What is a Clinical Trial?

We are in an exciting and unprecedented period of PWS research. DCCR and Carbetocin (LV-101) have shown promising results following phase 3 studies. Other interesting drugs are moving through the clinical trial process but what is a clinical trial?

The clinical trials carried out surrounding PWS are basically research investigations in which people volunteer to test new treatments, interventions or tests to prevent, detect, treat or manage various aspects of the condition.  There are three key phases to a clinical trial.

In phase 1, small doses of treatment are tested on a very small number of volunteers to check that it is safe.

In phase 2, the treatment is typically tested on a larger number of people who have PWS to test if the treatment is safe and effective and to find out what the optimum dose may be.

In phase 3, the treatment is tested on an even larger number of people who have PWS using a randomised, placebo-controlled, double blind protocol.

Following a successful phase 3 trial, results are submitted to the appropriate regulatory agency which for us in Europe is the EMA (European Medicines Agency).  Once approved, you then must get NICE technological appraisal which is essential if the new treatment is to become available through the NHS.