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NCT03037385 · OUTCOME

Phase 1 and Phase 2: ORR in RET-fusion Positive TC 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 activation has been implicated as a driver in both MTC and differentiated TC (DTC). 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. 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 TC

Participants who had RET-fusion positive TC 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 TC27

Reported measurements

KIF5B

Analysis denominator · Participants

GroupSource count
RET-fusion Positive TC0
CCDC6

Analysis denominator · Participants

GroupSource count
RET-fusion Positive TC17
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET-fusion Positive TC82.4Not reported56.696.2Not reported
NCOA4

Analysis denominator · Participants

GroupSource count
RET-fusion Positive TC6
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET-fusion Positive TC100Not reported54.1100Not reported
Other

Analysis denominator · Participants

GroupSource count
RET-fusion Positive TC4
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
GroupValueSpreadLowerUpperComment
RET-fusion Positive TC75.0Not reported19.499.4Not reported
Complete source fields
classes
  1. denoms
    1. counts
      1. group Id
        OG000
        value
        0
      units
      Participants
    title
    KIF5B
  2. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        56.6
        upper Limit
        96.2
        value
        82.4
    denoms
    1. counts
      1. group Id
        OG000
        value
        17
      units
      Participants
    title
    CCDC6
  3. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        54.1
        upper Limit
        100
        value
        100
    denoms
    1. counts
      1. group Id
        OG000
        value
        6
      units
      Participants
    title
    NCOA4
  4. categories
    1. measurements
      1. group Id
        OG000
        lower Limit
        19.4
        upper Limit
        99.4
        value
        75.0
    denoms
    1. counts
      1. group Id
        OG000
        value
        4
      units
      Participants
    title
    Other
denoms
  1. counts
    1. group Id
      OG000
      value
      27
    units
    Participants
description
Oncogenic RET activation has been implicated as a driver in both MTC and differentiated TC (DTC). 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. 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 TC received pralsetinib 400 mg, orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
    id
    OG000
    title
    RET-fusion Positive TC
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 TC 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