What is a clinical trial?
Clinical trials are research studies that explore whether a medical strategy, treatment, or device is safe and effective for humans. These studies also may show which medical approaches work best for certain illnesses or groups of people. They are a mechanism to bring new drugs and treatments forward for approval to address the challenges of any medical condition.
According to the Australian Clinical Trials website, until recently, children were rarely included in trials of medical interventions and much is still unknown about how children respond to some medications, medical devices and tests.
Parents and guardians choose to enrol their children in clinical trials for a range of reasons, including a desire to provide optimal care for their child and to contribute to improving the treatment and care of other children.
While new medicines cannot be brought to market without clinical trials and those who apply to participate in them, it is important to remember that a clinical trial is first and foremost an experiment. People typically participate in a clinical drug research in the hope of getting access to a potential treatment.
There are multiple clinical trials underway for Sanfilippo Syndrome, however there is still no FDA-approved drugs for treating Sanfilippo Syndrome.