Overview
Sponsor-declared trial summary
Phase
Phase I and Phase II (Integrated) - First administration to humans
Status
Ongoing, recruiting
Participants planned
15
Countries
1
Sites
1
Cleft lip and palate
Evaluate the safety and feasibility of using an autologous tissue-engineered nanostructured fibrin-agarose human palatal mucosa substitute in the treatment of cleft palate
Key facts
- Sponsor
- Fundacion Publica Para La Investigacion Biosanitaria De Andalucia Oriental Alejandro Otero
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Trial duration
- 17 Apr 2024 → ongoing
- Decision date (initial)
- 2023-11-21
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Others, Safety
Evaluate the safety and feasibility of using an autologous tissue-engineered nanostructured fibrin-agarose human palatal mucosa substitute in the treatment of cleft palate
Secondary objectives 1
- Use of BIOCLEFT in the treatment of patients with cleft palate, evaluating the clinical evolution of some parameters, in comparison with the control group: regeneration and healing; aesthetic appearance; craniofacial growth; hearing evaluation; speech therapist and quality of life
Conditions and MedDRA coding
Cleft lip and palate
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.1 | LLT | 10024533 | Lip and palate cleft | 10010331 |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- 1. Paediatric patients of either sex. 2. Diagnosis of non-syndromic total unilateral cleft lip and palate (FLPNS) who are going to undergo surgery for correction. 3. Children who have previously donated an oral mucosa sample during the cleft lip repair procedure (cheiloplasty). 4. 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 1
- 5. Active infectious diseases. 6. Allergies or hypersensitivity to any of the components/excipients of the investigational product. 7. Haematological disorders/severe blood dyscrasias. 8. Severe hepatic or renal dysfunction/insufficiency. 9. Severe endocrine disorders/dysfunction. 10. Malignant neoplasms. 11. Active HIV, HBV or HCV infection. 12. Metabolic bone diseases (Paget's disease, hypercalcaemia, etc.). 13. Children with cleft lip and palate who have other congenital malformations which, in the opinion of the investigator, could affect the outcome of the study or the interpretation of the study results. 14. Any other pathology that in the opinion of the investigator should not be included in the study for other medical or social reasons.
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Feasibility: if in at least 3 of the 5 patients in the initial phase it can be verified that the surgical implantation of the BIOCLEFT has been carried out satisfactorily; Safety: occurrence of any adverse event, serious or non-serious, and specifically, those adverse events and serious adverse events related to treatment and indications of efficacy regeneration and healing; aesthetic appearance; craniofacial growth; hearing evaluation; speech therapist and quality of life
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
PRD10597046 · Product
- Active substance
- Autologous Oral Mucosa-Derived Adult Keratinocytes, Ex-Vivo Expanded
- Pharmaceutical form
- LIVING TISSUE EQUIVALENT
- Route of administration
- IMPLANTATION
- Authorisation status
- Not Authorised
- MA holder
- FUNDACIÓN PARA LA INVESTIGACIÓN BIOSANITARIA DE ANDALUCÍA ORIENTAL - ALEJANDRO OTERO (FIBAO)
- Paediatric formulation
- No
- Orphan designation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Fundacion Publica Para La Investigacion Biosanitaria De Andalucia Oriental Alejandro Otero
- Sponsor organisation
- Fundacion Publica Para La Investigacion Biosanitaria De Andalucia Oriental Alejandro Otero
- Address
- Pab De Consultas Externas Floor 2, Avenida De Madrid 2 Avenida De Madrid 2
- City
- Granada
- Postcode
- 18012
- Country
- Spain
Scientific contact point
- Organisation
- Fundacion Publica Para La Investigacion Biosanitaria De Andalucia Oriental Alejandro Otero
- Contact name
- Elisa Mª Cubiles Montero de Espinosa
Public contact point
- Organisation
- Fundacion Publica Para La Investigacion Biosanitaria De Andalucia Oriental Alejandro Otero
- Contact name
- Elisa Mª Cubiles Montero de Espinosa
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Ongoing, recruiting | 15 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| Spain | 2024-04-17 | 2024-04-18 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 18 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol_BIOCLEFT_2023_506913_23_00 V4_24jun2025_CLEAN | 4 |
| Protocol (for publication) | D1_Protocol-BIOCLEFT-2023-506913-23-00 | 1 |
| Protocol (for publication) | D1_Protocol-BIOCLEFT-2023-506913-23-00 V3_13Aug2024_Clean_Fdo | 3 |
| Protocol (for publication) | D1_Protocol-BIOCLEFT-2023-506913-23-00 V3_13Aug2024_TC | 3 |
| Protocol (for publication) | D1_Protocol-BIOCLEFT-2023-506913-23-00 V4_24jun2025_Def_TC | 4 |
| Protocol (for publication) | New D1_Protocol-BIOCLEFT-2023-506913-23-00 V2 | 2 |
| Protocol (for publication) | New D1_Protocol-BIOCLEFT-2023-506913-23-00 V2 cc | 2 |
| Protocol (for publication) | Research team list Anexo IX | 1 |
| Recruitment arrangements (for publication) | K1_ Recruitment arrangements | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF infants_2023-506913-23-00 | 1 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF infants_2023-506913-23-00 CLEAN V 3_19Sep2024 | 3 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF infants_2023-506913-23-00 TC_V 3 _19Sep2024 | 3 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF infants_2023-506913-23-00 V 4_CLEAN | 4 |
| Subject information and informed consent form (for publication) | L1_SIS and ICF infants_2023-506913-23-00 V 4_TC | 4 |
| Subject information and informed consent form (for publication) | New L1_SIS and ICF infants_2023-506913-23-00 V 2 | 2 |
| Subject information and informed consent form (for publication) | New L1_SIS and ICF infants_2023-506913-23-00 V 2cc | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | Summary of product characteristics SmPC FIB-BIO-2023-03 | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis ES 2023-506913-23-00 | 1 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-08-29 | Spain | Acceptable with conditions 2023-11-20
|
2023-11-21 |
| 2 | SUBSTANTIAL MODIFICATION | SM-1 | 2024-10-15 | Spain | Acceptable 2025-01-22
|
2025-01-22 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-11-13 | Spain | Acceptable 2026-02-14
|
2026-02-18 |