By Katie Keating, R.N., M.S

This blog is a summary of a presentation by Emily Henkle, MPH, PhD, at the NTM & Bronchiectasis Patient Conference, San Diego, CA, May 16, 2024.

This blog was reviewed by the Bronchiectasis and NTM Content Review and Evaluation Committee

Clinical trials are done to test treatments or therapies for specific diseases such as NTM and bronchiectasis. These trials help us to understand what works and what does not. Clinical trials challenge current treatments or standards of care for advancement.

Participating in a clinical trial helps researchers address important questions and learn more about NTM lung disease and bronchiectasis. It also contributes to better, safer and more effective treatments. Treatment during a medication trial could involve active medication or a placebo. A placebo (also known as a “sugar pill”) is a substance or treatment that has no active medical properties but is given to make patients think they are receiving treatment.¹

There are four phases carefully designed to research and study participants in a clinical trial to evaluate the safety and effectiveness of new drugs, medical devices, treatments or vaccines.²

• Phase I – Explores whether a treatment is safe in a small group.
• Phase II – Evaluates whether a treatment is useful in a larger target population. Researchers continue to monitor safety and also look at how well the treatment works in different doses.
• Phase III (registration) – Explores how the new treatment compares to existing treatments and side effects in a much larger group. This phase also evaluates the new treatment’s effectiveness, safety and correct dose. Information from Phase I-III is used for U.S. Food and Drug Administration (FDA) approval and regulatory agencies in other countries.
• Phase IV – Monitors long-term safety, interactions with other medications and efficacy in real-world use.

Clinical trials take time to complete, and the enrollment process can be slow due to limited eligibility. Trials begin with pre-screening, followed by obtaining informed consent from participants. Informed consent confirms your knowledge of the procedure or treatment, possible risks and benefits.³ Individuals may undergo screening for eligibility for a specific research study. If approved, the participant can enroll in the study. Monitoring for safety occurs throughout and after the study concludes.

Suggested questions to ask about before beginning a research trial:

Participation

• What is the purpose of the trial?
• How will this treatment be different from what is currently being used?
• How long will I be in the trial?
• How many visits will I need to make to the hospital or clinic?
• What kinds of tests and treatments are involved?
• How will the doctor know if the treatment is working?
• Who will oversee my care?
• Who can answer questions I have during and after the trial?

Risk and benefits

• What are the possible side effects or risks of the new treatment?
• What are the possible benefits?
• How do the possible risks and benefits of this trial compare to those of the current treatment?

Rights and costs

• How will my health information be kept private?
• If I decide to leave the trial, what other options will I have?
• Who can help answer questions from my insurance company?
• Are there any out-of-pocket costs if I take part in the trial?
• Are there payments for participating or to cover travel?

Studies are closely monitored by physicians, regulators, and data safety monitoring boards. The U.S. FDA takes part in the entire process of the study.⁴ It is important to review the details of the Informed Consent Form and ask questions before signing it. You are not required to continue with the study if you decide to withdraw after starting it. Your involvement can help in the development of new drugs and treatments for the community. Patient involvement is key to advancing drug development.

If you have any questions, please call the Bronchiectasis and NTM Information Line: 1-833-411-LUNG (5864) Or email info@bronchiectasisandntm360.org

How do I find clinical trials?

• www.clinicaltrials.gov
• Bronchiectasis and NTM 360 Actively Recruiting and Ongoing Studies
• Talk with your doctor

References:

1. The Home of Language Data, Oxford Languages, Oxford University Press 2024, Date accessed m August 22^nd, 2024, https://languages.oup.com/
2. Why Participate in a Clinical Trial? Website Title, NCI, Date accessed, June 11, 2024, https://www.cancer.gov/research/participate/clinical-trials/why-participate
3. Informed consent. AMA. Accessed August 29, 2024. https://code-medical-ethics.ama-assn.org/ethics-opinions/informed-consent.
4. Bronchiectasis and NTM Research Registry, A COPD Foundation Initiative, Date accessed, June 11, 2024 https://www.bronchiectasisandntminitiative.org/Research/Registry/Bronchiectasis-and-NTM-Research-Registry