Clinical Trials

Many patients choose to participate in clinical trials after consulting with their medical physician. A clinical trial is a study performed to determine if an experimental treatment is safe and/or effective to treat a specific medical condition.

The research community is aggressively investigating new treatments for all forms of pulmonary fibrosis (PF). While the long-term goal of research is to prevent and cure the disease, present therapeutic approaches are focused on slowing disease progression and extending the life expectancy of PF patients.

about clinical trials

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

If you are considering participation in a clinical trial, it is very important that you first discuss your options with your medical provider.


  • 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).

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 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.
  • 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.
  • 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.
  • 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.

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

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


Benefits – Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

  • Play an active role in your own health care.
  • Gain access to new research treatments before they are widely available.
  • Obtain expert medical care at leading health care facilities during the trial.
  • Help others by contributing to medical research to find an effective treatment for pulmonary fibrosis.

Risks – There are also risks to participating in clinical trials.

  • There may be unpleasant, serious, or even life-threatening side effects to experimental treatment.
  • The experimental treatment may not be an effective treatment.
  • The study may require more of your time and attention than would taking a non-study treatment, including additional trips to the study site, hospital stays, completion of health diaries or complex dosage requirements.

Additional Resource Information on clinical trials can be found here.


For a complete list of active, enrolled, and recently completed clinical trials, please visit the National Institutes of Health (NIH) website: This list includes three types of clinical trials: expanded access studies, interventional studies, and observational studies. For an explanation of each type of clinical trial, please refer to the “About Clinical Trials” section above.  

A number of clinical trials are currently enrolling patients, each with slightly different enrollment criteria. To search for trials actively enrolling in your area:

  1. Go to
  2. Search for “pulmonary fibrosis” and “<your state>” in the search box.
  3. This will yield a list of studies. Select “include only open studies” to receive a list of those that are actively recruiting patients.
  4. Click on a study title and scroll down until you see “Contacts and Locations” for a list of study locations and contact information.

To learn more about clinical trials, please contact the PFF Patient Communication Center at 844.TalkPFF (844.825.5733) or

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