Glossary


Clinical trial
A clinical trial (also called clinical research) is a research study in human volunteers to answer specific health questions. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people and ways to improve health. Interventional trials determine whether experimental treatments or new ways of using known therapies are safe and effective under controlled environments. Observational trials address health issues in large groups of people or populations in natural settings.

Ankylosing spondylitis
Ankylosing spondylitis is an inflammatory disease that causes the small bones of the spine to fuse, which makes the spine less flexible and, in turn, affects posture. People with this condition experience pain and stiffness and may also have fatigue. There is no cure for ankylosing spondylitis, but symptoms can be managed.

Autosomal dominant polycystic kidney disease
Autosomal dominant polycystic kidney disease is an inherited condition resulting in the decline of kidney function. This is caused by cells in the kidney not developing properly, leading to cysts growing. People are born with the condition and the disease is not noticed in children until the cysts are large enough to cause symptoms, such as stomach pain, high blood pressure, blood in urine and kidney stones. Currently, there is no cure for autosomal dominant polycystic kidney disease, but the problems that occur as a result of the condition can be managed.

Crohn's disease
Crohn’s disease is a type of bowel disease that causes inflammation of the digestive tract. People can experience diarrhea, stomach aches and cramps, blood in poo, tiredness and weight loss. Symptoms can come and go over time. The exact cause is unknown, and although there is no cure, symptoms can be controlled.

Exclusion criteria
The factors that allow someone to participate in a clinical trial are called “inclusion criteria” and those that disallow someone from participating are called “exclusion criteria”. These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

Healthy volunteer
A healthy volunteer is a person with no known significant health problems participating in a clinical trial. Healthy volunteers are often used in Phase 1 clinical trials.

Idiopathic pulmonary fibrosis
Idiopathic pulmonary fibrosis is a lung disease that causes scar tissue to form on the lungs, which makes it harder to breathe. The condition gets worse over time, with people experiencing shortness of breath, a persistent dry cough, tiredness, loss of appetite, weight loss, and rounded and swollen fingertips. Its exact cause is unknown, but patients tend to be between 50 and 80 years of age; it is rare to occur in those who are younger than this. There is currently no cure for idiopathic pulmonary fibrosis and current management focuses on managing the symptoms.

Informed consent
Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant’s native language is not English, translation assistance can be provided. The research team then provides an informed consent document, which includes details about the study, such as its purpose, duration, required procedures and key contacts. Any risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.

Inclusion criteria
The factors that allow someone to participate in a clinical trial are called “inclusion criteria” and those that disallow someone from participating are called “exclusion criteria”. These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan to study.

Rheumatoid arthritis
A long-term condition that causes inflammation, swelling and pain in joints. It occurs when the immune system is not working as it should and the body attacks healthy cells by mistake. Rheumatoid arthritis can also affect other parts of the body, including the lungs, heart and eyes. Patients tend to experience ‘flare-ups’ where they are most likely to experience symptoms, and remission where their symptoms are better.

Osteoarthritis
The most common type of arthritis, osteoarthritis, is a long-term condition that can affect any joint in the body, causing it to become stiff and painful. The severity of symptoms experienced varies between individuals, with some people experiencing mild symptoms that come and go and others experiencing much more severe symptoms that can markedly affect day-to-day life. The exact cause is unknown, and although there is no cure, symptoms can be managed.

Placebo
A placebo is an inactive pill, liquid or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.

Protocol
A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial, the schedule of tests, procedures, medications and dosages, and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.

Psoriatic arthritis
Psoriatic arthritis is a long-term condition; it is a type of arthritis that affects some people with the skin condition psoriasis. It can cause symptoms in the skin, joints and nails and can also cause tiredness and fever. Symptoms can go away then come back in a period known as a flare-up; these flare-ups can last for a period of days or months.

Scleroderma/systemic sclerosis
Scleroderma is an autoimmune condition that results in the body attacking connective tissue that sits under the skin; this leads to hard, thickened areas and can affect internal organs and blood vessels. The condition can include lots of different types and some are associated with more severe symptoms than others. There is no cure for scleroderma, but the symptoms can be managed.

Side effects
Side effects are any undesired actions or effects of a drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.

Trial coordinator
This is the person responsible for managing the clinical trial day-to-day and, alongside your doctor, would be the contact point for you while on the clinical trial.

Ulcerative colitis
Ulcerative colitis is a condition that causes inflammation in the digestive tract leading to the formation of ulcers. Common symptoms include diarrhea and abdominal and rectal pain, rectal bleeding, weight loss, fatigue and fever, and these often develop gradually. The exact cause is unknown, but diet and stress can make the symptoms worse. There is no cure for ulcerative colitis, but symptoms can be managed.

Uveitis
Uveitis is a rare condition that causes inflammation of the eye, causing pain and changes in vision. It can affect either one or both eyes, and symptoms can appear suddenly in some people but more gradually for others. Uveitis is thought to be linked to issues with the body’s immune system in some cases and, less often, can occur as a result of eye injury; however, in some people the cause is unknown. Treatment for uveitis depends on what has caused it and the area of eye that is affected. Steroids and immunosuppressants may be used and surgery may be considered if uveitis comes back regularly.

Phase 1 clinical trial
These studies occur in only a small number (often 20 to 80 people) of people, and the main focus is to ensure the safety of a treatment. The researchers will test for side effects, determine how much of the treatment is safe to give and learn how the body copes with the treatment. Often, these studies will take place in healthy volunteers.

Phase 2 clinical trial
Phase 2 studies are tested in a larger group of people (100 to 300) and include those who have the disease or condition the treatment is looking to treat. During these studies, it is possible to begin to get an idea as to whether the drug is likely to work and researchers will look to see what dose may work best.

Phase 3 clinical trial
If the results from the studies in Phase 1 and 2 are positive (they have passed safety checks and have shown evidence the treatment may work), then Phase 3 studies will take place. Here, the treatment is tested in a much larger number of people (1000 to 3000) with the disease or condition and the treatment is compared with an existing treatment or placebo to see how well it works.

Phase 4 clinical trial
Phase 4 studies occur after the treatment has been shown to be effective and is available to patients. During these studies, the long-term safety of the treatment will be reviewed. How well the treatment continues to work in patients will also be monitored.