Phase I-IIa, to Evaluate the Safety, Feasibility, and Efficacy of the Use of BIOCLEFT in the Treatment of Cleft Palate.

Elisa María Cubiles Montero de Espinosa

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Cleft Lip and Palate

Treatments: Procedure: implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT).

Study type: Interventional

Funder types: Other

Identifiers

NCT06408337

FIB-BIO-2023-03

Details and patient eligibility

About

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.

Full 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.

Enrollment: 15 estimated patients

Sex: All

Ages: 10 to 14 months old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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15 participants in 2 patient groups

experimental

Experimental group

Description:

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

Treatment:

Procedure: implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT).

control group

No Intervention group

Description:

These patients will be included in the control group and will receive the usual treatment for their disease (uranoestafilorrafia), without applying any type of restriction to the area used for the movement of the arms.