Clinical trials, in our setting, means testing of medicines or treatment on humans. A clinical observational study, means in our setting, that you observe patients to measure disease progression or development. Whithout doing any particular change or intervention, you observe over time if there for instance are any symptom development like behavioral changes, alteration in blood values or in the brain volume or structure.
In summary clinical trials are done in order to provide scientific evidence for the safety and efficacy/benefit of a drug or an intervention (for instance medicine, dietary supplements or other non-drug therapies). A clinical observational study is done to learn more about how Huntingtons disease develop over time. In essence to answer the question: How is the course of the disease in humans? This knowlegde is crucial in order to understand the full complexity of HD and use it both to target treatment and to enable us to track benefit of treatment. When we know in great detail how the disease progresses without any treatment, we can measure if the development of the disease slows down or haults when the person is treated.
How are the trials and studies done?
Everyone who wants to conduct a trial or study needs approval from the authoroties and ethic committees. Drug trials performed in Europe needs approval both from the European Medicines Agency and from the ethic committees in each country where there are participants. These authorities are responsible for vetting the risk/benefit ratio of the trial – their approval does not mean that the therapy is ‘safe’ or effective, only that the trial may be conducted.
Depending on product type and development stage, investigators initially enroll volunteers or patients into small pilot studies and subsequently conduct progressively larger scale comparative studies. Clinical trials can vary in size and cost, and they can involve a single research center or multiple centers, in one country or in multiple countries. Clinical study design aims to ensure the data collected in the study holds high standards and measure the potential effect – positive, negative or no effect – in a scientifically sound way. The procedures has to be publicly available to allow independent evaluation of the reported results.