Clinical Trials Day: Empowering Patients to Participate in Active Pulmonary Fibrosis Trials
The very first clinical trial took place on May 20, 1747. Now, nearly 300 years later, clinical trials continue to serve as a critical component in drug development — especially in the pulmonary fibrosis (PF) sector. To mark Clinical Trials Day on May 20, the Pulmonary Fibrosis Foundation (PFF) aims to herald the positive impact clinical trials have made on the PF community, the current advancements in PF research and the importance of patient involvement.
Pulmonary fibrosis is a group of debilitating and incurable lung diseases which are associated with shortness of breath and a significant symptom burden, and clinical trials play a central role in leading to innovative treatments for patients who urgently need them. Data generated from clinical trials is critical to accelerating disease research and providing a better understanding of the complexity of the disease and its variable course.
Phases of Clinical Trials
Clinical trials study the effectiveness of an intervention on outcomes for patients, whether it’s a medical strategy, medication or device. There are four phases of clinical trials, including:
· Phase I — Safety: 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 — Effectiveness: 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 — Safety & Effectiveness: 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 — Long-term Use: 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.
We encourage patients to actively participate in identifying clinical trials, which will allow them to have a voice in their care process, improve patient outcomes and help find a cure.
The most important aspect of clinical trials is patient involvement. With active patient participation and collaboration with various funding agencies and investigators, the PF community will continue its efforts in improving the lives of the tens of thousands of patients living with this devastating disease.
For patients who don’t have access to clinical trials at their primary physician’s office, there are other ways to find them. One of those resources is the PFF Clinical Trial Finder, where patients can navigate more than 100 clinical research opportunities that will advance the treatment for PF, including idiopathic pulmonary fibrosis (IPF).
Active Trials in the PF Space
In 2014, the FDA approved two medications for treating IPF: nintedanib and pirfenidone — a huge success for the community. This year also holds much promise, as the research community aggressively investigates new therapeutics for all forms of PF. With studies currently in various stages of development, there are numerous clinical trials that are actively recruiting 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
For example, enrollment is now underway for PRECISIONS, an NIH-supported study which is looking at genetic risk factors and responses to therapy, applying the principles of precision medicine to the treatment of IPF patients. The trial will enroll 200 patients from approximately 20 PFF Care Center Network (CCN) sites. Other active trials now recruiting include:
· STARSCAPE Phase 3: Clinical study of Recombinant Human Pentraxin-2 (PRM-151) in patients with IPF
· REBUILD Phase 3: Clinical study of Pulsed Inhaled Nitric Oxide (INOpulse) in patients with PF and pulmonary hypertension
· ZEPHYRUS Phase 3: Clinical study of pamrevlumab in patients with IPF
Now more than ever, there are many opportunities for patients to participate in clinical trials, and the PFF plays a key role in supporting those trials. To help educate and connect patients, the PFF developed a Clinical Trials Education Center, which features a Clinical Trial Finder tool and Drug Development Pipeline, where patients can learn about the latest in drug development for PF-related conditions. The Foundation also recently launched a new Clinical Trials newsletter so patients can stay up to date on enrolling trials. In addition, the PFF Registry allows patients to participate in a very positive way to help accelerate research efforts.
While we’ve learned a tremendous amount about pulmonary fibrosis in the last several decades, more needs to be done. We don’t have curative therapies for this disease. And in order to do that, we need patients to participate in promising clinical trials to change the landscape of PF treatment in the future.
To learn more about PF clinical trials, please visit https://www.pulmonaryfibrosis.org/life-with-pf/clinical-trials and tune into this video here. For more info about PF, please visit www.AboutPF.org.