评估组织工程生成的腭黏膜用于治疗腭裂患儿的安全性、可行性和有效性的I-IIa期临床试验：BIOCLEFT研究方案
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.
作者信息
España-López Antonio, Fernández-Valadés Ricardo, Cubiles Elisa, Garzón Ingrid, Martin-Piedra Miguel Angel, Carriel Víctor, Campos Fernando, Martínez-Plaza Adoración, Vallejo Daniel, Liceras-Liceras Esther, Chato-Astrain Jesús, García-García Oscar Dario, Sánchez-Porras David, Ávila-Fernández Paula, Etayo-Escanilla Miguel, Quijano Blanca, Aguilar Elisabet, Campos Antonio, Carmona Gloria, Alaminos Miguel
机构信息
Craniofacial Malformations and Cleft Lip and Palate Management Unit (Unidad de Fisurados Labiopalatinos y Malformaciones Craneofaciales), University Hospital Virgen de las Nieves, Granada, Spain.
Department of Stomatology, Faculty of Dentistry, University of Granada, Granada, Spain.
出版信息
BMJ Open. 2024 Dec 5;14(12):e093491. doi: 10.1136/bmjopen-2024-093491.
INTRODUCTION
The current gold standard treatment for patients with orofacial clefts is surgical repair of the palatal defect (uranostaphylorrhaphy), which is associated with growth defects and hypoplasia of the maxillofacial structures. This trial aims to evaluate the potential of a bioengineered artificial palate mucosa, created through tissue engineering with autologous stromal and epithelial cells and nanostructured fibrin-agarose biomaterials, to enhance treatment outcomes for patients with unilateral cleft lip and palate.
METHODS AND ANALYSIS
This phase I-IIa clinical trial aims to evaluate the feasibility and biosafety of a procedure involving grafting bioartificial palate mucosa onto the areas of denudated bone in patients undergoing uranostaphylorrhaphy. The control patients will undergo standard surgical treatment. Five patients will be included in the first biosafety phase. In the second phase, 10 patients will be randomly assigned to the intervention or control group (1:1). The intervention group will undergo standard surgical treatment followed by the application of autologous bioartificial palate mucosa. Feasibility will be analysed at the time of surgery. Nine postimplant visits will be scheduled over a 2-year follow-up period, in which local and systemic biosafety will be investigated by determining graft evolution, including signs of necrosis, rejection, inflammation and patient factors. Preliminary signs of efficiency will be explored by sequentially evaluating craniomaxillofacial development, hearing impairment, speech capability and quality of life of the family. The research will be published in journals and posted in the relevant repositories when available.
ETHICS AND DISSEMINATION
This study has been approved by the Committee of Ethics in Research with Medicinal Products (CEIm) and authorised by the Spanish Medicines Agency (AEMPS). The results of this study will be published in peer-reviewed journals.
TRIAL REGISTRATION NUMBER
NCT06408337; ClinicalTrials.gov: EuclinicalTrials. eu: 2023-506913-23-00.
引言
目前，口面部裂隙患者的金标准治疗方法是腭裂缺损修复术（腭成形术），但该手术会导致颌面结构生长缺陷和发育不全。本试验旨在评估通过自体基质和上皮细胞以及纳米结构纤维蛋白 - 琼脂糖生物材料进行组织工程构建的生物工程人工腭黏膜，对单侧唇腭裂患者治疗效果的提升潜力。
方法与分析
本I - IIa期临床试验旨在评估将生物人工腭黏膜移植到接受腭成形术患者的裸露骨区域这一手术的可行性和生物安全性。对照组患者将接受标准手术治疗。在首个生物安全阶段将纳入5名患者。在第二阶段，10名患者将被随机分配至干预组或对照组（1:1）。干预组将接受标准手术治疗，随后应用自体生物人工腭黏膜。手术时将分析可行性。在2年的随访期内安排9次植入后访视，通过确定移植物的演变情况，包括坏死、排斥、炎症迹象以及患者因素，来研究局部和全身的生物安全性。通过依次评估颅颌面发育、听力障碍、言语能力和家庭生活质量，探索有效性的初步迹象。研究结果将在可用时发表在期刊上并发布至相关资料库。
伦理与传播
本研究已获得药品研究伦理委员会（CEIm）批准，并得到西班牙药品管理局（AEMPS）授权。本研究结果将发表在同行评审期刊上。
试验注册号
NCT06408337；ClinicalTrials.gov：EuclinicalTrials.eu：2023 - 506913 - 23 - 00
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