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LIBRETTO-001

A Study of Selpercatinib (LOXO-292) in Participants With Advanced Solid Tumors, RET Fusion-Positive Solid Tumors, and Medullary Thyroid Cancer (LIBRETTO-001)

Phase 1/2NCT03157128

Population
Advanced RET fusion-positive NSCLC: previously treated with platinum-based chemotherapy, and previously untreated
Design
Phase 1-2 trial of selpercatinib; the NSCLC report enrolled patients with advanced RET fusion-positive NSCLC previously treated with platinum-based chemotherapy and, separately, previously untreated patients. Primary endpoint objective response by independent review committee. Data cutoff 16 December 2019 per the full text.
Primary endpoint
ORR
Arms
  • Selpercatinib (experimental): Selpercatinib 160 mg orally twice daily (recommended phase 2 dose, per the full text)
People
Drilon A
Show the numbers
Results reported for LIBRETTO-001
EndpointAnalysisSelpercatinibControlEffect
ORRFirst 105 consecutively enrolled patients previously treated with at least platinum-based chemotherapyComplete or partial response determined by an independent review committeeprimarymedian follow-up 12.1 monthsDrilon 202064% (54–73%)—63% of responses were ongoing at a median follow-up of 12.1 months. 105 patients.
DORPreviously treated cohort (105 patients)primarymedian follow-up 12.1 monthsDrilon 2020median 17.5 months—95% CI 12.0 to could not be evaluated.
ORRPreviously untreated patients (n=39)primaryDrilon 202085% (70–94%)—39 patients; 90% of responses were ongoing at 6 months.
ORRPatients with measurable CNS metastasis at enrolment by independent review (n=11; 38 of the 105 previously treated patients had CNS metastasis at baseline)Objective intracranial responseexploratoryDrilon 202091% (59–100%)—The paper lists objective intracranial response as a secondary end point; 'exploratory' is used as the closest label in the schema's analysis vocabulary (which has no 'secondary').
Results reported for LIBRETTO-001
EndpointAnalysisSelpercatinibControlEffect
ORRFirst 105 consecutively enrolled patients previously treated with at least platinum-based chemotherapyComplete or partial response determined by an independent review committeeprimarymedian follow-up 12.1 monthsDrilon 202064% (54–73%)—63% of responses were ongoing at a median follow-up of 12.1 months. 105 patients.
DORPreviously treated cohort (105 patients)primarymedian follow-up 12.1 monthsDrilon 2020median 17.5 months—95% CI 12.0 to could not be evaluated.
ORRPreviously untreated patients (n=39)primaryDrilon 202085% (70–94%)—39 patients; 90% of responses were ongoing at 6 months.
ORRPatients with measurable CNS metastasis at enrolment by independent review (n=11; 38 of the 105 previously treated patients had CNS metastasis at baseline)Objective intracranial responseexploratoryDrilon 202091% (59–100%)—The paper lists objective intracranial response as a secondary end point; 'exploratory' is used as the closest label in the schema's analysis vocabulary (which has no 'secondary').
Illustrative — modelled from reported values, not trial data. DOR, primary.
0%25%50%75%100%010203040Months (modelled)
  • Selpercatinib (experimental)

Median-only curves assume a constant hazard (exponential) and fit plateaus, as seen with some immunotherapies, poorly.

Data table (modelled values)
MonthsSelpercatinib
0100.0%
1164.7%
2241.8%
3327.1%
4417.5%
Primary publication: Drilon A, Oxnard GR, Tan DSW, Loong HHF, Johnson M, Gainor J, et al. Efficacy of Selpercatinib in RET Fusion-Positive Non-Small-Cell Lung Cancer. N Engl J Med. 2020;383(9):813-824. PubMedDOI

Appears in

Sources

  1. Drilon A, Oxnard GR, Tan DSW, Loong HHF, Johnson M, Gainor J, et al. Efficacy of Selpercatinib in RET Fusion-Positive Non-Small-Cell Lung Cancer. N Engl J Med. 2020;383(9):813-824.

    PubMedDOI