Skip to content
Triangle logo TriangleGet started
← Full study record

NCT03037385 · OUTCOME

Phase 1 and Phase 2: Disease Control Rate (DCR) 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., KIF5B, CCDC6, NCOA4). RET genotypes were determined by local testing and/or central analysis of ctDNA. DCR was assessed in participants having specific RET rearrangements. DCR was defined as the percentage of participants with a confirmed CR/PR, or SD. CR and PR were defined per RECIST as outlined in the description for OM 3. SD was defined as neither sufficient shrinkage to qualify for CR or PR nor sufficient increase to qualify for PD. PD was defined as at least a 20% increase in SOD of target lesions, taking as reference the smallest SOD on study (including baseline).

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 Treatment93.4Not reported86.297.5Not reported
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment94.1Not reported71.399.9Not reported
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment88.2Not reported78.794.4Not 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 Treatment88.0Not reported68.897.5Not reported
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment100Not reported39.8100Not reported
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment89.5Not reported66.998.7Not 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 Treatment84.6Not reported54.698.1Not reported
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment50.0Not reported1.398.7Not reported
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment100Not reported71.5100Not reported
Complete source fields
classes
  1. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        86.2
        upper Limit
        97.5
        value
        93.4
      2. group Id
        OG001
        lower Limit
        71.3
        upper Limit
        99.9
        value
        94.1
      3. group Id
        OG002
        lower Limit
        78.7
        upper Limit
        94.4
        value
        88.2
    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
        68.8
        upper Limit
        97.5
        value
        88.0
      2. group Id
        OG001
        lower Limit
        39.8
        upper Limit
        100
        value
        100
      3. group Id
        OG002
        lower Limit
        66.9
        upper Limit
        98.7
        value
        89.5
    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
        54.6
        upper Limit
        98.1
        value
        84.6
      2. group Id
        OG001
        lower Limit
        1.3
        upper Limit
        98.7
        value
        50.0
      3. group Id
        OG002
        lower Limit
        71.5
        upper Limit
        100
        value
        100
    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., KIF5B, CCDC6, NCOA4). RET genotypes were determined by local testing and/or central analysis of ctDNA. DCR was assessed in participants having specific RET rearrangements. DCR was defined as the percentage of participants with a confirmed CR/PR, or SD. CR and PR were defined per RECIST as outlined in the description for OM 3. SD was defined as neither sufficient shrinkage to qualify for CR or PR nor sufficient increase to qualify for PD. PD was defined as at least a 20% increase in SOD of target lesions, taking as reference the smallest SOD on study (including baseline).
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: Disease Control Rate (DCR) in RET-fusion Positive NSCLC Participants With Specific RET Gene Status
type
SECONDARY
unit Of Measure
percentage of participants

Download exact source JSON → · Snapshot ctgov-results-191f4516d4760045304b24a0

Preserved source evidence · Not medical advice