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

NCT06408337 | Recruiting Phase 1 DMC

• 1 other identifier: FIB-BIO-2023-03
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Cleft Lip and Palate

Outcome Measures

Primary Outcomes (1)

• Evaluate implant´s feasibility and appearance of adverse events and serious adverse events related to treatment

  Incidence of Serious Adverse Events after at the time of the implant of study drug.

  24 months

Secondary Outcomes (6)

• 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

• Preliminary evaluation of craniofacial growth through analysis of craniofacial photographs

  24 months

• Hearing evaluation

  24 months

• Quality of life evaluation

  24 months

Study Arms (2)

experimental

EXPERIMENTAL

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

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

control group

NO INTERVENTION

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.

Interventions

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

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 uncoated, monofilament long-term absorbable synthetic suture.

experimental

Eligibility Criteria

• Age: 10 Months - 14 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Elisa María Cubiles Montero de Espinosa: lead
• Andalusian Network for Design and Translation of Advanced Therapies: collaborator

Study Sites (1)

University Hospital Virgen de las Nieves

Granada, Spain/Granada, 18014, Spain

RECRUITING

Related Publications (1)

• 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 generated by tissue engineering for the treatment of children with cleft palate: the BIOCLEFT study protocol. BMJ Open. 2024 Dec 5;14(12):e093491. doi: 10.1136/bmjopen-2024-093491.

  PMID: 39638596 DERIVED

MeSH Terms

Conditions

Cleft Lip

Interventions

Drug Implants

Condition Hierarchy (Ancestors)

Lip Diseases; Mouth Diseases; Stomatognathic Diseases; Mouth Abnormalities; Stomatognathic System Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Delayed-Action Preparations; Dosage Forms; Pharmaceutical Preparations

Central Study Contacts

Elisa Cubiles

CONTACT

955048278; emontero@fibao.es

Soraya Santana

CONTACT

618 93 00 77; ssantana@fibao.es

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: responsible for clinical trials

Model Details: 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.

Study Record Dates

• First Submitted: April 25, 2024
• First Posted: May 10, 2024
• Study Start: April 17, 2024
• Primary Completion (Estimated): April 17, 2028
• Study Completion (Estimated): December 17, 2028
• Last Updated: May 10, 2024

Record last verified: 2024-05

Locations

ES (1)