First-in-man mesenchymal stem cells for radiation-induced xerostomia (MESRIX): study protocol for a randomized controlled trial

Trials. 2017 Mar 7;18(1):108. doi: 10.1186/s13063-017-1856-0.


Background: Salivary gland hypofunction and xerostomia are major complications following radiotherapy for head and neck cancer and may lead to debilitating oral disorders and impaired quality of life. Currently, only symptomatic treatment is available. However, mesenchymal stem cell (MSC) therapy has shown promising results in preclinical studies. Objectives are to assess safety and efficacy in a first-in-man trial on adipose-derived MSC therapy (ASC) for radiation-induced xerostomia.

Methods: This is a single-center, phase I/II, randomized, placebo-controlled, double-blinded clinical trial. A total of 30 patients are randomized in a 1:1 ratio to receive ultrasound-guided, administered ASC or placebo to the submandibular glands. The primary outcome is change in unstimulated whole salivary flow rate. The secondary outcomes are safety, efficacy, change in quality of life, qualitative and quantitative measurements of saliva, as well as submandibular gland size, vascularization, fibrosis, and secretory tissue evaluation based on contrast-induced magnetic resonance imaging (MRI) and core-needle samples. The assessments are performed at baseline (1 month prior to treatment) and 1 and 4 months following investigational intervention.

Discussion: The trial is the first attempt to evaluate the safety and efficacy of adipose-derived MSCs (ASCs) in patients with radiation-induced xerostomia. The results may provide evidence for the effectiveness of ASC in patients with salivary gland hypofunction and xerostomia and deliver valuable information for the design of subsequent trials.

Trial registration: EudraCT, Identifier: 2014-004349-29. Registered on 1 April 2015., Identifier: NCT02513238 . First received on 2 July 2015. The trial is prospectively registered.

Keywords: Mesenchymal stem cells; Xerostomia.

Publication types

  • Clinical Trial, Phase I
  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adipose Tissue / cytology*
  • Biopsy, Large-Core Needle
  • Clinical Protocols
  • Denmark
  • Double-Blind Method
  • Feasibility Studies
  • Female
  • Humans
  • Magnetic Resonance Imaging
  • Male
  • Mesenchymal Stem Cell Transplantation* / adverse effects
  • Oropharyngeal Neoplasms / radiotherapy*
  • Prospective Studies
  • Radiation Injuries / diagnostic imaging
  • Radiation Injuries / etiology
  • Radiation Injuries / physiopathology
  • Radiation Injuries / surgery*
  • Recovery of Function
  • Research Design
  • Salivation
  • Submandibular Gland / diagnostic imaging
  • Submandibular Gland / physiopathology
  • Submandibular Gland / surgery*
  • Time Factors
  • Treatment Outcome
  • Ultrasonography, Interventional
  • Xerostomia / diagnostic imaging
  • Xerostomia / etiology
  • Xerostomia / physiopathology
  • Xerostomia / surgery*

Associated data

  • EudraCT/2014-004349-29