PF Foundation Urging Patients to Learn About, Enroll in Ongoing Trials

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by Patricia Inacio PhD |

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As part of the worldwide commemoration of Clinical Trials Day, held each year on May 20, the Pulmonary Fibrosis Foundation (PFF) is urging people with pulmonary fibrosis (PF) to join ongoing clinical studies.

In its call for participants, the PFF highlighted how patient involvement is the “most important aspect of clinical trials” — a key step in advancing research into the causes of PF and in the search for potential cures.

“We encourage patients to actively participate in clinical trials, which will allow them to have a voice in their care process, improve patient outcomes and help find a cure,” Joyce S. Lee, MD, senior medical advisor for research and health care quality at the PFF, said in a press release.

According to the Association of Clinical Research Professionals (ACRP), Clinical Trials Day is “a well-deserved ‘time out’ to recognize the people who conduct clinical trials and to say ‘thanks’ for what they do every day to improve public health.” On its website, the ACRP said the date of the annual Clinical Trials Day was chosen to “recognize the day that James Lind started what is often considered the first randomized clinical trial aboard a ship on May 20, 1747.”

Lind, a British surgeon, served on board the HMS Salisbury of Britain’s Royal Navy.

Among the PFF’s initiatives to help people with PF to know which clinical trials are active and enrolling participants, the foundation created the clinical trials education center, which includes a clinical trial finder tool. Armed with knowledge on trials that may interest them, patients can then discuss with their healthcare providers if any studies may be a good match.

Another initiative offered by the PFF is a drug development pipeline, which patients can use to learn about the latest candidate therapies for PF and related disorders.

Additionally, the PFF has launched a newsletter to keep people with PF and their families and caregivers in the loop concerning trial enrollment. One important available tool is the PFF Registry, a platform where patients can share detailed medical information, including symptoms, test results, and the use of certain medications. That data, once de-identified, is then made available to scientists investigating PF, so they may use it to “discover the causes of PF” and potentially a cure. Before the data is released, all identifiers are eliminated to ensure patient privacy, the PFF notes.

“Pulmonary fibrosis encompasses a group of conditions with a significant symptom burden, and clinical trials play a central role in leading to additional treatments for patients who urgently need them,” Lee said.

“Data generated from clinical trials is critical to accelerating disease research and providing a better understanding of the complexity of these diseases and its variable course,” she added.

Examples of current trials enrolling PF patients include the Phase 3 trials PRECISIONS (NCT04300920), REBUILD (NCT03267108), ZEPHYRUS (NCT03955146), and STARSCAPE (NCT04594707).

PRECISIONS will evaluate the effectiveness and safety of N-acetyl cysteine (NAC), an antioxidant that may help prevent lung damage. In the trial, NAC or a placebo will be given to approximately 200 IPF patients, ages 40 and older, who have a gene variant called TOLLIP rs3750920 T/T. This particular variant is found in approximately 25% of IPF cases and is associated with better clinical responses to NAC treatment.

The study is underway at seven clinical sites in the U.S., and enrollment is active on four sites: Stanford University, in California; Tulane University, in New Orleans; the University of Michigan; and the University of Rochester, in New York.

The first patient was enrolled at the University of Michigan earlier this year. The trial is expected to conclude in 2025.

In the REBUILD study, scientists will evaluate the safety and effectiveness of Bellerophon Therapeutics’ investigational therapy INOpulse — inhaled nitric oxide — in up to 300 PF patients, ages 18–80. Participants must have been on oxygen therapy by nasal cannula for at least four weeks, and must be at risk for developing pulmonary hypertension (PH).

INOpulse delivers nitric oxide (NO), a potent vasodilator, in a form that can be inhaled through a nasal cannula. Of note, vasodilators are compounds that cause blood vessels to relax and widen, lowering blood pressure and improving blood flow.

Researchers will be randomly assign participants to receive either inhaled NO (at a dose of 45 mcg/kg) or a placebo through the INOpulse device for four months. Patients who complete the four-month treatment period may choose to enter the trial’s open-label extension, in which all will receive INOpulse-NO.

The study is ongoing at more than 30 U.S. clinical sites. The first patient was enrolled in December 2020 and the study is expected to conclude in October 2022.

Around 565 IPF patients who have not responded, or are intolerant to treatment with IPF-approved therapies, such as Ofev (nintedanib) and Esbriet (pirfenidone), are expected to enroll in ZEPHYRUS.

During the trial, participants will be randomly assigned to receive FibroGen’s investigational therapy pamrevlumab (30 mg/kg) or a placebo, both given directly into the bloodstream, every three weeks, for 48 weeks (around 11 months). Those who complete the study may be eligible to continue treatment with pamrevlumab in a separate open-label extension study.

Pamrevlumab, formerly known as FG-3019, is a man-made antibody that blocks the activity of connective tissue growth factor (CTGF), a protein that is known to contribute to the progressive and excessive tissue scarring characteristic of IPF and other fibrotic diseases.

The first patient enrolled in ZEPHYRUS in July 2019. The trial is expected to wrap up in January 2023.

The STARSCAPE study is open to patients previously given PRM-151, an investigational therapy for PF, in the open-label stage of the Phase 2 PRM-151-202 study (NCT02550873). Patients who have completed the EU Phase 3 study WA42293 (2020-000791-38) also are eligible to enroll.

PRM-151, originally developed by Promedior, now part of the Roche group, is a lab-made form of pentraxin 2, an anti-fibrotic protein that can resolve fibrosis by blocking further inflammation. Lower levels of pentraxin 2 in IPF patients are associated with increased fibrotic damage.

Aiming to enroll around 700 IPF patients, the study offers multiple clinical sites in the U.S. and several countries in Europe. It is excepted to complete by December 2028.

“While we’ve learned a tremendous amount about pulmonary fibrosis in the last several decades, more needs to be done,” Lee said. “We don’t have curative therapies for these diseases. And in order to do that, we need patients to participate in promising clinical trials to change the landscape of pulmonary fibrosis treatment in the future.”

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