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

Overview

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.

Interventions

• Procedure implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT).
  cleft palate repair surgery, all patients in the study will receive the standard treatment (uranostaphyllorrhaphy), with the only difference that patients assigned to the experimental study group will additionally have BIOCLEFT tissue obtained through tissue engineering grafted onto them. the lateral area of the palatine bone exposed when mobilizing the flaps. For the implant, the BIOCLEFT fabric will be placed in the area of exposed bone, sized to the size of the defect and sutured using uncoat

Primary outcome measures

• Evaluate implant´s feasibility and appearance of adverse events and serious adverse events related to treatment [Time frame: 24 months]

Secondary outcome measures (6)

• Evaluation of the time of the regeneration and healing of the lateral defect of the palatine bone from the moment of the intervention [Time frame: 24 months]
• Evaluation of the aesthetic result through analysis of photographs [Time frame: 24 months]
• Preliminary evaluation of craniofacial growth through analysis of craniofacial photographs [Time frame: 24 months]
• Hearing evaluation [Time frame: 24 months]
• Quality of life evaluation [Time frame: 24 months]
• Functional evaluation by the speech therapist [Time frame: 24 months]

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Open label

Primary purpose

Treatment

Study locations

Spain · 1 center

• University Hospital Virgen de las Nieves — Granada

Publications

• Espana-Lopez A, Fernandez-Valades R, Cubiles E, Garzon I, Martin-Piedra MA, Carriel V, Campos F, Martinez-Plaza A, Vallejo D, Liceras-Liceras E, Chato-Astrain J, Garcia-Garcia OD, Sanchez-Porras D, Avila-Fernandez P, Etayo-Escanilla M, Quijano B, Aguilar E, Campos A, Carmona G, Alaminos M. Phase I-IIa clinical trial to evaluate the safety, feasibility and efficacy of the use of a palate mucosa gen PMID 39638596

Identifiers

NCT: NCT06408337 · FIB-BIO-2023-03

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗