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The Huntington Study Group (HSG) has been conducting research in Huntington disease for over 15 years. Please see below for some basic information about research.

What is the point of clinical research?
What types of clinical research studies are there?
Are there different stages of experimental drug studies?
What are ‘randomized, double blind’ studies?
Are there risks in clinical research?
How is clinical research different than seeing my doctor?


What is the point of clinical research?

The primary point of research is to answer a general (or “scientific”) question by collecting information in an organized way and the primary responsibility of an investigator is to follow the research plan (or “protocol”). Research subjects, like patients, are asked to report  their problems and benefits to the investigator.  But, unlike traditional patient care  which is highly individualized, reserarch procedures and assignments to experimental treatment are based on a uniform and  pre-specified research protocols. Thus, research carries different risks from clinical care and requires the consent of research participants. At the same time, nothing should be done in clinical research that would intentionally harm the research participant.

What types of clinical research studies are there?

Clinical research involves studies that follow research participants over time without changing their treatment, and these are called observational studies. Other types of studies make some change in individuals’ lives, for example, asking them to take an experimental medication or follow certain procedures, and these studies are called interventional studies. Interventional studies involving experimental medication are frequently called clinical trials. This name can be confusing at times because the word “trials” suggest that the experimental medication is something that the patient is trying, but this is just a name that is used for interventional clinical research studies involving experimental medications.

Are there different stages of experimental drug studies?

In general clinical trials of experimental medication are conducted in phases and these are usually called Phase I, II, and III trials. Phase I trials generally test the experimental medication in small groups of people (a few dozen) to identify side effects, to determine safe dosages, and to learn about the experimental medication levels in the body. Phase II trials usually have larger groups of people, a few hundred, to get more information about the safety in individuals with the disease as well as to determine initial hints of effectiveness. Phase III trials are often larger involving hundreds to thousands of people and used to try to confirm that an experimental medication has a beneficial effect. For example in Huntington disease the aim of the study might be to improve the motor symptoms, to improve thinking, or to slow the overall progression of disability. In addition to demonstrating benefit, Phase III trials also help to show what side effects are common with an experimental medication or experimental treatment.

What are ‘randomized, double blind’ studies?

Most commonly Phase III trials involve some comparison to a new experimental medication, either a standard experimental medication (active control) or against no active experimental medication at all (placebo control) depending on what other treatments are available. Other words used to describe clinical trials include “randomized”, which indicates that research participants are assigned to their treatment by chance rather than choice and, “blinded” meaning that the research participants and investigators do not know which treatment an individual has been assigned to. Using comparison groups, blinding and randomization are all thought to help reduce the chance of the study giving a false answer and improve the quality of the study. Clinical research studies, especially clinical trials, are how new knowledge is generated, and they help to find new treatments. Being a research participant is not for everyone, but without participants there cannot be new treatments in the future.

Are there risks in clinical research?

Because research is designed to follow a specific protocol there are potential risks in participating in the research, as it is not designed to benefit the research participant. Participation in research is voluntary. All clinical research participants are requested to read and sign an informed consent document. This consent document is an important way about learning the facts about a research study before deciding whether or not to participate. There are good materials available to give further information about participating in clinical trials including the section on “Understanding Clinical Trials” on the clinicaltrials.gov webpage (www.clinicaltrials.gov) and on the HD Drug Works webpage (www.hddrugworks.org).

How is clinical research different than seeing my doctor?

Everyone has seen a doctor (or some other health care provider, also known as a “clinician”) at some point in their life. A clinician provides care to a person in order to help that individual in some way, usually to help them feel better. Decisions to change treatment are usually based on the individual’s response, and it is the clinician’s responsibility to try to do the best for a patient. Clinical research is intended to answer a general question, not to help a specific individual (although it may also do that). The people involved in clinical research are called investigators and research participants. In clinical research, like clinical care, these people have responsibilities to one another, but they are different than the responsibilities in clinical care. In these ways clinical research is different than but similar to clinical care. It is important for participants and investigators to understand these differences.


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last updated: April 2010