Boehringer Ingelheim today announced that the first patient has enrolled in InPedILD™, a global Phase III trial assessing the dosing and safety profile of nintedanib in children and adolescents between 6 and 17 years old with clinically significant fibrosing interstitial lung disease (ILD).
Childhood ILD (chILD) includes more than 200 rare respiratory disorders that can affect infants, children and adolescents, making it difficult for them to breathe. In some cases, fibrosis can develop that involves scarring and damage to the lungs. This can lead to a significant impact on the daily life of those affected, as well as their families, including high morbidity and mortality. There are currently no approved therapies available for the treatment of chILD.
“Some children with interstitial lung disease may develop serious fibrosis that progresses,” said the coordinating investigator Prof. Robin Deterding, M.D., Director of the Breathing Institute, Children’s Hospital Colorado. “Though the underlying causes of pulmonary fibrosis may be different in children, we’re excited to determine if treating the mechanism of fibrosis improves children’s lung fibrosis, as it does in adults.”
Nintedanib, which is marketed as Ofev®, is currently approved in more than 80 countries for the treatment of people living with idiopathic pulmonary fibrosis (IPF). It is also approved in more than 40 countries as the first and only therapy to slow the rate of decline in pulmonary function in patients with systemic sclerosis-associated ILD (SSc-ILD). Ofev® recently also obtained approval in the USA, Canada, Japan, Brazil, Argentina and the EU, for a third indication, as the first and only treatment for patients living with chronic fibrosing ILDs with a progressive phenotype.
“The initiation of this clinical trial further advances critical research aimed to address significant unmet needs in one of the most vulnerable pediatric patient communities impacted by ILDs,” said Gregory Porta, founder and member of the board of directors, chILD Foundation (www.child-foundation.org).
“Boehringer Ingelheim is proud to start this trial to provide valuable insights as we evaluate this potential treatment for children and adolescents with these rare and heterogenous conditions for which there are currently no treatments with proven efficacy and no randomized controlled trials,” said Susanne Stowasser, M.D., associate head of pulmonology medicine at Boehringer Ingelheim. “This study represents our ongoing commitment to address unmet needs and advance research for adult and pediatric patients living with pulmonary fibrosis.”
This double-blind, randomized, placebo-controlled, multicenter international Phase III trial [NCT04093024] will enroll patients in approximately 70 sites in 24 countries. The study, in children and adolescents (6 to 17 years old) with clinically significant fibrosing ILD, will evaluate the dose-exposure and safety of nintedanib, administered orally on top of usual care for 24 weeks, followed by open-label treatment with nintedanib of variable duration. The primary endpoints are blood concentration of nintedanib at week two and week 26, and the number of patients with treatment-emergent adverse events (TEAE) at week 24. Secondary endpoints, among others, include change in forced vital capacity (FVC) percent predicted from baseline at week 24 and week 52, which is an established measurement of lung function; absolute change from baseline in health-related quality of life, as measured by the Pediatric Quality of Life Questionnaire™ (PedsQL™) at week 24 and week 52; time to first respiratory-related hospitalization over the whole trial; and time to first acute ILD exacerbation or death over the whole trial. For more information about the trial, please visit the website.