Evidence map›Paper›PMID 32026407›Full record

ArticlePulmonary therapy2019

A Phase 1b Study of Vismodegib with Pirfenidone in Patients with Idiopathic Pulmonary Fibrosis.

Antje Prasse, Murali Ramaswamy, Shaun Mohan, Lin Pan, Andrew Kenwright, Margaret Neighbors, Paula Belloni, Peter P LaCamera

Registry-linked trialOpen access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Pulmonary therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02648048 (A Single Arm, Multicenter, Open-label, Phase 1b Study to Assess the Safety and Tolerability of Oral Vismodegib in Combination With Pirfenidone in Patients With Idiopathic Pulmonary Fibrosis), which is not on this map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
0.6field-weighted citation impact, top 33% of its field
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.

NCT02648048 phase1completednot on this map

A Single Arm, Multicenter, Open-label, Phase 1b Study to Assess the Safety and Tolerability of Oral Vismodegib in Combination With Pirfenidone in Patients With Idiopathic Pulmonary Fibrosis

TypeinterventionalSponsorHoffmann-La RocheRan2016 to 2016Enrolled21ConditionsIdiopathic Pulmonary FibrosisArmsPirfenidone, Vismodegib
3 · Its place in the literature

Who cites it

8 citing papers in PubMed, 18 citations in OpenAlex.

  1. Targeting Fibrosis: From Molecular Mechanisms to Advanced Therapies.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025
    Review
  2. Article
  3. Review
  4. Review
  5. Article
  6. Review
  7. Targeting Alveolar Repair in Idiopathic Pulmonary Fibrosis.American journal of respiratory cell and molecular biology · 2021
    Review
  8. Chaotic activation of developmental signalling pathways drives idiopathic pulmonary fibrosis.European respiratory review : an official journal of the European Respiratory Society · 2020
    Review
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

8 authors at 3 institutions in 2 countries.

Antje PrasseHannover Medical School and Fraunhofer Institute for Toxicology and Experimental Medicine, Hannover, Germany. prasse.antje@mh-hannover.de.
Murali RamaswamyPulmonIx, LLC, and Cone Health, Greensboro, NC, USA.
Shaun MohanGenentech, Inc., South San Francisco, CA, USA.
Lin PanGenentech, Inc., South San Francisco, CA, USA.
Andrew KenwrightGenentech, Inc., South San Francisco, CA, USA.
Margaret NeighborsGenentech, Inc., South San Francisco, CA, USA.
Paula BelloniGenentech, Inc., South San Francisco, CA, USA.
Peter P LaCameraSt. Elizabeth's Medical Center, Boston, MA, USA.
Cone Health · USFraunhofer Institute for Toxicology and Experimental Medicine · DESt. Elizabeth's Medical Center · US

Funding

F. Hoffmann-La Roche Ltd N/AGenentech, Inc. N/A
6 · The paper itself

Abstract

introductionComponents of the hedgehog signaling pathway are upregulated in patients with idiopathic pulmonary fibrosis (IPF). Vismodegib, a small-molecule inhibitor of hedgehog signaling, when used in combination with currently available antifibrotic therapy, may be more efficacious than antifibrotics alone. The objective of this study was to evaluate the safety and tolerability of vismodegib plus pirfenidone in patients with IPF.

methodsTwenty-one patients were enrolled in a phase 1b open-label trial to receive vismodegib 150 mg plus pirfenidone 2403 mg/day once daily. Key endpoints were safety, tolerability, and pharmacokinetics. Exploratory endpoints included change from baseline to week 24 in % predicted forced vital capacity (FVC) and University of California, San Diego Shortness of Breath Questionnaire (UCSD-SOBQ) scores, as well as pharmacodynamic changes in hedgehog biomarker C-X-C motif chemokine ligand 14 (CXCL14).

resultsAll patients reported at least one treatment-emergent adverse event (AE), most frequently muscle spasms (76.2%). Serious AEs were reported in 14.3% of patients; one event of dehydration was considered related to vismodegib. One patient died due to IPF progression, unrelated to either treatment. More patients discontinued vismodegib than pirfenidone (42.9% vs. 33.3%, respectively). Changes from baseline to week 24 in % predicted FVC and UCSD-SOBQ scores were within known endpoint variability. In contrast to findings in basal cell carcinoma, vismodegib had no effect on circulating CXCL14 levels.

conclusionThe safety profile was generally consistent with the known profiles of both drugs, with no new safety signals observed in this small cohort. There was no pharmacodynamic effect on CXCL14 levels. Future development of vismodegib for IPF may be limited due to tolerability issues.

trial registrationClinicalTrials.gov NCT02648048. Plain language summary available for this article.

fundingF. Hoffmann-La Roche Ltd. and Genentech, Inc.

Indexed as

Hedgehog signaling pathwayIdiopathic pulmonary fibrosisPirfenidoneSafetyTolerabilityVismodegib

Identifiers

PMID32026407
PMCPMC6967289
OpenAlexW2963847814

What OpenQuestion holds

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LicenceCC BY
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Registered trials

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.