Evidence map›Paper›PMID 24170610›Full record

Trial reportAnnals of oncology : official journal of the European Society for Medical Oncology2013

GDC-0449 in patients with advanced chondrosarcomas: a French Sarcoma Group/US and French National Cancer Institute Single-Arm Phase II Collaborative Study.

A Italiano, A Le Cesne, C Bellera, S Piperno-Neumann, F Duffaud, N Penel, P Cassier, J Domont, N Takebe, M Kind and 3 more

Registry-linked trialAbstract readClinical Trial, Phase II
In one paragraph

Trial report in Annals of oncology : official journal of the European Society for Medical Oncology, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT01267955 (A Phase 2 Study of GDC-0449 in Patients With Advanced Chondrosarcomas), which is not on this map. Cited by 51 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
51citing papers in PubMed, 1 pooled it
–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.

NCT01267955 phase2active not recruitingnot on this map

A Phase 2 Study of GDC-0449 in Patients With Advanced Chondrosarcomas

TypeinterventionalSponsorNational Cancer Institute (NCI)Ran2010 to 2027Enrolled45ConditionsClear Cell Chondrosarcoma, Dedifferentiated Chondrosarcoma, Locally Advanced Chondrosarcoma, Mesenchymal ChondrosarcomaArmsLaboratory Biomarker Analysis, Pharmacogenomic Study, Vismodegib
3 · Its place in the literature

Who cites it

51 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Trial
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  4. A Phase Ib/II Randomized Study of RO4929097, a Gamma-Secretase or Notch Inhibitor with or without Vismodegib, a Hedgehog Inhibitor, in Advanced Sarcoma.Clinical cancer research : an official journal of the American Association for Cancer Research · 2022
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  7. Review
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  11. Inhibition of hedgehog signaling ameliorates severity of chronic pancreatitis in experimental mouse models.American journal of physiology. Gastrointestinal and liver physiology · 2025
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  14. Review
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  17. Hedgehog pathway in sarcoma: from preclinical mechanism to clinical application.Journal of cancer research and clinical oncology · 2023
    Review
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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

13 authors.

A ItalianoDepartment of Medical Oncology, Institut Bergonié, Bordeaux.
A Le Cesne
C Bellera
S Piperno-Neumann
F Duffaud
N Penel
P Cassier
J Domont
N Takebe
M Kind
J-M Coindre
J-Y Blay
B Bui

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundPre-clinical data have suggested a therapeutic role of Hedgehog (Hh) pathway inhibitors in chondrosarcoma.

methodsThis phase II trial included patients with progressive advanced chondrosarcoma. They received GDC-0449 150 mg/day (days 1-28, 28-day cycle). The primary end point was the 6-month clinical benefit rate (CBR) defined as the proportion of patients with non-progressive disease at 6 months. A 6-month CBR of 40% was considered as a reasonable objective to claim drug efficacy.

resultsBetween February 2011 and February 2012, 45 patients were included. Twenty had received prior chemotherapy. Thirty-nine were assessable for efficacy. The 6-month CBR was 25.6% (95% confidence interval 13.0-42.1). All stable patients had grade 1 or 2 conventional chondrosarcoma with documented progression within the 6 months before inclusion. All but one with available data also had overexpression of the Hh ligand. Median progression-free and overall survivals were 3.5 and 12.4 months, respectively. The most frequent adverse events were grade 1 or 2 myalgia, dysgeusia and alopecia.

conclusionsGDC-0449 did not meet the primary end point of this trial. Results suggest some activity in a subset of patients with progressive grade 1 or 2 conventional chondrosarcoma. Further studies assessing its role in combination with chemotherapy are warranted. CLINICALTRIALSGOV IDENTIFIER: NCT01267955.

Indexed as

AdultAgedAged, 80 and overAnilidesChondrosarcomaDisease-Free SurvivalDrug-Related Side Effects and Adverse ReactionsFemaleFranceGene Expression Regulation, NeoplasticHedgehog ProteinsHumansMaleMiddle AgedPyridinesAnilidesHedgehog ProteinsHhAntag691PyridineschondrosarcomaGDC-0449Hedgehog pathwaytreatment

Identifiers

PMID24170610
PMCPMC3811907

What OpenQuestion holds

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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.