Clinical Trials Education Center

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

• 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.

• 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 above.
  

• 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, which identifies genes and genetic variants that may be associated with the development and progression of PF.

The intervention is a process or action that is the focus of a clinical study, including drugs and medical devices.

• Oral Drug:  A drug taken through the mouth, typically in a tablet or capsule, but could also include liquids.
  

• Inhaled Drug:  A drug breathed directly into the lungs, most often through an aerosol device or nebulizer.
  

• Intravenous (IV) Injection:  A drug injected or infused directly into a vein via needle or tube, typically administered by a trained healthcare professional.
  

• Subcutaneous (SC) Injection:  A drug injected into the tissue layer under the skin via a short needle, typically in the side of the upper arm.

Some studies include a “placebo,” sometimes called a “sugar pill.” A placebo looks like medicine but has no active ingredients in it. Having one group of volunteers take the drug being studied and another group take the placebo is an important way for researchers to measure how well the new drug will work. If you’re part of a trial that uses placebos, you won’t know which group you’re in—but you’ll be closely monitored even if you’re on a placebo.

• The principal investigator (PI) leads the study, records and analyzes the data, and directs the study staff. The PI creates and follows the study protocol for how the study will be carried out. 

• The clinical research coordinator (CRC) handles day-to-day study activities and will likely be your main point of contact.

• Organizations and people responsible for volunteer protections include the U.S. Food and Drug Administration (FDA) and the institutional review board (IRB). They review the study before it starts, make sure the team follows the rules, and keep you safe and informed. An IRB is made up of doctors, researchers, and members of the community. Its role is to make sure the study is ethical and the rights and welfare of participants are protected.

As a clinical trial volunteer, you’ll have several tools in place to protect your safety and well-being. Researchers use a process called informed consent to provide possible participants with information about the potential benefits, risks, and alternatives to participation. Before joining the study, you must sign an informed consent document to show you received and understand this information. Signing the document is not a contract. You may still withdraw from a study at any time, even if the study is not over.

• What’s the purpose of this study?

• How long is the clinical trial anticipated to last?

• How long will my participation last?

• What will I have to do to participate?

• What are the risks?

• How often and how many times will I have to visit the clinic?

• Who will pay for my participation? (Some trials—not all—will pay volunteers for their commitment or travel time.)

• If there is medical treatment involved as part of the study, who will pay for it?

• Can I maintain all my current treatments and medications while participating?

• Who should I contact for different issues like symptoms, side effects, and scheduling routine appointments?

• Will the study results be provided to me?

• If the study results are favorable, will I have an opportunity to receive the drug or treatment later?

An early or unexpected end to a clinical trial can be disappointing. However, researchers can still collect critical information even if the results aren’t positive. This is an essential part of the scientific discovery process and contributes toward the broader body of PF research.

Whether or not you are participating in a clinical trial, you can join the PFF Community Registry! The PFF Registry is a series of surveys, and just about everyone who has been affected by PF is eligible to join. Your survey responses will be used for scientific analysis in attempts to better understand how PF and ILD progresses, responds to treatments, and affects individuals.