Clinical trials investigate dosage, safety, efficacy and potential outcomes of drugs or treatments in disease-specific populations through controlled trials. There are typically three stages or phases of clinical trials that must be performed before a drug or treatment may be submitted to regulatory agencies (ie: U.S. Food & Drug Administration) for approval. Prior to commencing a clinical trial, a drug must have demonstrated safety and efficacy in a laboratory model.
Phase 1 Researchers test a new drug or treatment in a small group of people (usually 20-100 volunteers) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. Doses start small and increase in different patient groups until the desired effect of the treatment is observed or side effects of the treatment become too severe.
Phase 2 The drug or treatment is given to a larger group of people (up to several hundred volunteers) to see if it is effective and to further evaluate its safety. Most Phase 2 studies have randomized control (placebo) groups and treatment groups. Most of these trials are “double-blind” which means that neither the patients nor the researchers know whether the patient is receiving the treatment or placebo.
Phase 3 The drug or treatment is given to large groups of people (several hundred to thousands of volunteers) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments (or placebo), and collect information that will allow the drug or treatment to be used safely. Phase 3 trials are also randomized and most are double-blind.
To Patients The drug was submitted for review by the US Food & Drug Administration, and was approved for marketing and distribution to a patient group via prescription.
Phase 4 The drug or treatment has been FDA approved for marketing. These studies can include postmarket requirement and commitment studies that are required of or agreed to by the study sponsor. Phase 4 trials gather additional information about a drug's safety, efficacy, or optimal use.
The disease, disorder, syndrome, illness, or injury that is being studied.
Interstitial Lung Disease A broad category of over 200 lung diseases that affect the lung interstitium (walls of the air sacs of the lung). Typically, ILDs causes inflammation, fibrosis (scarring), or an accumulation of cells in the lung not due to infection or cancer.
Idiopathic Pulmonary Fibrosis A scarring disease of the lungs of unknown cause; the scarring pattern of idiopathic pulmonary fibrosis (IPF) is technically called usual interstitial pneumonia (UIP). A diagnosis of IPF requires that your doctor cannot find a cause and the presence of a pattern of UIP on either HRCT or a surgical lung biopsy sample.
Hypersensitivity Pneumonitis Hypersensitivity pneumonitis, or “HP”, is a lung disease in which inflammation and/or scar tissue (“fibrosis”) builds up around the airways and in the walls of the air sacs of the lungs.
Myositis-ILD Myositis-associated interstitial lung disease is a chronic lung disease in which scar tissue (“fibrosis”) and/or inflammation builds up in the walls of the air sacs of the lungs in a person with a diagnosis of myositis.
Rheumatoid Arthritis-ILD Rheumatoid arthritis-associated interstitial lung disease, or “RA-ILD”, is a chronic lung disease in which scar tissue (“fibrosis”) and/or inflammation builds up in the walls of the air sacs of the lungs in a person with a diagnosis of rheumatoid arthritis
Sarcoidosis ILD Sarcoidosis-associated interstitial lung disease is a chronic lung disease in which scar tissue (“fibrosis”) and/or inflammation builds up in the walls of the air sacs of the lungs in a person with a diagnosis of sarcoidosis.
Systemic Sclerosis-ILD Scleroderma-associated interstitial lung disease, or “SSc-ILD”, is a chronic lung disease in which scar tissue (“fibrosis”) and/or inflammation builds up in the walls of the air sacs of the lungs in a person with a diagnosis of scleroderma (also called “systemic sclerosis” or “SSc”).
Lung Transplant The replacement of one or both lungs with a new lung (or lungs) from a donor.
Chronic Cough Pulmonary fibrosis is often characterized by a distressing, dry cough that is difficult to control and significantly impacts quality of life. At present there are few evidence-based strategies to reduce cough in PF, but there are several agents being investigated in clinical trials.
Pulmonary Hypertension Abnormal high blood pressure in the pulmonary arteries, which connect the heart to the lungs. PH has been reported to occur in 20-85% of patients with PF.