Clinical Trials

Clinical trials typically consist of four phases: 

Phase 0: A very small (less than 10) pilot study to test the safety and tolerability of a new drug or treatment in humans. 

Phase 1: Involves a small group (20-80) of healthy volunteers to evaluate the safety, dosage, and side effects of the intervention. 

Phase 2: A larger group (100-300) of patients with the target condition is studied to assess the efficacy (effectiveness) and safety of the intervention. 

Phase 3: A large-scale (1,000-3,000) study to confirm the efficacy, safety, and optimal dosage of the intervention compared to existing treatments. 

Phase 4: Post-marketing surveillance studies conducted after the drug or treatment has been approved to monitor its long-term effects and safety in a wider population. 

The numbers are a bit smaller for Duchenne Muscular Dystrophy and mostly companies apply for accelerated approval when significant improvements are observed.

A list of ongoing clinical trials will be added here soon.