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NCT03037385 · OUTCOME

Phase 1 and Phase 2: ORR in RET-fusion Positive NSCLC Participants With Specific RET Gene Status

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

Oncogenic RET rearrangements have been identified in 1%-2% of NSCLC. These rearrangements typically produce chimeric transcripts that encode a fusion protein consisting of the RET kinase domain coupled to a protein with a dimerization domain (e.g., Kinesin family member 5B (KIF5B), coiled-coil domain containing 6 (CCDC6), nuclear receptor coactivator 4 (NCOA4)). RET genotypes were determined by local testing and/or central analysis of circulating tumor deoxyribonucleic acid (ctDNA). ORR was assessed in participants having specific RET rearrangements. ORR was defined as the percentage of participants with a confirmed CR or PR for at least 2 assessments with at least 28 days apart and no PD in between. CR, PR, and PD were defined per RECIST as outlined in the description for OM 3.

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 prespecified in the SAP, Phase 1 participants treated at 400 mg QD are pooled with Phase 2 participants for efficacy analysis. Number analyzed is the number of participants with the specified mutation.

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 Treatment130
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment23
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment106

Reported measurements

KIF5B

Analysis denominator · Participants

GroupSource count
RET- Fusion Positive NSCLC With Prior Platinum Treatment91
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment17
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment76
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET- Fusion Positive NSCLC With Prior Platinum Treatment63.7Not reported53.073.6Not reported
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment82.4Not reported56.696.2Not reported
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment78.9Not reported68.187.5Not reported
CCDC6

Analysis denominator · Participants

GroupSource count
RET- Fusion Positive NSCLC With Prior Platinum Treatment25
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment4
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment19
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET- Fusion Positive NSCLC With Prior Platinum Treatment68.0Not reported46.585.1Not reported
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment75.0Not reported19.499.4Not reported
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment84.2Not reported60.496.6Not reported
NCOA4

Analysis denominator · Participants

GroupSource count
RET- Fusion Positive NSCLC With Prior Platinum Treatment1
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment0
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment0
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET- Fusion Positive NSCLC With Prior Platinum Treatment100Not reported2.5100Not reported
Other

Analysis denominator · Participants

GroupSource count
RET- Fusion Positive NSCLC With Prior Platinum Treatment13
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment2
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment11
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET- Fusion Positive NSCLC With Prior Platinum Treatment46.2Not reported19.274.9Not reported
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment0Not reportedNANAThe 95% CI was not estimable due to insufficient number of participants with events.
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment63.6Not reported30.889.1Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        53.0
        upper Limit
        73.6
        value
        63.7
      2. group Id
        OG001
        lower Limit
        56.6
        upper Limit
        96.2
        value
        82.4
      3. group Id
        OG002
        lower Limit
        68.1
        upper Limit
        87.5
        value
        78.9
    denoms
    1. counts
      1. group Id
        OG000
        value
        91
      2. group Id
        OG001
        value
        17
      3. group Id
        OG002
        value
        76
      units
      Participants
    title
    KIF5B
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        46.5
        upper Limit
        85.1
        value
        68.0
      2. group Id
        OG001
        lower Limit
        19.4
        upper Limit
        99.4
        value
        75.0
      3. group Id
        OG002
        lower Limit
        60.4
        upper Limit
        96.6
        value
        84.2
    denoms
    1. counts
      1. group Id
        OG000
        value
        25
      2. group Id
        OG001
        value
        4
      3. group Id
        OG002
        value
        19
      units
      Participants
    title
    CCDC6
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        2.5
        upper Limit
        100
        value
        100
    denoms
    1. counts
      1. group Id
        OG000
        value
        1
      2. group Id
        OG001
        value
        0
      3. group Id
        OG002
        value
        0
      units
      Participants
    title
    NCOA4
  4. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        19.2
        upper Limit
        74.9
        value
        46.2
      2. comment
        The 95% CI was not estimable due to insufficient number of participants with events.
        group Id
        OG001
        lower Limit
        NA
        upper Limit
        NA
        value
        0
      3. group Id
        OG002
        lower Limit
        30.8
        upper Limit
        89.1
        value
        63.6
    denoms
    1. counts
      1. group Id
        OG000
        value
        13
      2. group Id
        OG001
        value
        2
      3. group Id
        OG002
        value
        11
      units
      Participants
    title
    Other
denoms
  1. counts
    1. group Id
      OG000
      value
      130
    2. group Id
      OG001
      value
      23
    3. group Id
      OG002
      value
      106
    units
    Participants
description
Oncogenic RET rearrangements have been identified in 1%-2% of NSCLC. These rearrangements typically produce chimeric transcripts that encode a fusion protein consisting of the RET kinase domain coupled to a protein with a dimerization domain (e.g., Kinesin family member 5B (KIF5B), coiled-coil domain containing 6 (CCDC6), nuclear receptor coactivator 4 (NCOA4)). RET genotypes were determined by local testing and/or central analysis of circulating tumor deoxyribonucleic acid (ctDNA). ORR was assessed in participants having specific RET rearrangements. ORR was defined as the percentage of participants with a confirmed CR or PR for at least 2 assessments with at least 28 days apart and no PD in between. CR, PR, and PD were defined per RECIST as outlined in the description for OM 3.
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 prespecified in the SAP, Phase 1 participants treated at 400 mg QD are pooled with Phase 2 participants for efficacy analysis. Number analyzed is the number of participants with the specified mutation.
reporting Status
POSTED
time Frame
Up to approximately 79.8 months
title
Phase 1 and Phase 2: ORR in RET-fusion Positive NSCLC Participants With Specific RET Gene Status
type
SECONDARY
unit Of Measure
percentage of participants

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Preserved source evidence · Not medical advice