NCT03037385 · OUTCOME

Phase 2: Intracranial DCR 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

DCR=percentage of participants with a confirmed CR/PR, or SD. CR=disappearance of all target CNS/brain lesion, including lesions in brain stem \&/or 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 the SOD of any CNS/brain lesion, identified as RECIST 1.1 target lesions at baseline \& if in the absence of unequivocal progression of any non-target CNS/brain lesion at baseline, or the identification of new CNS/brain lesion. SD=neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD for target/non-target CNS/brain lesion, taking as reference the 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, DCR 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

Group	Source count
RET- Fusion Positive NSCLC With Prior Platinum Treatment	13
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment	1
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment	1

Reported measurements

Source class 1

Values in percentage of participants · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
RET- Fusion Positive NSCLC With Prior Platinum Treatment	76.9	Not reported	46.2	95.0	Not reported
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment	100	Not reported	2.5	100	Not reported
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment	0	Not reported	NA	NA	The 95% CI was not estimable due to insufficient number of participants with events.

Complete source fields

classes

categories
measurements
group Id
OG000
lower Limit
46.2
upper Limit
95.0
value
76.9
group Id
OG001
lower Limit
2.5
upper Limit
100
value
100
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

counts
group Id
OG000
value
13
group Id
OG001
value
1
group Id
OG002
value
1
units
Participants

description

dispersion Type

95% Confidence Interval

groups

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
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
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, DCR 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 DCR in RET-fusion Positive NSCLC CNS Metastases Participants

type

SECONDARY

unit Of Measure

percentage of participants

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

Preserved source evidence · Not medical advice