NCT07312656

Aerosolized Mesenchymal Stem Cell-Derived Exosomes for the Prevention and Treatment of Radiation-Induced Oral Mucositis

Aerosolized Mesenchymal Stem Cell-Derived Exosomes for the Prevention and Treatment of Radiation-Induced Oral Mucositis: A Randomized Controlled Trial

• Status: Not Yet Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 200 (estimated)
• Sponsor: First Affiliated Hospital of Guangxi Medical University · Academic / Other
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

This study employs a randomized controlled design, aiming to enroll a total of 200 head and neck cancer patients scheduled to undergo radiotherapy from three medical centers. The experimental group (100 patients) will receive a mesenchymal stem cell-derived exosome spray, while the control group (100 patients) will follow a placebo-controlled design without any pharmacological intervention, receiving only standard radiotherapy and supportive care. The primary endpoint is the incidence of grade 3 or higher radiation-induced oral mucositis, which will be used to evaluate the efficacy of the investigational agent in preventing and ameliorating this condition through comparison with the control group.

Conditions

• Oral Mucositis (Ulcerative) Due to Radiation

Interventions

Type	Name	Description
DRUG	mesenchymal stem cell-derived exosomes	Mesenchymal Exosome Administration Protocol: 1. Administration Timing: Begin 7 days prior to radiotherapy initiation and continue until 14 days post-radiotherapy completion. 2. Preparation: Remove the exosome vial, open the cap, add 5mL of saline solution, and secure the spray cap. 3. Administration Frequency: 3 times daily (30 minutes after breakfast, lunch, and dinner). 4. Dosage: Approximately 1.5mL per spray. 5. Administration Method: Oral spray. Evenly spray onto the oral mucosal surface. Avoid eating or rinsing for 20 minutes after administration.

Timeline

• Start date: 2026-01-01
• Primary completion: 2027-06-01
• Completion: 2028-02-01
• First posted: 2025-12-31
• Last updated: 2025-12-31

Locations

1 site across 1 country: China