NCT03037385 · OUTCOME
Phase 1 and Phase 2: DOR in RET-mutation MTC 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
- MEDIAN
- Unit
- months
- 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 MTC. 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., M918T, cysteine rich domain, V804X). RET genotypes were determined by local testing and/or central analysis of ctDNA. DOR was assessed in participants having specific RET rearrangements. DOR=time from first documented CR/PR to date of first documented PD or death due to any cause, whichever occurs first. Per RECIST, CR= disappearance of all target lesions or any pathological lymph nodes (target or non-target) having a reduction in the short axis to \<10 mm. PR=at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in absence of CR. PD=as at least a 20% increase in SOD of target lesions, taking as reference smallest SOD on study (including baseline). DOR was analyzed using the Kaplan-Meier (KM) method.
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 \& sufficient evidence of a RET alteration. As prespecified in SAP, Phase 1 participants treated at 400 mg QD are pooled with Phase 2 participants for efficacy analysis. Participants who had a response of CR/PR were analyzed in this outcome measure. Number analyzed is the number of participants with the specified mutation.
Groups in this outcome
RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment
Participants who had RET- mutation positive MTC previously treated with either cabozantinib and/or vandetanib therapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment
Participants who had RET- mutation positive MTC not previously treated with cabozantinib or vandetanib therapies 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-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | 34 |
|---|
| RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment | 56 |
|---|
Reported measurements
M918T
Analysis denominator · Participants
| Group | Source count |
|---|
| RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | 22 |
|---|
| RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment | 32 |
|---|
Values in months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | 18.4 | Not reported | 15.1 | 25.8 | Not reported |
|---|
| RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment | 51.8 | Not reported | 40.0 | NA | The upper limit of 95% CI was not estimable due to insufficient number of participants with events. |
|---|
Cysteine Rich Domain
Analysis denominator · Participants
| Group | Source count |
|---|
| RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | 6 |
|---|
| RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment | 22 |
|---|
Values in months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | 29.5 | Not reported | 8.8 | NA | The upper limit of 95% CI was not estimable due to insufficient number of participants with events. |
|---|
| RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment | 36.8 | Not reported | 23.1 | NA | The upper limit of 95% CI was not estimable due to insufficient number of participants with events. |
|---|
V804X
Analysis denominator · Participants
| Group | Source count |
|---|
| RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | 2 |
|---|
| RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment | 0 |
|---|
Values in months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | 21.8 | Not reported | 14.2 | 29.4 | Not reported |
|---|
Other
Analysis denominator · Participants
| Group | Source count |
|---|
| RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | 4 |
|---|
| RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment | 2 |
|---|
Values in months · Interval/dispersion: 95% Confidence Interval| Group | Value | Spread | Lower | Upper | Comment |
|---|
| RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment | NA | Not reported | 14.5 | NA | The median and upper limit of 95% CI was not estimable due to insufficient number of participants with events. |
|---|
| RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment | NA | Not reported | NA | NA | The median and 95% CI was not estimable due to insufficient number of participants with events. |
|---|
Complete source fields
- classes
- categories
- measurements
- group Id
- OG000
- lower Limit
- 15.1
- upper Limit
- 25.8
- value
- 18.4
- comment
- The upper limit of 95% CI was not estimable due to insufficient number of participants with events.
- group Id
- OG001
- lower Limit
- 40.0
- upper Limit
- NA
- value
- 51.8
- denoms
- counts
- group Id
- OG000
- value
- 22
- group Id
- OG001
- value
- 32
- units
- Participants
- title
- M918T
- 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
- 8.8
- upper Limit
- NA
- value
- 29.5
- comment
- The upper limit of 95% CI was not estimable due to insufficient number of participants with events.
- group Id
- OG001
- lower Limit
- 23.1
- upper Limit
- NA
- value
- 36.8
- denoms
- counts
- group Id
- OG000
- value
- 6
- group Id
- OG001
- value
- 22
- units
- Participants
- title
- Cysteine Rich Domain
- categories
- measurements
- group Id
- OG000
- lower Limit
- 14.2
- upper Limit
- 29.4
- value
- 21.8
- denoms
- counts
- group Id
- OG000
- value
- 2
- group Id
- OG001
- value
- 0
- units
- Participants
- title
- V804X
- categories
- measurements
- comment
- The median and upper limit of 95% CI was not estimable due to insufficient number of participants with events.
- group Id
- OG000
- lower Limit
- 14.5
- upper Limit
- NA
- value
- NA
- comment
- The median and 95% CI was not estimable due to insufficient number of participants with events.
- group Id
- OG001
- lower Limit
- NA
- upper Limit
- NA
- value
- NA
- denoms
- counts
- group Id
- OG000
- value
- 4
- group Id
- OG001
- value
- 2
- units
- Participants
- title
- Other
- denoms
- counts
- group Id
- OG000
- value
- 34
- group Id
- OG001
- value
- 56
- units
- Participants
- description
- Oncogenic RET activation has been implicated as a driver in MTC. 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., M918T, cysteine rich domain, V804X). RET genotypes were determined by local testing and/or central analysis of ctDNA. DOR was assessed in participants having specific RET rearrangements. DOR=time from first documented CR/PR to date of first documented PD or death due to any cause, whichever occurs first. Per RECIST, CR= disappearance of all target lesions or any pathological lymph nodes (target or non-target) having a reduction in the short axis to \<10 mm. PR=at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in absence of CR. PD=as at least a 20% increase in SOD of target lesions, taking as reference smallest SOD on study (including baseline). DOR was analyzed using the Kaplan-Meier (KM) method.
- dispersion Type
- 95% Confidence Interval
- groups
- description
- Participants who had RET- mutation positive MTC previously treated with either cabozantinib and/or vandetanib therapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
- id
- OG000
- title
- RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment
- description
- Participants who had RET- mutation positive MTC not previously treated with cabozantinib or vandetanib therapies received pralsetinib, 400 mg orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
- id
- OG001
- title
- RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment
- param Type
- MEDIAN
- 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 \& sufficient evidence of a RET alteration. As prespecified in SAP, Phase 1 participants treated at 400 mg QD are pooled with Phase 2 participants for efficacy analysis. Participants who had a response of CR/PR were analyzed in this outcome measure. 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: DOR in RET-mutation MTC Participants With Specific RET Gene Status
- type
- SECONDARY
- unit Of Measure
- months
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