What Asthma Clinical Trials Study

Asthma clinical trials are regulated research studies that look for better ways to prevent, diagnose, monitor or treat asthma. Many test investigational medicines, meaning products that have not been approved for that use, while others compare treatments that are already available to learn which approach works better for certain groups of people. Studies are carried out by universities, hospitals, research networks, government agencies and drug companies, and in the United States clinical trials of new drugs and medical devices are overseen by the Food and Drug Administration. Some studies focus on adults, others on children or teenagers, and some also enroll healthy volunteers so researchers can compare results. Common areas of asthma research include:

  • New medications, including biologics, which are injected or infused medicines that target specific parts of the immune response involved in airway inflammation
  • New inhalers, inhaled medicine combinations and different ways of delivering medicine to the lungs
  • Respiratory and breathing therapies, from airway procedures to structured breathing exercise programs
  • Symptom management approaches, such as action plans, trigger reduction, digital monitoring tools and education programs
  • Severe asthma, and other asthma that stays hard to control even with standard treatment

How Asthma Clinical Trials Work

Trials of new medicines usually move through phases. Phase I studies look mainly at safety and dosing in a small group of people, often healthy volunteers. Phase II and III studies look at safety and effectiveness in progressively larger groups, and phase III studies often compare a new treatment with standard treatment. Phase IV studies continue to monitor safety after a medicine is approved. Many trials assign participants to groups by chance, a process called randomization, so the groups can be compared fairly. One group may receive the study treatment while another receives standard treatment or a placebo, which looks like the study medicine but contains no active drug. In many trials neither participants nor research staff know who is in which group, which is called masking or blinding.

Before joining, each person goes through informed consent. The research team explains the purpose, procedures, schedule, possible risks and possible benefits, and answers questions. For a child, a parent or guardian gives permission, and children who are old enough are usually asked to agree as well. Informed consent continues throughout the study, so participants can keep asking questions as it goes on. The consent form is not a contract, and participants may leave a study at any time, for any reason. In the United States, clinical trials are generally reviewed and monitored by an Institutional Review Board, a group that includes physicians, statisticians, patient advocates and other community members, to help make sure risks are as low as possible and are worth any potential benefits.

Who Can Take Part in an Asthma Study

Each study sets its own eligibility criteria, which describe who can and cannot join. These rules help protect participants and make sure the results can answer the research question. Because criteria differ so much from one study to the next, someone who does not fit one study may fit another, and meeting the listed criteria is only the first step, since the study team makes the final decision after screening. Interested people usually attend one or more screening visits, where study staff review medical history and current medicines, perform a physical exam, and may run breathing tests such as spirometry, blood tests or allergy tests. Some asthma studies also include a run-in period, during which symptoms and medicine use are tracked for a set time before any study treatment begins. Criteria often include:

  • Age range, since many studies enroll only adults or only children
  • Asthma type and severity, sometimes confirmed with lung function tests
  • Current and past asthma medicines, including how long they have been used
  • History of flare-ups, emergency visits or courses of oral steroids
  • Smoking history and other health conditions, such as heart disease or other lung diseases
  • For some biologic studies, blood markers linked to certain types of airway inflammation

Possible Benefits and Risks to Weigh

Taking part in a trial has possible benefits and possible risks, and participants may or may not benefit personally. Some people join to be closely monitored by a research team, to try an investigational approach that is not otherwise available, or to help researchers learn information that may improve care for others in the future. At the same time, researchers often do not know whether a study treatment will be helpful, harmful or no different from standard care. Risks can include side effects, some of which may not yet be known, and some participants receive a placebo or standard treatment instead of the new approach. Study procedures such as blood draws or breathing tests can be uncomfortable, and some studies ask participants to adjust usual medicines under supervision. Many trials also have an independent data and safety monitoring board that reviews results while the study is underway, and a study can be paused or stopped if safety concerns arise.

Time, Visits, Costs and Questions to Ask

Participation takes time, and schedules vary widely. Studies may last a few weeks, several months or longer, with visits to a hospital, clinic or research center, and some add phone or online check-ins. Participants may be asked to keep a symptom diary, record peak flow readings or use an electronic inhaler monitor at home. The informed consent form describes payments and costs. Many studies pay for the study treatment and research-related tests and visits, and some cover research-related travel costs such as parking or meals. Participants or their health insurance plans generally remain responsible for regular health care that is not part of the research, so checking coverage in advance can help avoid surprises. Helpful questions for the study team include:

  • What main question is this study trying to answer?
  • How many visits are required, how long does each one take, and how long does the study last?
  • Could the study involve a placebo, and can usual asthma medicines continue?
  • Which costs does the study cover, and which might be billed to insurance?
  • What happens if symptoms get worse during the study?
  • Will study results be shared with participants and their regular clinician?

Working With Asthma Specialists and Finding a Study

A clinical trial is one option to discuss with a clinician alongside standard asthma care. Established treatments include daily controller medicines such as inhaled corticosteroids, quick-relief inhalers, add-on long-acting bronchodilators, allergy shots for some people, and biologic medicines for certain types of severe asthma. Asthma that seems hard to control is not always severe asthma; inhaler technique, missed doses, triggers at home or other health conditions can play a role. An allergist or immunologist, or a pulmonologist, can confirm the diagnosis, review the current treatment plan and help decide whether a study makes sense. Questions about the cost of standard treatment, insurance coverage or lower-cost alternatives for a specific prescription are worth raising with a clinician, pharmacist or insurer. The American Academy of Allergy, Asthma and Immunology and the American College of Allergy, Asthma and Immunology offer online tools for finding allergists, and a doctor's board certification can be checked through the American Board of Medical Specialties.

ClinicalTrials.gov, run by the National Library of Medicine, lists studies in the United States and other countries and can be searched by condition, location and recruitment status. The National Heart, Lung, and Blood Institute offers its own search for studies it supports, and the American Lung Association's Airways Clinical Research Centers network conducts asthma and COPD trials at sites across the country. A listing does not mean a study has been evaluated or endorsed by the federal government, so it is worth reviewing the sponsor, study locations and contact details carefully. Saving or printing a study record and bringing it to an appointment gives a licensed clinician the chance to review whether the study fits a person's health history and current treatment.

Conclusion

Asthma clinical trials study new medications such as biologics and inhaled therapies, along with respiratory therapies and symptom management approaches, and some focus on severe or hard-to-control asthma. Each study follows a written plan called a protocol and uses informed consent, trials in the United States are generally reviewed and monitored by an Institutional Review Board, and participation remains voluntary throughout. Eligibility depends on criteria set by each study, and possible benefits should be weighed against risks, time commitments and practical details such as visits and which costs are covered. Searching ClinicalTrials.gov, preparing questions for the study team and reviewing options with an allergist, pulmonologist or other licensed clinician can help people with asthma and their families make informed decisions about research participation alongside standard care.

Citations

  • ClinicalTrials.gov, National Library of Medicine - Learn About Studies (clinicaltrials.gov)
  • National Institutes of Health - NIH Clinical Research Trials and You: The Basics (nih.gov)
  • NIH Clinical Center - FAQs About Clinical Studies (cc.nih.gov)
  • National Heart, Lung, and Blood Institute - Find NHLBI Clinical Trials and Studies (nhlbi.nih.gov)
  • National Heart, Lung, and Blood Institute - Asthma: Treatment and Action Plan (nhlbi.nih.gov)
  • U.S. Food and Drug Administration - Basics About Clinical Trials (fda.gov)
  • American Lung Association - Clinical Trials (lung.org)
  • American Lung Association - Airways Clinical Research Centers (lung.org)
  • American Lung Association - Severe Asthma (lung.org)
  • American Academy of Allergy, Asthma and Immunology - Asthma Symptoms, Diagnosis, Management and Treatment (aaaai.org)
  • American College of Allergy, Asthma and Immunology - Asthma Treatment (acaai.org)
  • American Board of Medical Specialties - Certification Matters (certificationmatters.org)
  • Centers for Disease Control and Prevention - Controlling Asthma (cdc.gov)