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RecruitingNCT06408337Updated May 10, 2024

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

A Phase 1/2 interventional study of implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT). in Cleft Lip and Palate, sponsored by Elisa María Cubiles Montero de Espinosa. Recruiting at 1 site in Spain. Open to participants aged 10 Months to 14 Months. Per ClinicalTrials.gov, last updated 2024-05-10.

Sponsored by Elisa María Cubiles Montero de Espinosa · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
15
Allocation
Randomized
Ages
10 Months to 14 Months
Sex
All
01

Study 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.

Read the 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.

02

Conditions studied

  • Cleft Lip and Palate
03

Who can participate

Ages eligible
10 Months to 14 Months
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
04

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
15 participants (estimated)

Study arms

  • 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).

  • No intervention
    control group

    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

  • Procedureimplantation 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 uncoated, monofilament long-term absorbable synthetic suture.

05

What researchers measure

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.

    Time frame: 24 months

Secondary outcomes

  1. 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

  2. Evaluation of the aesthetic result through analysis of photographs

    A scale designed ad hoc for the present trial will be used (aesthetic appearance assessment scale)

    Time frame: 24 months

  3. Preliminary evaluation of craniofacial growth through analysis of craniofacial photographs

    Time frame: 24 months

  4. Hearing evaluation

    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.

    Time frame: 24 months

  5. Quality of life evaluation

    Changes in quality of life will be assessed through the TAPQOL questionnaire for children between 1-5 years old.

    Time frame: 24 months

  6. Functional evaluation by the speech therapist

    Evaluation of nasal escape; palate mobility; swallowing and articulation of functional language by the speech therapist.

    Time frame: 24 months

06

Study locations

1 of 1 sites recruiting
  • University Hospital Virgen de las Nieves
    Granada, Spain/Granada 18014, Spain
    • Ricardo Fernández, MD,PhD · Contact · rfdezvalades@me.com · 671 593 192
    • Miguel Alaminos, MD, PhD · Contact · malaminos@ugr.es · 958 241000
    • Ricardo Fernández, MD,PhD · Principal investigator
    • Antonio España, MD,PhD · Sub investigator
    • Esther Liceras, MD, PhD · Sub investigator
    • Daniel Vallejo, MD, PhD · Sub investigator
    • Adoración Martínez, MD, PhD · Sub investigator
    • Ana Marín, MD, PhD · Sub investigator
    • Rosario Cortés, MD, PhD · Sub investigator
    • Belén Pérez, MD, PhD · Sub investigator
    Recruiting
07

Registry details

Key details

Study ID
NCT06408337
Lead sponsor
Elisa María Cubiles Montero de Espinosa
Collaborators
Andalusian Network for Design and Translation of Advanced Therapies
Responsible party
Elisa María Cubiles Montero de Espinosa (responsible for clinical trials, Fundación Pública Andaluza para la Investigación Biomédica Andalucía Oriental) — Sponsor-investigator
First posted
May 10, 2024
Start date
Apr 17, 2024
Primary completion
Apr 17, 2028 (estimated)
Completion
Dec 17, 2028 (estimated)
Last update
May 10, 2024

Study contacts

Elisa Cubiles
Contact
emontero@fibao.es
955048278
Soraya Santana
Contact
ssantana@fibao.es
618 93 00 77

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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