Clinical trial NCT06408337: Phase I-IIa, to Evaluate the Safety, Feasibility, and Efficacy of the Use of BIOCLEFT in the Treatment of Cleft Palate.
Official title: Phase I-IIa, Randomized, Controlled, Open-label, Single-center Clinical Trial to Evaluate Safety, Feasibility, and Efficacy of the Use of BIOCLEFT in the Treatment of Patients With Cleft Palate
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Cleft Lip and Palate
Lead sponsor: Fundación Pública Andaluza para la Investigación Biomédica Andalucía Oriental
Sponsor class: OTHER
Collaborators: name: Andalusian Network for Design and Translation of Advanced Therapies
First posted: 2024-05-10
Enrollment: 15
Sex: ALL
Minimum age (years): 10
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: The initial phase of the trial is non-randomized and involves the sequential recruitment of 5 subjects. All of them will be implanted with the autologous substitute for the human palatal mucosa of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT), after reconstruction of the palate. In the second phase, the remaining patients (10) will be recruited, who will be randomized 1:1, so 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.
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Evaluate implant´s feasibility and appearance of adverse events and serious adverse events related to treatment
timeFrame: 24 months
Secondary outcomes:
measure: Evaluation of the time of the regeneration and healing of the lateral defect of the palatine bone from the moment of the intervention
timeFrame: 24 months
measure: Evaluation of the aesthetic result through analysis of photographs
timeFrame: 24 months
measure: Preliminary evaluation of craniofacial growth through analysis of craniofacial photographs
timeFrame: 24 months
measure: Hearing evaluation
timeFrame: 24 months
measure: Quality of life evaluation
timeFrame: 24 months
measure: Functional evaluation by the speech therapist
timeFrame: 24 months
Other outcomes:
Locations:
city: Granada
country: Spain
facility: University Hospital Virgen de las Nieves
state: Spain/Granada
Study officials:
References: