Lurbinectedin in patients with pretreated neuroendocrine tumours: Results from a phase II basket study

Eur J Cancer. 2022 Sep;172:340-348. doi: 10.1016/j.ejca.2022.06.024. Epub 2022 Jul 10.


Background: Patients with neuroendocrine tumours (NETs) need alternative therapies after failure of first-line therapy.

Patients and methods: This phase II trial evaluated lurbinectedin, a selective inhibitor of oncogenic transcription, at 3.2 mg/m2 as a 1-h intravenous infusion every 3 weeks in 32 NETs patients treated in the second- or third-line setting. The primary efficacy endpoint was overall response rate (ORR) according to RECIST v1.1 assessed by the investigators. Secondary endpoints included duration of response (DoR), progression-free survival (PFS), overall survival (OS) and safety.

Results: Two of 31 evaluable patients had confirmed partial responses (ORR = 6.5%; 95%CI, 0.8-21.4%). Median DoR was 4.7 months (95% CI, 4.0-5.4 months), median PFS was 1.4 months (95% CI, 1.2-3.0 months) and median OS was 7.4 months (95% CI, 3.4-16.2 months). Lurbinectedin showed an acceptable, predictable and manageable safety profile. The most common grade 3/4 toxicity was neutropenia (40.6%; grade 4, 12.4%; febrile neutropenia, 3.1%).

Conclusions: Considering the exploratory aim of this trial that evaluated a heterogeneous population of NETs patients, and the signs of antitumour activity observed (two confirmed partial responses and seven long disease stabilisations), further development of lurbinectedin is warranted in a more selected NETs population.

Trial registration number: Sponsor Study Code: PM1183-B-005-14. EudraCT number: 2014-003773-42.

Clinicaltrials: gov reference: NCT02454972.

Keywords: Lurbinectedin; Neuroendocrine tumours; Phase II trial; Small cell.

Publication types

  • Clinical Trial, Phase II

MeSH terms

  • Carbolines* / adverse effects
  • Heterocyclic Compounds, 4 or More Rings* / adverse effects
  • Humans
  • Neuroendocrine Tumors* / drug therapy
  • Neuroendocrine Tumors* / pathology
  • Response Evaluation Criteria in Solid Tumors


  • Carbolines
  • Heterocyclic Compounds, 4 or More Rings
  • PM 01183

Associated data