Corneal Confocal Microscopy: a Surrogate Endpoint for Neurodegeneration in Clinical Trials



Neurodegeneration due to genetic, metabolic, autoimmune, inflammatory and toxic insult can result in a range of peripheral neuropathies (DPN, HIV, CIPN, chemotherapy induced peripheral neuropathy, amyloid neuropathy, Friedreich’s ataxia, idiopathic small fiber neuropathy) and central neurodegenerative disorders (Parkinson’s disease, dementia, multiple sclerosis, ischemic stroke and traumatic brain injury. The accurate quantification of neurodegeneration and regeneration is key to assessing outcomes in clinical trials of disease modifying therapies for peripheral and central neurodegenerative diseases. Corneal confocal microscopy (CCM) is an imaging method to quantify neurodegeneration and regeneration. It is time to start the conversation on how to translate evidence into action and provide a roadmap to assess the current evidence and key hurdles for the adoption of CCM as a biomarker and surrogate endpoint for neurodegenerative disease.


  1. Define the current advantages and disadvantages for the end-points in clinical trials of peripheral neuropathies and major central neurodegenerative diseases.
  2. Assess the current evidence for CCM as a surrogate marker for peripheral neuropathies and central neurodegenerative diseases.
  3. Identify the challenges for U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) acceptance of CCM as a surrogate end point for disease modifying clinical trials of neurodegenerative disease. 

Anticipated Outcomes

  1. Doha CCM consensus statement: Publication of a document identifying the key challenges, opportunities and actions for the wider adoption of CCM for the assessment of peripheral and central neurodegenerative disease in the Journal of the Peripheral Nervous System. 
  2. Consortium study: Establish multi-center consortium studies utilizing CCM in peripheral and central neurodegenerative diseases.