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Aerosolized Mesenchymal Stem Cell-Derived Exosomes for the Prevention and Treatment of Radiation-Induced Oral Mucositis: A Randomized Controlled Trial
Radiation-induced oral mucositis (RIOM) is an inevitable acute complication in radiotherapy for head and neck malignancies, characterized by a complex pathogenesis involving multiple biological processes. According to the latest data from the International Agency for Research on Cancer (IARC), approximately 650,000 new cases of head and neck cancers are diagnosed globally each year, with about 70% of patients undergoing radiotherapy. RIOM develops through a multistep pathophysiological cascade, including initiation of mucosal injury, signaling amplification, inflammatory response, ulceration, and eventual healing. Ionizing radiation induces DNA damage in oral mucosal epithelial cells, triggering increased apoptosis. This cellular injury promotes the activation and release of pro-inflammatory mediators such as tumor necrosis factor-α (TNF-α), interleukin-1β (IL-1β), and interleukin-6 (IL-6), compromising the integrity of the mucosal barrier and ultimately leading to ulcer formation. Current clinical management of RIOM remains largely supportive, relying on oral hygiene, nutritional supplementation, and pain control, with no effective prophylactic agents available. Despite extensive research into potential interventions internationally, no drugs specifically approved by the FDA or NMPA for the prevention of RIOM have reached the market. This significant unmet clinical need calls for urgent scientific and therapeutic advancement. This study aims to evaluate the therapeutic efficacy and safety profile of aerosolized exosomes derived from mesenchymal stem cells in the prevention and treatment of radiation-induced oral mucositis in patients receiving radiotherapy for head and neck cancers. The ultimate objective is to establish a novel and effective therapeutic strategy for clinical application.
Details
| Lead sponsor | First Affiliated Hospital of Guangxi Medical University |
|---|---|
| Phase | Phase 2 |
| Status | NOT_YET_RECRUITING |
| Enrolment | 200 |
| Start date | 2026-01 |
| Completion | 2028-02 |
Conditions
- Oral Mucositis (Ulcerative) Due to Radiation
Interventions
- mesenchymal stem cell-derived exosomes
Primary outcomes
- The incidence of grade ≥3 radiation-induced oral mucositis — Within 90 days from the initiation to the completion of radiotherapy
The primary endpoint is defined as the proportion of patients who develop grade 3 or higher radiation-induced oral mucositis (RIOM) from the start of radiotherapy until 90 days after the completion of radiotherapy. RIOM was graded according to the WHO oral toxicity scale criteria: Grade 0: No changes in the oral mucosa; Grade 1: Erythema of the oral mucosa with mild pain, not requiring analgesics; Grade 2: Patchy mucositis with serous exudate, moderate pain, usually not requiring analgesics; Grade 3: Confluent mucositis with severe pain requiring analgesics; Grade 4: Ulceration, hemorrhage, or necrosis, with intense pain affecting oral intake.
Countries
China