous Oral Mucosa-Derived Adult Keratinocytes, 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 same individual. In this context, it refers to using the patient's own cells to create the treatment.
  • Keratinocytes: The predominant cell type in the outer layer of the skin (epidermis) and mucous membranes, responsible for forming a protective barrier.
  • Fibroblasts: Cells that produce collagen and other proteins important for maintaining the structure and function of tissues.
  • Ex-vivo expanded: Cells that have been grown and multiplied outside the body in a laboratory setting.
  • Cleft lip and palate: A birth defect where there is an opening in the upper lip and/or the roof of the mouth (palate) due to incomplete development during pregnancy.
  • Fibrin-agarose matrix: A biocompatible scaffold made from fibrin (a protein involved in blood clotting) and agarose (a polysaccharide polymer) used to support cell growth and tissue formation.
  • Nanostructured: Having features or structures at the nanoscale level (extremely small), which can enhance certain properties of the material.
  • Phase I-IIa clinical trial: An early stage of clinical research that combines elements of both Phase I (initial safety testing) and Phase II (preliminary efficacy testing) studies.
  • Cheiloplasty: A surgical procedure to repair a cleft lip.
  • BIOCLEFT: The name given to the investigational product in this trial, which is an autologous tissue-engineered oral mucosa substitute.

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/