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Clinical Trials 101

ABOUT CLINICAL TRIALS

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. Clinical trials produce the best data available for health care decision making.

The purpose of clinical trials is research, so the studies follow strict scientific standards. These standards protect patients and help produce reliable study results.

CLINICAL RESEARCH 101

Types of Clinical Trials: There are three (3) different types of clinical trials: interventional, observational, and expanded access.

  1. Interventional studies are those in which the research subjects are assigned by the investigator to a treatment or other intervention, and their outcomes are measured.

  2. Observational studies are those in which individuals are observed (no intervention in the study) and their outcomes are measured by the investigators.

  3. Expanded access studies are those in which the Food and Drug Administration (FDA) allows manufacturers to provide investigational new drugs (under certain circumstances), to patients with serious diseases or conditions who cannot participate in a clinical trial.

TYPES OF RESEARCH

Basic science studies are done to increase the fundamental understanding of the disease process on a molecular and cellular level. In the long term, this type of research can provide the scientific basis for the development of effective treatments.

Epidemiological research studies a disease within certain populations; research in this area can lead to better understanding of potential risk factors, including occupational or environmental exposures that may lead to development of a disease.

Translational research translates basic research findings into meaningful treatments and clinical applications. This research is becoming increasingly prominent and the National Institute of Health is proposing the creation of a National Center for Advancing Translational Sciences (NCATS).

Clinical Research studies are performed to determine the safety and efficacy of medications, treatment regiments, and diagnostic procedures. The different phases of clinical trials for the development of new therapies are described below.

Quality of Life/Social Science studies are a subcategory of Clinical Research, which had been previously often ignored but has gained increased attention by the research community and the regulatory agencies. The term is used to refer to the general “well-being” of individuals undergoing specific treatment modalities, not only including medications but also supportive therapies (nutrition, exercise, respiratory therapy and psycho-social support).

With studies currently in various stages of development, there are a variety of clinical trials that are actively seeking the participation of patients. Some of the therapeutic approaches currently being studied include:

  • Anti-fibrotic therapies, which may slow or inhibit the production of scar tissue (fibrosis).

  • Inhibitors of “growth factor” proteins, which block proteins that stimulate the lungs to make scar tissue

  • Stem cell therapies, which may help damaged lungs to heal

  • Genetic research to identify genes and genetic variants that may be associated with the development and progression of PF
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.

  1. Phase I: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.

  2. Phase II: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.

  3. Phase III: The drug or treatment is given to large groups of people 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.

  4. Phase IV: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

The PFF does not promote or represent that any investigational new drugs mentioned are safe or effective. The information provided is for general information only and is not intended as medical or other professional advice. It should not be relied upon as a substitution for consultation with qualified professionals who are familiar with your individual needs. PFF does not recommend any commercial product or services. The safety and scientific validity of this study is the responsibility of the study sponsor and investigators

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