Evidence map›Paper›PMID 38987119›Full record

Trial reportJournal of cystic fibrosis : official journal of the European Cystic Fibrosis Society2025

Efficacy and safety of LAU-7b in a Phase 2 trial in adults with cystic fibrosis.

Michael W Konstan, Deepika Polineni, James F Chmiel, Lara Bilodeau, Peter G Middleton, Elias Matouk, Jean-Marie Houle, Radu Pislariu, Patrick Colin, Irenej Kianicka and 8 more

Abstract readClinical Trial, Phase IIRandomized Controlled TrialMulticenter Study
PubMed Full text at PMC Publisher
In one paragraph

Trial report in Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

1 citing paper in PubMed.

  1. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

18 authors.

Michael W KonstanCase Western Reserve University School of Medicine and Rainbow Babies and Children's Hospital, OH, USA.
Deepika PolineniWashington University School of Medicine, MO, USA.
James F ChmielIndiana University School of Medicine and Riley Hospital for Children at IU Health, IN, USA.
Lara BilodeauInstitut Universitaire de Cardiologie et de Pneumologie de Québec-Université Laval, QC, Canada.
Peter G MiddletonCITRICA, Department of Respiratory & Sleep Medicine, Westmead Hospital and Clinical School University of Sydney, NSW, Australia.
Elias MatoukResearch Institute of the McGill University Health Centre, QC, Canada.
Jean-Marie HouleLaurent Pharmaceuticals Inc, Montreal, Canada.
Radu PislariuLaurent Pharmaceuticals Inc, Montreal, Canada.
Patrick ColinLaurent Pharmaceuticals Inc, Montreal, Canada.
Irenej KianickaLaurent Pharmaceuticals Inc, Montreal, Canada.
Diane PotvinInnovaderm Research, QC, Canada.
Danuta RadziochResearch Institute of the McGill University Health Centre, QC, Canada.
Tom KotsimbosThe Alfred Hospital, Melbourne VIC, Australia.
Jonathan B ZuckermanMaine Medical Center, Portland, ME, USA.
Samya Z NasrUniversity of Michigan Health System, Ann Arbor, MI, USA.
Theodore G LiouUniversity of Utah, Salt Lake City, UT, USA.
Larry C LandsResearch Institute of the McGill University Health Centre, QC, Canada. Electronic address: larry.lands@mcgill.ca.
study Investigators

Funding

CTSA UM1 Program at University of UtahUM1TR004409 · NCATS · UTAH STATE HIGHER EDUCATION SYSTEM--UNIVERSITY OF UTAH · PI RACHEL HESS, Jennifer Juhl Majersik · 2023 to 2026
$21.9M
NCATS NIH HHS UM1 TR004409
6 · The paper itself

Abstract

backgroundLung inflammation is associated with tissue damage in cystic fibrosis (CF). LAU-7b, a novel oral drug candidate, was shown to control inflammation and stabilize CFTR protein in the epithelial membrane during inflammatory stress in preclinical models of CF.

methodsA double-blind, randomized, placebo-controlled Phase 2 study was conducted to evaluate efficacy and safety of LAU-7b in adults with CF. LAU-7b or placebo was administered over 24 weeks as six 21-day treatment cycles each separated by 7 days. The primary efficacy endpoint was the absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV

resultsA total of 166 subjects received at least one dose of study drug (Intent-To-Treat population, ITT), of which 122 received ≥5 treatment cycles (Per-Protocol population, PP). Both treatment arms showed a mean lung function loss at 24 weeks of 1.18 ppFEV

conclusionAlthough the study did not meet its primary efficacy endpoint in the ITT population, LAU-7b was generally well tolerated and showed evidence of preservation of lung function to support further development.

Indexed as

Cystic FibrosisAdolescentAdultDouble-Blind MethodFemaleForced Expiratory VolumeHumansMaleMiddle AgedRespiratory Function TestsTreatment OutcomeYoung AdultCFTR modulatorsCystic fibrosisInflammationLAU-7bLung function loss reduction

Identifiers

PMID38987119
PMCPMC13472192

What OpenQuestion holds

Textmetadata
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.