Clinical Trial of INOpulse Reduces Size, Results Expected Next Year

REBUILD's main goal is to see if treatment can improve physical activity

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has accepted a proposal from Bellerophon Therapeutics to reduce the size of a Phase 3 clinical trial that’s testing its INOpulse device on people with pulmonary fibrosis (PF) at risk of pulmonary hypertension (PH).

The change, made based on analyses from a prior Phase 2 study, is expected to allow the trial to finish faster; it’s now anticipated to complete enrollment in early 2023 with results available by the third quarter of next year, according to Bellerophon.

“With this study size change, we believe that we are well-positioned to accelerate the completion of our Phase 3 REBUILD study,” Naseem Amin, MD, chairman of the company’s board of directors, said in a press release.

PF is characterized by fibrosis (scarring) in the lungs, which can lead to increased pressure in their blood vessels. INOpulse delivers inhaled nitric oxide, a vasodilator that can lower blood pressure by prompting blood vessels to relax and widen.

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The Phase 3 REBUILD clinical trial (NCT03267108) is comparing INOpulse with nitric oxide against a placebo (inactive nitrogen gas) in people with PF at risk of PH. The study is actively enrolling patients, ages 18 to 80, at dozens of sites across the U.S. and Canada.

“With over 100 subjects randomized to date, we expect to complete enrollment in the first quarter of 2023, and anticipate the availability of pivotal top-line data in the third quarter of 2023,” Amin said.

The main goal of REBUILD is to determine whether INOpulse treatment can improve moderate to vigorous physical activity (MVPA), as measured with a worn sensor, after four months.

The decision to reduce the size of the trial came after analyses of a Phase 2 study that assessed whether the treatment could improve MVPA in 44 patients. Based on these analyses, REBUILD will be sufficiently statistically powered with 140 participants — in other words, there will be enough participants that, based on the expected effect, it’s mathematically almost guaranteed that if the effect is there, it will be detected.

“The target of 140 subjects maintains a statistical power of greater than 90% for MVPA, which has been accepted by the FDA as the primary endpoint for the Phase 3 REBUILD study,” Peter Fernandes, Bellerophon’s principal executive officer, said.

“The analysis presented to the FDA indicated that the trial remains adequately powered to demonstrate a statistically significant result on MVPA. We look forward to working with the Company to get this study over the finish line soon and build upon and validate the existing body of clinical evidence generated to date for INOpulse,” Steven Nathan, MD, chair of the steering committee for the REBUILD trial, said.

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