NCT03037385 · OUTCOME
Phase 1 and Phase 2: DCR 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 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. 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 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
| Group | Source count |
|---|
| RET-fusion Positive TC | 27 |
|---|
Reported measurements
KIF5B
Analysis denominator · Participants
| Group | Source count |
|---|
| RET-fusion Positive TC | 0 |
|---|
CCDC6
Analysis denominator · Participants
| Group | Source count |
|---|
| RET-fusion Positive TC | 17 |
|---|
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| RET-fusion Positive TC | 94.1 | Not reported | 71.3 | 99.9 | Not reported |
|---|
NCOA4
Analysis denominator · Participants
| Group | Source count |
|---|
| RET-fusion Positive TC | 6 |
|---|
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| RET-fusion Positive TC | 100 | Not reported | 54.1 | 100 | Not reported |
|---|
Other
Analysis denominator · Participants
| Group | Source count |
|---|
| RET-fusion Positive TC | 4 |
|---|
Values in percentage of participants · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| RET-fusion Positive TC | 100 | Not reported | 39.8 | 100 | Not reported |
|---|
Complete source fields
- classes
- title
- KIF5B
- categories
- measurements
- group Id
- OG000
- lower Limit
- 71.3
- upper Limit
- 99.9
- value
- 94.1
- title
- CCDC6
- categories
- measurements
- group Id
- OG000
- lower Limit
- 54.1
- upper Limit
- 100
- value
- 100
- title
- NCOA4
- categories
- measurements
- group Id
- OG000
- lower Limit
- 39.8
- upper Limit
- 100
- value
- 100
- title
- Other
- description
- Oncogenic RET activation has been implicated as a driver in both MTC and 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. 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
- 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: DCR 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
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