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NCT03037385 · OUTCOME

Phase 2: Intracranial CBR in RET-fusion Positive NSCLC CNS Metastases Participants

Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors · Source last updated 2025-05-31

Results reported by the registry submitting party. Population, time horizon, units and missing values must be read together.

Measure
NUMBER
Unit
percentage of participants
Interval / dispersion
95% Confidence Interval
Time frame
Up to approximately 79.8 months

What was measured

CBR=percentage of participants with CR/PR/SD which has been lasting at least 16 weeks (i.e. 4 cycles if 28 days are in 1 cycle) from first dose date. CR=disappearance of all target CNS/brain lesion, including lesions in brain stem \&/ cerebellum identified at baseline\&disappearance of all non-target CNS/brain lesion at baseline \& no identification of new CNS/brain lesion. PR=at least a 30% decrease in SOD of any CNS/brain lesion, identified as target lesions at baseline \& if in absence of unequivocal progression of any non-target CNS/brain lesion at baseline, or identification of new CNS/brain lesion. SD=neither sufficient shrinkage for PR nor sufficient increase for PD for target/non-target CNS/brain lesion, taking as reference smallest SOD while on study. PD=either at least 20% increase in SOD of target CNS/brain lesion, taking as reference smallest sum on study. Unequivocal progression of any CNS/brain lesion nontarget lesions at baseline, or identification of new CNS/brain lesion.

Analysis population: RET-altered measurable disease population included participants in the efficacy population who have measurable (target) disease per RECIST v1.1, at baseline according to BICR and sufficient evidence of a RET alteration. As specified in the SAP, CBR was to be assessed in RET-fusion CNS metastases sub-population (participants with CNS metastases) only. Hence only NSCLC arms are presented here. Phase 1 participants treated at 400 mg QD are pooled with Phase 2 participants for efficacy analysis.

Groups in this outcome

RET- Fusion Positive NSCLC With Prior Platinum Treatment

Participants who had RET-fusion positive NSCLC previously treated with platinum-based chemotherapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.

RET-fusion Positive NSCLC With Non-platinum Systemic Treatment

Participants who had RET-fusion positive NSCLC previously treated with non-platinum or systemic therapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.

RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment

Participants who had RET-fusion positive NSCLC not previously treated with systemic or platinum-based chemotherapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.

Group identifiers and denominators belong to this outcome only. They may differ in another outcome or in safety reporting.

Analysis denominator · Participants

GroupSource count
RET- Fusion Positive NSCLC With Prior Platinum Treatment13
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment1
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment1

Reported measurements

Source class 1
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET- Fusion Positive NSCLC With Prior Platinum Treatment61.5Not reported31.686.1Not reported
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment100Not reported2.5100Not reported
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment0Not reportedNANAThe 95% CI was not estimable due to insufficient number of participants with events.
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        31.6
        upper Limit
        86.1
        value
        61.5
      2. group Id
        OG001
        lower Limit
        2.5
        upper Limit
        100
        value
        100
      3. comment
        The 95% CI was not estimable due to insufficient number of participants with events.
        group Id
        OG002
        lower Limit
        NA
        upper Limit
        NA
        value
        0
denoms
  1. counts
    1. group Id
      OG000
      value
      13
    2. group Id
      OG001
      value
      1
    3. group Id
      OG002
      value
      1
    units
    Participants
description
CBR=percentage of participants with CR/PR/SD which has been lasting at least 16 weeks (i.e. 4 cycles if 28 days are in 1 cycle) from first dose date. CR=disappearance of all target CNS/brain lesion, including lesions in brain stem \&/ cerebellum identified at baseline\&disappearance of all non-target CNS/brain lesion at baseline \& no identification of new CNS/brain lesion. PR=at least a 30% decrease in SOD of any CNS/brain lesion, identified as target lesions at baseline \& if in absence of unequivocal progression of any non-target CNS/brain lesion at baseline, or identification of new CNS/brain lesion. SD=neither sufficient shrinkage for PR nor sufficient increase for PD for target/non-target CNS/brain lesion, taking as reference smallest SOD while on study. PD=either at least 20% increase in SOD of target CNS/brain lesion, taking as reference smallest sum on study. Unequivocal progression of any CNS/brain lesion nontarget lesions at baseline, or identification of new CNS/brain lesion.
dispersion Type
95% Confidence Interval
groups
  1. description
    Participants who had RET-fusion positive NSCLC previously treated with platinum-based chemotherapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
    id
    OG000
    title
    RET- Fusion Positive NSCLC With Prior Platinum Treatment
  2. description
    Participants who had RET-fusion positive NSCLC previously treated with non-platinum or systemic therapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
    id
    OG001
    title
    RET-fusion Positive NSCLC With Non-platinum Systemic Treatment
  3. description
    Participants who had RET-fusion positive NSCLC not previously treated with systemic or platinum-based chemotherapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
    id
    OG002
    title
    RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment
param Type
NUMBER
population Description
RET-altered measurable disease population included participants in the efficacy population who have measurable (target) disease per RECIST v1.1, at baseline according to BICR and sufficient evidence of a RET alteration. As specified in the SAP, CBR was to be assessed in RET-fusion CNS metastases sub-population (participants with CNS metastases) only. Hence only NSCLC arms are presented here. Phase 1 participants treated at 400 mg QD are pooled with Phase 2 participants for efficacy analysis.
reporting Status
POSTED
time Frame
Up to approximately 79.8 months
title
Phase 2: Intracranial CBR in RET-fusion Positive NSCLC CNS Metastases Participants
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice