TriangleNCT03037385 · STUDY IN CONTEXT
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
Draft scope assessment: narrower. Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm. Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
Read the full study record and all posted results →
Linked outcomes
MTD was defined as the highest tolerated dose of pralsetinib without causing dose limiting toxicities (DLTs). DLT was defined as any Grade ≥3 adverse event (AE) occurring during Cycle 1 during Phase 1 (dose escalation) that is not clearly caused by something other than pralsetinib. RP2D was defined as the highest dose with acceptable toxicity as determined from dose-escalation phase.
An AE was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. A SAE is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above.
ORR was defined as the percentage of participants with a confirmed complete response (CR) or partial response (PR) for at least two assessments with at least 28 days apart and no disease progression (PD) in between. Per Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1), CR was defined as the disappearance of all target lesions or any pathological lymph nodes (whether target or non-target) having a reduction in the short axis to \<10 millimeters (mm). PR was defined as at least a 30% decrease in the sum of diameters (SOD) of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in SOD of target lesions, taking as reference the smallest SOD on study (including baseline). ORR and its two-sided 95% CI, based on the exact binomial distribution (Clopper-Pearson), were presented.
ORR was defined as the percentage of participants with a confirmed CR or PR for at least two assessments with at least 28 days apart and no PD in between. Per RECIST v1.1, CR was defined as the disappearance of all target lesions or any pathological lymph nodes (whether target or non-target) having a reduction in the short axis to \<10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in SOD of target lesions, taking as reference the smallest SOD on study (including baseline). ORR and its two-sided 95% CI, based on the exact binomial distribution (Clopper-Pearson), was presented.
Oncogenic RET rearrangements have been identified in 1%-2% of NSCLC. 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., Kinesin family member 5B (KIF5B), coiled-coil domain containing 6 (CCDC6), nuclear receptor coactivator 4 (NCOA4)). RET genotypes were determined by local testing and/or central analysis of circulating tumor deoxyribonucleic acid (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.
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. 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.
Reported safety
Quality of life and patient-reported measures
No linked quality-of-life measure is recorded for this setting and study.
Full draft applicability and source-link record
- trial
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- trial label
- 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
- applicability
- narrower
- applicability note
- Linked by condition-string match at the cancer-entity level. The study's eligibility - stage, biomarker, treatment line and prior therapy - has not been reconciled against this page's definition, so its population is narrower than this page in ways not yet recorded. The condition was transformed or resolved through the terminology hierarchy to reach this cancer type, so the link itself is a candidate a reviewer must confirm.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- provenance class
- regulator-cited
- discovery note
- Found because this study's registry identifier is printed in an FDA approval notice, an FDA product label or an NCI summary held by this site. The citing document and the exact position of the identifier inside it are recorded. Being cited is not a statement that the study supports this treatment.
- outcome Count
- 6
- harms Count
- 4
- qol Count
- 0
- outcomes
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0000
- source section id
- NCT03037385-om-0000
- source outcome index
- 0
- endpoint
- Phase 1 : Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of Pralsetinib
- endpoint definition
- MTD was defined as the highest tolerated dose of pralsetinib without causing dose limiting toxicities (DLTs). DLT was defined as any Grade ≥3 adverse event (AE) occurring during Cycle 1 during Phase 1 (dose escalation) that is not clearly caused by something other than pralsetinib. RP2D was defined as the highest dose with acceptable toxicity as determined from dose-escalation phase.
- time origin
- Up to approximately 30.8 months
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Dose-determining population included all participants in the dose-escalation part who have received ≥75% (21 days) of the study drug and completed safety evaluations through Cycle 1 Day 28 or experienced a DLT.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0001
- source section id
- NCT03037385-om-0001
- source outcome index
- 1
- endpoint
- Phase 1 and Phase 2: Number of Participants With AEs and Serious AEs (SAEs)
- endpoint definition
- An AE was any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An AE can be any unfavorable and unintended sign (e.g., an abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, without any judgment about causality. A SAE is any significant hazard, contraindication, side effect that is fatal or life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital anomaly or birth defect, is medically significant or requires intervention to prevent one or other of the outcomes listed above.
- time origin
- From Cycle 1 Day 1 up to 30 days after the final dose of study drug (up to approximately 6.7 years)
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Safety Population included all participants who have received at least 1 dose of the study drug regardless of starting dose levels. As pre-specified in the SAP, safety data was to be analyzed and reported as per the pre-planned grouped dose level II (SAP section 3.6.5.2). Hence, per dose safety data is not presented for this study.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0002
- source section id
- NCT03037385-om-0002
- source outcome index
- 2
- endpoint
- Phase 2: Overall Response Rate (ORR)
- endpoint definition
- ORR was defined as the percentage of participants with a confirmed complete response (CR) or partial response (PR) for at least two assessments with at least 28 days apart and no disease progression (PD) in between. Per Response Evaluation Criteria in Solid Tumors, Version 1.1 (RECIST v1.1), CR was defined as the disappearance of all target lesions or any pathological lymph nodes (whether target or non-target) having a reduction in the short axis to \<10 millimeters (mm). PR was defined as at least a 30% decrease in the sum of diameters (SOD) of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in SOD of target lesions, taking as reference the smallest SOD on study (including baseline). ORR and its two-sided 95% CI, based on the exact binomial distribution (Clopper-Pearson), were presented.
- time origin
- Up to approximately 79.8 months
- analysis role
- primary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- RET-altered measurable disease population included participants in the efficacy population who had measurable (target) disease per RECIST v1.1 at baseline according to blinded central review (BICR) and sufficient evidence of a RET alteration. As prespecified in the SAP, Phase 1 participants treated at 400 mg QD are also included in Phase 2 efficacy analysis.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0003
- source section id
- NCT03037385-om-0003
- source outcome index
- 3
- endpoint
- Phase 1: ORR
- endpoint definition
- ORR was defined as the percentage of participants with a confirmed CR or PR for at least two assessments with at least 28 days apart and no PD in between. Per RECIST v1.1, CR was defined as the disappearance of all target lesions or any pathological lymph nodes (whether target or non-target) having a reduction in the short axis to \<10 mm. PR was defined as at least a 30% decrease in the SOD of all target lesions, taking as reference the baseline SOD, in the absence of CR. PD was defined as at least a 20% increase in SOD of target lesions, taking as reference the smallest SOD on study (including baseline). ORR and its two-sided 95% CI, based on the exact binomial distribution (Clopper-Pearson), was presented.
- time origin
- Up to approximately 28 months
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- Efficacy population included all participants who have been exposed to at least one dose of the study drug on or prior to 11 July 2019.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0004
- source section id
- NCT03037385-om-0004
- source outcome index
- 4
- endpoint
- Phase 1 and Phase 2: ORR in RET-fusion Positive NSCLC Participants With Specific RET Gene Status
- endpoint definition
- Oncogenic RET rearrangements have been identified in 1%-2% of NSCLC. 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., Kinesin family member 5B (KIF5B), coiled-coil domain containing 6 (CCDC6), nuclear receptor coactivator 4 (NCOA4)). RET genotypes were determined by local testing and/or central analysis of circulating tumor deoxyribonucleic acid (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.
- time origin
- Up to approximately 79.8 months
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0005
- source section id
- NCT03037385-om-0005
- source outcome index
- 5
- endpoint
- Phase 1 and Phase 2: ORR in RET-mutation MTC Participants With Specific RET Gene Status
- endpoint definition
- 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. 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.
- time origin
- Up to approximately 79.8 months
- analysis role
- secondary
- reporting status
- POSTED
- data cutoff
- Source null
- population note
- 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.
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- arms
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0000
- arm key
- OG000
- arm label
- Phase 1: All Participants QD
- regimen
- Participants received pralsetinib at varying doses at a QD schedule until discontinuation due to toxicity, disease progression, or other reasons.
- analyzed count
- 51
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0000#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0001
- arm key
- OG000
- arm label
- Pralsetinib ≤ 300 mg QD
- regimen
- Participants received pralsetinib, 300 mg or less, orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
- analyzed count
- 37
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0001#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0001
- arm key
- OG001
- arm label
- Pralsetinib 400 mg QD
- regimen
- Participants received pralsetinib, 400 mg, orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
- analyzed count
- 540
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0001#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0001
- arm key
- OG002
- arm label
- Pralsetinib BID Dosing Schedule
- regimen
- Participants received pralsetinib, 100 mg, orally, BID or 200 mg in the morning and then 100 mg in the evening, orally, until discontinuation due to toxicity, disease progression, or other reasons.
- analyzed count
- 9
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0001#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0001
- arm key
- OG003
- arm label
- Pralsetinib All Doses
- regimen
- Participants received pralsetinib at varying doses at the QD and BID schedule until discontinuation due to toxicity, disease progression, or other reasons.
- analyzed count
- 590
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0001#groups/OG003
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0002
- arm key
- OG000
- arm label
- RET- Fusion Positive NSCLC With Prior Platinum Treatment:
- regimen
- 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.
- analyzed count
- 130
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0002#groups/OG000
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0002
- arm key
- OG001
- arm label
- RET-fusion Positive NSCLC With Non-platinum Systemic Treatment
- regimen
- 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.
- analyzed count
- 23
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0002#groups/OG001
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0002
- arm key
- OG002
- arm label
- RET-fusion Positive NSCLC With No Prior Systemic or Platinum Treatment
- regimen
- 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.
- analyzed count
- 106
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0002#groups/OG002
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0002
- arm key
- OG003
- arm label
- RET-mutation MTC With Prior Cabozantinib and/or Vandetanib Treatment
- regimen
- 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.
- analyzed count
- 60
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0002#groups/OG003
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0002
- arm key
- OG004
- arm label
- RET-mutation MTC With No Prior Cabozantinib or Vandetanib Treatment
- regimen
- 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.
- analyzed count
- 72
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0002#groups/OG004
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0002
- arm key
- OG005
- arm label
- RET-fusion Positive TC
- regimen
- Participants who had RET-fusion positive TC received pralsetinib, 400 mg, orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
- analyzed count
- 27
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0002#groups/OG005
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- outcome key
- om-0002
- arm key
- OG006
- arm label
- RET-fusion Positive Solid Tumors Other Than NSCLC and TC
- regimen
- Participants who had other RET-positive solid tumors other than NSCLC and TC received pralsetinib, 400 mg, orally, QD until discontinuation due to toxicity, disease progression, or other reasons.
- analyzed count
- 28
- analyzed unit
- Participants
- is comparator
- Source null
- source pointer
- NCT03037385-om-0002#groups/OG006
- harms
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- harm key
- EG000
- source section id
- NCT03037385-ae
- event group key
- EG000
- event group label
- Pralsetinib ≤ 300 mg QD
- serious affected
- 26
- serious at risk
- 37
- other affected
- 36
- other at risk
- 37
- frequency threshold
- 5
- collection period
- AEs and SAEs: From Cycle 1 Day 1 up to 30 days after the final dose of study drug (up to approximately 6.7 years) All-cause Mortality: Up to approximately 7 years Safety population included all participants who have received at least 1 dose of the study drug regardless of starting dose levels.
- event definition
- As pre-specified in SAP, safety data was to be analyzed \& reported per pre-planned grouped dose level II. Per dose level II, ≤ 300 mg QD arm included participants dosed with pralsetinib at 30 mg, 60 mg , 100 mg , 200 mg \& 300 mg QD; 400 mg arm included participants dosed at 400 mg QD, BID Dosing Schedule arm included participants dosed with 100/100 mg \& 200/100 mg BID, \& All doses included participants treated at all QD \& BID doses \& 600 mg QD dose. Hence, AEs cannot be presented per dose level.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- harm key
- EG001
- source section id
- NCT03037385-ae
- event group key
- EG001
- event group label
- Pralsetinib 400 mg QD
- serious affected
- 381
- serious at risk
- 540
- other affected
- 536
- other at risk
- 540
- frequency threshold
- 5
- collection period
- AEs and SAEs: From Cycle 1 Day 1 up to 30 days after the final dose of study drug (up to approximately 6.7 years) All-cause Mortality: Up to approximately 7 years Safety population included all participants who have received at least 1 dose of the study drug regardless of starting dose levels.
- event definition
- As pre-specified in SAP, safety data was to be analyzed \& reported per pre-planned grouped dose level II. Per dose level II, ≤ 300 mg QD arm included participants dosed with pralsetinib at 30 mg, 60 mg , 100 mg , 200 mg \& 300 mg QD; 400 mg arm included participants dosed at 400 mg QD, BID Dosing Schedule arm included participants dosed with 100/100 mg \& 200/100 mg BID, \& All doses included participants treated at all QD \& BID doses \& 600 mg QD dose. Hence, AEs cannot be presented per dose level.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- harm key
- EG002
- source section id
- NCT03037385-ae
- event group key
- EG002
- event group label
- Pralsetinib BID Dosing Schedule
- serious affected
- 7
- serious at risk
- 9
- other affected
- 9
- other at risk
- 9
- frequency threshold
- 5
- collection period
- AEs and SAEs: From Cycle 1 Day 1 up to 30 days after the final dose of study drug (up to approximately 6.7 years) All-cause Mortality: Up to approximately 7 years Safety population included all participants who have received at least 1 dose of the study drug regardless of starting dose levels.
- event definition
- As pre-specified in SAP, safety data was to be analyzed \& reported per pre-planned grouped dose level II. Per dose level II, ≤ 300 mg QD arm included participants dosed with pralsetinib at 30 mg, 60 mg , 100 mg , 200 mg \& 300 mg QD; 400 mg arm included participants dosed at 400 mg QD, BID Dosing Schedule arm included participants dosed with 100/100 mg \& 200/100 mg BID, \& All doses included participants treated at all QD \& BID doses \& 600 mg QD dose. Hence, AEs cannot be presented per dose level.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- release id
- clinical-indications-draft-1
- disease state id
- ds-primitive-neuroectodermal-tumor
- nct id
- NCT03037385
- harm key
- EG003
- source section id
- NCT03037385-ae
- event group key
- EG003
- event group label
- Pralsetinib All Doses
- serious affected
- 416
- serious at risk
- 590
- other affected
- 585
- other at risk
- 590
- frequency threshold
- 5
- collection period
- AEs and SAEs: From Cycle 1 Day 1 up to 30 days after the final dose of study drug (up to approximately 6.7 years) All-cause Mortality: Up to approximately 7 years Safety population included all participants who have received at least 1 dose of the study drug regardless of starting dose levels.
- event definition
- As pre-specified in SAP, safety data was to be analyzed \& reported per pre-planned grouped dose level II. Per dose level II, ≤ 300 mg QD arm included participants dosed with pralsetinib at 30 mg, 60 mg , 100 mg , 200 mg \& 300 mg QD; 400 mg arm included participants dosed at 400 mg QD, BID Dosing Schedule arm included participants dosed with 100/100 mg \& 200/100 mg BID, \& All doses included participants treated at all QD \& BID doses \& 600 mg QD dose. Hence, AEs cannot be presented per dose level.
- undenominated note
- Source null
- curation status
- draft
- reviewed by
- Source null
- reviewed at
- Source null
- created at
- 2026-09-10T00:00:00Z
- quality of life
- setting source links
Preserved source evidence · Not medical advice