Clinical Trials for EB

Phase I: First Application in Humans

Goals:

• Safety and tolerability testing with low doses of the drug
• Analysis of the period of effectiveness of the drug
• Analysis of the behavior of the drug in the human body (degradation products)

Attributes:

• Small number of participants (n = 20-100)
• Healthy volunteers (rarely patients)
• Specialized centers
• Open

Phase II: Therapeutic Trial Phase

Goals:

• Verification of the therapeutic effect
• Ascertaining the optimal dose
• Safety (toxicity) testing
• Checking the feasibility of the application
   

Attributes:

• First application in patients with disease (n = 100-500)
• Medical institutions and private practices
• Open
• Blinded
• Comparative
• Multiple doses

Phase III: Therapeutic Confirmation

Goals: 

• Confirmation of its effectiveness and safety
• Review of effectiveness compared to known drugs
   

Attributes:

• Extensive studies (n = 1000-5000)
• Medical institutions and private practices
• Multi-centric
• Blinded
• Comparative
   

Phase IV: Long-Term Observation

Goals: 

• Data “from real life”
• Safety monitoring (does the product interact with other drugs?)
• Therapy optimization of approved drugs
   

Attributes:

• Very large number of patients
• Further development (i.e. new areas of application)

[Source: https://www.eb-clinet.org/clinical-trials/about-clinical-trials/]