1st Participant Enrolled in Phase 3 Trial Testing Ofev in Children With Fibrosing ILDs

The first participant has been enrolled in a Phase 3 clinical trial evaluating Ofev (nintedanib) in children with fibrosing interstitial lung diseases (ILDs).

The Phase 3 trial, called INPEDILD (NCT04093024), is sponsored by Boehringer Ingelheim, Ofev’s manufacturer. The trial is currently recruiting participants from 6–17 years old at multiple locations in the U.S., Australia, and Europe; additional information is available here.

Childhood ILD encompasses more 200 rare respiratory diseases that affect infants, children, and adolescents, causing breathing difficulties. Some individuals with childhood ILD will develop scarring in the lungs — pulmonary fibrosis — which can exacerbate breathing problems.

There are no therapies approved yet for the treatment of childhood ILD.

Ofev is widely approved for the treatment of idiopathic pulmonary fibrosis and systemic sclerosis–associated interstitial lung disease; it has also recently received approvals for the treatment of progressive fibrosing ILDs. The medication works by blocking molecular signaling pathways that promote fibrosis. However, its safety and effectiveness in children has not yet been established.

“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,” Robin Deterding, MD, director of the Breathing Institute at Children’s Hospital Colorado and investigator on INPEDILD, said in a press release.

INPEDILD is enrolling approximately 30 children with fibrosing ILDs. Participants will be randomly assigned to receive either oral Ofev or a placebo for 24 weeks, after which all participants will be given Ofev in an open-label extension.

The study’s main goals are to evaluate the safety of Ofev, as assessed by adverse events that may occur during treatment, and to determine dose exposure — essentially, how much of the medication administered ends up in the bloodstream.

Other measurements will include lung function (measured by forced vital capacity), quality of life (measured by the Pediatric Quality of Life Questionnaire), and the occurrence of hospitalizations and exacerbations.

“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, MD, associate head of medicine pulmonology at Boehringer Ingelheim.