Cleft Lip and PalatePhase 1/2NCT06408337
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 study testing implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT). in people with Cleft Lip and Palate, run by Elisa María Cubiles Montero de Espinosa at 1 site in Spain.
Trial 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… Read more about this study
- Who is it for?
- Ages 10 to 14 months
- Conditions the study is for
- Cleft Lip and Palate
- What is being tested?
- implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT).
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteSpain
- Study dates
- 17 Apr 2024to 17 Dec 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 14 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
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- 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 locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Spain1 site, 1 recruiting
- Recruiting
University Hospital Virgen de las Nieves
Granada, Spain/Granada, 18014
Ricardo Fernández, MD,PhD, 671 593 192, rfdezvalades@me.com
Miguel Alaminos, MD, PhD, 958 241000 ext. 20461, malaminos@ugr.es
About this study
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
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.
- Conditions
- Cleft Lip and Palate
- Being tested
- implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT).
- Medical subject headings
- Cleft Lip
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT).Procedure or surgery
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: 24 months
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)
Measured: 24 months
Preliminary evaluation of craniofacial growth through analysis of craniofacial photographs
Measured: 24 months
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.
Measured: 24 months
Quality of life evaluation
Changes in quality of life will be assessed through the TAPQOL questionnaire for children between 1-5 years old.
Measured: 24 months
Functional evaluation by the speech therapist
Evaluation of nasal escape; palate mobility; swallowing and articulation of functional language by the speech therapist.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 May 2024
- Study start17 Apr 2024
- Primary completion17 Apr 2028 (estimated)
- Study completion17 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Cleft Lip and PalateStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- 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.
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Elisa María Cubiles Montero de Espinosa
- Sponsor type
- University, hospital or other
- Collaborators
- Andalusian Network for Design and Translation of Advanced Therapies
- Responsible party
- Sponsor-investigator, Elisa María Cubiles Montero de Espinosa, Fundación Pública Andaluza para la Investigación Biomédica Andalucía Oriental
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06408337
- Sponsor's protocol ID
- FIB-BIO-2023-03
- Status last verified
- May 2024
- First submitted
- 25 Apr 2024
- Last updated
- 10 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
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 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.
PubMed 39638596
Study at a glance
From the registry record, last updated by the sponsor on 10 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06408337
- Acronym / sponsor ID
- FIB-BIO-2023-03
- Conditions
- Cleft Lip and Palate
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Elisa María Cubiles Montero de EspinosaUniversity, hospital or other
- Collaborators
- Andalusian Network for Design and Translation of Advanced Therapies
- Study start
- 17 Apr 2024 (actual)
- Primary completion
- 17 Apr 2028 (estimated)
- Study completion
- 17 Dec 2028 (estimated)
- First posted
- 10 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| experimental | Experimental | Procedure: implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT).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 |
| 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. |
Who may qualify
- Ages
- 10 to 14 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 uncoated, monofilament long-term absorbable synthetic suture. | Procedure or surgery | experimental |
Primary outcome
Evaluate implant´s feasibility and appearance of adverse events and serious adverse events related to treatment
Time frame: 24 months
Incidence of Serious Adverse Events after at the time of the implant of study drug.
6 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2024.
- Derived from this study: PubMed 39638596Espana-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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing implantation with an autologous human palatal mucosa substitute made of nanostructured fibrin-agarose generated by tissue engineering (BIOCLEFT). in people with Cleft Lip and Palate, run by Elisa María Cubiles Montero de Espinosa at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Elisa Cubiles (955048278, emontero@fibao.es), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evaluate implant´s feasibility and appearance of adverse events and serious adverse events related to treatment (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06408337, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2024.