NCT03037385 · OUTCOME

Phase 2: Intracranial DOR 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: MEDIAN
Unit: months
Interval / dispersion: 95% Confidence Interval
Time frame: Up to approximately 79.8 months

What was measured

DOR=time from first documented CR/PR to the date of first documented PD/death due to any cause, whichever occurs first. 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 RECIST 1.1 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. 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. DOR was analyzed using the KM methods.

Analysis population: RET-altered measurable disease population=participants in efficacy population who have measurable (target) disease per RECIST v1.1, at baseline according to BICR\&sufficient evidence of a RET alteration. As specified in SAP, DOR was to be assessed in RET-fusion CNS metastases sub-population only. Hence only NSCLC arms are presented here. Participants with a CR/PR were assessed for this OM. 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	7
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment	1
RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment	0

Reported measurements

Source class 1

Values in months · Interval/dispersion: 95% Confidence Interval
Group	Value	Spread	Lower	Upper	Comment
RET- Fusion Positive NSCLC With Prior Platinum Treatment	28.3	Not reported	10.5	NA	The upper limit of 95% CI was not estimable due to insufficient number of participants with events.
RET-fusion Positive NSCLC With Non-platinum Systemic Treatment	11.3	Not reported	NA	NA	The 95% CI was not estimable due to insufficient number of participants with events.

Complete source fields

classes

categories
measurements
comment
The upper limit of 95% CI was not estimable due to insufficient number of participants with events.
group Id
OG000
lower Limit
10.5
upper Limit
NA
value
28.3
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
11.3

denoms

counts
group Id
OG000
value
7
group Id
OG001
value
1
group Id
OG002
value
0
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

MEDIAN

population Description

RET-altered measurable disease population=participants in efficacy population who have measurable (target) disease per RECIST v1.1, at baseline according to BICR\&sufficient evidence of a RET alteration. As specified in SAP, DOR was to be assessed in RET-fusion CNS metastases sub-population only. Hence only NSCLC arms are presented here. Participants with a CR/PR were assessed for this OM. 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 DOR in RET-fusion Positive NSCLC CNS Metastases Participants

type

SECONDARY

unit Of Measure

months

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Preserved source evidence · Not medical advice