Phase I-IIa, to Evaluate the Safety, Feasibility, and Efficacy of the Use of BIOCLEFT in the Treatment of Cleft Palate.
- Conditions
- Cleft Lip and Palate
- Interventions
- Procedure: implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT).
- Registration Number
- NCT06408337
- Lead Sponsor
- Elisa María Cubiles Montero de Espinosa
- Brief Summary
Phase I/II, controlled, open-label, randomized, single site clinical trial to assess the safety and feasibility, as well as hint efficacy evidence of a bioengineered palate mucosa substitute on nanostructured fibrin-agarose scaffolds with autologous mucosa tissue-specific cells (culture-expanded fibroblasts and keratinocytes), for tissue replacement and repair of donor area after the reconstruction of palate cleft defects (staphylorraphy), in comparison with standard care for donor mucosa.
- Detailed Description
The initial phase of the trial is non-randomized, and involves the sequential recruitment of 5 eligible subjects with a minimum safety period of 30 days between them. All of them will be implanted with the autologous human palatal mucosa substitute of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT), after reconstruction of the palate. Once the 5th treated patient has reached 1.5 months of follow-up after the intervention (Visit 8), the safety and feasibility data analysis will be carried out by the Independent Data Safety and Monitoring Committee (interim analysis), after which will allow continuation to the randomized phase of the trial, if the results allow it.
In the second phase, the rest of the patients (10) will be recruited, who will be randomized 1:1, such that 5 of these patients will be implanted with the investigational medication while undergoing standard surgical reconstruction. (uranostaphyllorrhaphy), while the other 5 will only undergo standard surgical reconstruction and will constitute the control group.
The total number of patients to be included in the study will be 15, who will be followed for 24 months according to a protocolized scheme that includes 2 pre-implant visits, 1 implant visit and 9 post-implant evaluation visits.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 15
- Pediatric patients, of both sexes.
- Diagnosis of total unilateral non-syndromic cleft lip and palate (FLPNS) that will undergo surgery for correction.
- Children who have previously donated a sample of oral mucosa during the cleft lip repair procedure (cheiloplasty).
- Informed consent signed by one or both parents (or legal guardian) adequately informed of the study and willing to follow the trial procedures and instructions.
- Active infectious diseases.
- Allergies or hypersensitivity to any of the components/excipients of the Investigational Product.
- Severe hematological disorders/blood dyscrasias.
- Severe hepatic or renal dysfunction/failure.
- Serious endocrine disorders/dysfunctions.
- Malignant neoplasms
- Active HIV, HBV or HCV infection.
- Metabolic bone diseases (Paget's disease, hypercalcemia, etc.).
- Children with cleft lip and palate who present other congenital malformations that, in the opinion of the researcher, could affect the result of the study or the interpretation of the results of the study.
- Any other pathology that in the opinion of the investigator should not be included in the study for other medical or social reasons.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description experimental implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT). The experimental group will be implanted with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT). The implantation of this advanced therapy medication will cover the lateral defect generated by the mobilization of fibromucous flaps during standard surgery (uranostaphylorrhaphy
- Primary Outcome Measures
Name Time Method Evaluate implant´s feasibility and appearance of adverse events and serious adverse events related to treatment 24 months Incidence of Serious Adverse Events after at the time of the implant of study drug.
- Secondary Outcome Measures
Name Time Method Evaluation of the time of the regeneration and healing of the lateral defect of the palatine bone from the moment of the intervention 24 months Evaluation of the aesthetic result through analysis of photographs 24 months A scale designed ad hoc for the present trial will be used (aesthetic appearance assessment scale)
Hearing evaluation 24 months Otoscopic/otomicroscopic examination and tympanogram by the otorhinolaryngologist, and placement of aeration tubes if considered necessary. The need or not to perform follow-up during the trial visits will be assessed depending on whether or not the patient presents any type of pathology.
Preliminary evaluation of craniofacial growth through analysis of craniofacial photographs 24 months Quality of life evaluation 24 months Changes in quality of life will be assessed through the TAPQOL questionnaire for children between 1-5 years old.
Functional evaluation by the speech therapist 24 months Evaluation of nasal escape; palate mobility; swallowing and articulation of functional language by the speech therapist.
Trial Locations
- Locations (1)
University Hospital Virgen de las Nieves
🇪🇸Granada, Spain/Granada, Spain