This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for 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 (NCT03037385), a non-randomized phase 1, phase 2 study. The primary outcome is Phase 1 and Phase 2: Number of Participants With AEs and Serious AEs (SAEs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03037385 |
| Phase | Phase 1, Phase 2 |
| Condition | RET-altered Non Small Cell Lung Cancer, Medullary Thyroid Cancer, RET-altered Papillary Thyroid Cancer |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 590 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hoffmann-La Roche |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled participants with medullary thyroid cancer, RET-altered non-small cell lung cancer and other advanced solid tumors. The registry also lists RET-altered papillary thyroid cancer and RET-altered colon cancer among its conditions. The safety population was defined as participants who received at least 1 dose of study drug, regardless of starting dose level. The registry record does not report the detailed eligibility criteria or the number of participants in each cancer-specific efficacy population.
Intervention
The intervention was oral pralsetinib, also identified as BLU-667. Phase 1 used multiple doses in dose escalation. Phase 2 used an oral dose determined during dose escalation. The record does not report the detailed dose-escalation rules or the rationale for selecting the dose used in expansion.
Comparator
There was no randomized control group. The study was non-randomized and open-label, with parallel dose groups. The listed groups describe pralsetinib doses and schedules rather than a like-for-like comparison against another treatment or placebo. Differences between groups cannot therefore be interpreted as randomized treatment effects.
Primary question
The primary outcome asks how many participants experienced adverse events (AEs) and serious adverse events (SAEs) during treatment and follow-up. It is a safety question, not a primary test of antitumor activity. The registry separately lists response and disease-control outcomes as secondary outcomes for specified cancer and RET-status subgroups. The primary safety outcome timeframe runs from Cycle 1 Day 1 to 30 days after the final dose, up to approximately 6.7 years.
3. Trial Design
This was a Phase 1/2, first-in-human study of pralsetinib, described in the registry as a highly selective RET inhibitor. The study was open-label, non-randomized and organized in parallel groups. Its stated aims included evaluating safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary antineoplastic activity in participants with medullary thyroid cancer, RET-altered non-small cell lung cancer and other RET-altered solid tumors.
The Phase 1 portion was dose escalation, with multiple oral doses of pralsetinib. The Phase 2 portion was dose expansion, using an oral dose selected during dose escalation. The registry lists groups for Pralsetinib ≤ 300 mg QD, Pralsetinib 400 mg QD, a Pralsetinib BID dosing schedule, and all doses. Because allocation was non-randomized, these groups are not equivalent to randomized treatment arms. Differences in participants, dose selection, or study period may affect comparisons, and the record does not report a controlled comparison.
The registry reports enrollment of 590 and a completed status. The study start is recorded as 2017-03-17, with primary completion on 2024-03-21. Its primary outcome is the number of participants with AEs and SAEs, expressed as a percentage of participants. The registry also lists secondary outcomes including objective response rate, clinical benefit rate and disease control rate in selected cancer and RET-status subgroups. The record does not report the full protocol, detailed cohort allocation, or the statistical plan for those secondary outcomes.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: For Phase I, The number of participants in each arm within the participant flow reflects the arms they were originally assigned to, whereas numbers reported in the pharmacokinetics outcome measure (OM) represent the number based on actual treatment received by the participants. 1 participant assigned to the 200/100 mg arm received the 100/100 mg treatment. Hence, was counted under the 100/100 mg arm in the PK-evaluable population.
Only the first 6 of 10 groups are shown.
Overall Study
| Stage | Phase I: Pralsetinib 30 mg | Phase I: Pralsetinib 60 mg | Phase I: Pralsetinib 100 mg | Phase I: Pralsetinib 200 mg | Phase I: Pralsetinib 300 mg | Phase I: Pralsetinib 400 mg |
|---|---|---|---|---|---|---|
| STARTED | 2 | 6 | 5 | 13 | 11 | 12 |
| COMPLETED | 0 | 0 | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 2 | 6 | 5 | 13 | 11 | 12 |
Reasons for not completing this period:
| Reason | Phase I: Pralsetinib 30 mg | Phase I: Pralsetinib 60 mg | Phase I: Pralsetinib 100 mg | Phase I: Pralsetinib 200 mg | Phase I: Pralsetinib 300 mg | Phase I: Pralsetinib 400 mg |
|---|---|---|---|---|---|---|
| Adverse Event | 0 | 0 | 1 | 0 | 0 | 0 |
| Death | 1 | 4 | 2 | 2 | 5 | 2 |
| Initiation of Another Therapy | 1 | 0 | 0 | 0 | 0 | 0 |
| Lost to Follow-up | 0 | 0 | 0 | 0 | 0 | 1 |
| Physician Decision | 0 | 0 | 0 | 0 | 0 | 0 |
| Progressive Disease | 0 | 1 | 1 | 5 | 2 | 0 |
| Withdrawal by Subject | 0 | 0 | 1 | 2 | 1 | 3 |
| Reason Not Specified | 0 | 1 | 0 | 4 | 3 | 6 |
Analysis population for the primary outcome, as defined in the registry: 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.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
Population: Safety population included all participants who had received at least 1 dose of the study drug regardless of the starting dose levels.
Only the first 6 of 11 groups are shown.
| Characteristic | Unit | Phase I: Pralsetinib 30 mg | Phase I: Pralsetinib 60 mg | Phase I: Pralsetinib 100 mg | Phase I: Pralsetinib 200 mg | Phase I: Pralsetinib 300 mg | Phase I: Pralsetinib 400 mg |
|---|---|---|---|---|---|---|---|
| Participants analysed | 2 | 6 | 5 | 13 | 11 | 12 | |
| Age, Continuous | years | 54.5 (SD 9.2) | 49.5 (SD 18.0) | 51.2 (SD 13.2) | 53.8 (SD 13.6) | 60.6 (SD 11.0) | 49.2 (SD 17.8) |
| Sex: Female, Male: Female | Participants | 1 | 3 | 4 | 5 | 4 | 3 |
| Sex: Female, Male: Male | Participants | 1 | 3 | 1 | 8 | 7 | 9 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 | 2 | 1 | 1 | 0 | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 0 | 4 | 4 | 12 | 11 | 9 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Asian | Participants | 0 | 1 | 2 | 0 | 0 | 2 |
| Race/Ethnicity, Customized: Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Black or African American | Participants | 0 | 0 | 0 | 1 | 0 | 1 |
| Race/Ethnicity, Customized: White | Participants | 0 | 3 | 2 | 11 | 11 | 9 |
| Race/Ethnicity, Customized: Other | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Unknown or Not Reported | Participants | 2 | 2 | 1 | 1 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Phase 1 and Phase 2: Number of Participants With AEs and Serious AEs (SAEs), measured over From Cycle 1 Day 1 up to 30 days after the final dose of study drug (up to approximately 6.7 years).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants who experienced an adverse event or a serious adverse event. The registry defines an AE as an untoward medical occurrence associated with drug use, whether or not it is considered drug related. It may include an unfavorable sign, symptom or disease temporally associated with use of the drug, without a causal judgment. An SAE is an event meeting specified seriousness criteria, including being fatal or life-threatening, requiring or prolonging hospitalization, causing persistent or significant disability or incapacity, involving a congenital anomaly or birth defect, or being medically significant or requiring intervention to prevent one of those outcomes.
The endpoint is used to describe safety during exposure and follow-up. The registry’s safety population includes participants who received at least 1 dose, regardless of starting dose level. The timeframe begins at Cycle 1 Day 1 and extends to 30 days after the final dose, up to approximately 6.7 years. The outcome is reported as a percentage of participants, while the posted results also give counts and group denominators. An event being counted does not by itself establish that pralsetinib caused it; the AE definition explicitly does not require a judgment about causality.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted primary results are safety counts, not evidence from a randomized comparison. In the all-doses group, 416 of 590 participants had at least one SAE, and 585 of 590 had other AEs. The record lists the same 590 participants at risk for each of these outcomes. These figures describe the share of the safety population recorded with events over the specified observation period; they do not indicate how many events each participant experienced.
The dose-group results are also descriptive. For Pralsetinib ≤ 300 mg QD, the SAE count is 26 among 37 at risk, and the other-AE count is 36 among 37. For Pralsetinib 400 mg QD, the corresponding counts are 381 among 540 and 536 among 540. For the BID dosing schedule, they are 7 among 9 and 9 among 9. These groups were not randomized control arms. The registry record does not establish that their participants were comparable, so apparent differences across groups should not be read as dose effects.
The record lists deaths by group: 15 in the ≤ 300 mg QD group, 245 in the 400 mg QD group, 7 in the BID dosing group and 268 in the all-doses group. These are recorded counts; the record does not state that each death was attributed to treatment or was an SAE. Nor does it provide enough information to interpret differences in death counts across dose groups as causal.
Among the listed serious-event terms, Sepsis is reported as 33.3%, Pneumonia as 16.1%, and Anaemia, Cardiac arrest, Abdominal pain and Abdominal pain upper as 11.1% each. The denominator and counting convention for these term percentages are not reported in the registry record, so they should not be treated as percentages of all enrolled or all treated participants without further information.
The registry does not report confidence intervals or hypothesis-test results for this primary safety outcome. Therefore, the precision of the event proportions and the statistical uncertainty around comparisons cannot be assessed from the record. The findings describe the observed safety experience in this study population; they do not establish comparative safety or efficacy.
9. Statistical Methods Explained
What does a participant-level event percentage describe?
The primary outcome is a participant-level measure: whether a participant was recorded as having an AE or SAE during the defined timeframe. A participant with one or more events contributes to the count for that outcome; the measure is not a count of every event occurrence. The registry gives counts and denominators for each dose group and reports the outcome unit as a percentage of participants. This helps describe how common recorded events were among participants in the safety population, but it does not capture event duration, recurrence or severity beyond the separate SAE classification.
Why does the safety population matter?
The registry defines the safety population as participants who received at least 1 dose, regardless of starting dose level. This is an exposure-based population suited to describing safety observations after treatment begins. It is distinct from an analysis population for tumor response, which may use cancer-specific or biomarker-defined eligibility. The registry record does not report detailed analysis-population definitions for the secondary response outcomes. The stated safety timeframe starts on Cycle 1 Day 1 and ends 30 days after the final dose, up to approximately 6.7 years. Longer observation can allow more time for events to be recorded, so the time under observation is relevant when interpreting the counts.
Why are dose groups not treatment comparisons?
The study was non-randomized and open-label. Participants were assigned across dose-escalation and dose-expansion portions rather than randomized between comparable treatment groups. Dose selection during escalation can result in groups that differ in their characteristics, follow-up, or treatment exposure. Consequently, a higher or lower event count in one group does not isolate the effect of dose. A randomized control group and an appropriate analysis would be needed for a causal comparative claim; neither is described for this safety endpoint in the registry record.
What is the role of uncertainty estimates?
General statistical explanation: a confidence interval expresses the range of values reasonably compatible with an estimate under a specified statistical model. It helps readers judge precision, not whether an outcome is important or whether treatment caused it. The registry record does not report confidence intervals for the primary safety outcome, so the precision of the posted percentages cannot be evaluated using such intervals from the record. It also reports no analysis results for the primary endpoint in the analyses listing.
What can the serious-event categories tell us?
The SAE definition groups events by seriousness criteria, such as life-threatening events, hospitalization or medical significance. It does not mean every SAE was caused by study drug. The separate list of serious-event terms names categories and gives percentages, but the denominator and counting convention for those percentages are not reported in the registry record. A percentage attached to a term should therefore be read only as a registry-listed figure, not assumed to use all participants as its denominator. The death counts are also descriptive; the record does not specify their relationship to treatment or to the SAE count.
How should planning calculations be understood?
General statistical explanation: a sample-size planning illustration asks how many participants a hypothetical future study might need under specified assumptions. It is not a retrospective measure of whether this trial had adequate power, and it does not establish an observed treatment effect. The planning assumptions stated for the illustrations are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation and an effect defined as a stated fraction of the observed effect. For time-to-event outcomes, the stated convention is to give total events; for other outcomes, it is participants per arm. These assumptions are not the trial’s own design plan. The registry record does not report the trial’s sample-size justification, and planning illustrations cannot turn this non-randomized safety description into a controlled comparison.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 44 outcome measures and 0 analyses. The primary outcome concerns AEs and SAEs; listed secondary outcomes include response, clinical benefit and disease control rates in specified cancer and RET-status subgroups. Testing or estimating multiple outcomes can raise questions about multiplicity: when many statistical claims are considered, the chance of at least one apparently notable result can increase unless the analysis plan addresses that issue. The record does not report a multiplicity adjustment or a prespecified hierarchy for interpreting secondary outcomes. No statistical analyses are listed, so adjusted or unadjusted inferential results cannot be assessed from this record.
Missing-data handling is not reported in the registry record. The record gives the safety population definition and the event counts, but does not explain how incomplete follow-up, withdrawal, or missing event information was handled. The endpoint timeframe can extend to 30 days after the final dose, up to approximately 6.7 years, and the record does not describe how variation in time under observation was handled in the analysis.
The safety population includes participants who received at least 1 dose, regardless of starting dose level. The record says safety data were to be analyzed and reported as per a pre-planned grouped dose level II, and that this was specified in the SAP. The detailed SAP and the analysis methods are not reported in the registry record. The displayed dose-group counts are descriptive; they do not establish a randomized comparison. Definitions of the analysis populations for the cancer-specific secondary outcomes are also not reported in the registry record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Pralsetinib ≤ 300 mg QD | 26 | 37 | 70.3% |
| Pralsetinib 400 mg QD | 381 | 540 | 70.6% |
| Pralsetinib BID Dosing Schedule | 7 | 9 | 77.8% |
| Pralsetinib All Doses | 416 | 590 | 70.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Pralsetinib ≤ 300 mg QD | 15 of 37 |
| Pralsetinib 400 mg QD | 245 of 540 |
| Pralsetinib BID Dosing Schedule | 7 of 9 |
| Pralsetinib All Doses | 268 of 590 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Pralsetinib ≤ 300 mg QD | 36 of 37 |
| Pralsetinib 400 mg QD | 536 of 540 |
| Pralsetinib BID Dosing Schedule | 9 of 9 |
| Pralsetinib All Doses | 585 of 590 |
Most frequent serious adverse event terms
| Event term | Organ system | Pralsetinib ≤ 300 mg QD | Pralsetinib 400 mg QD | Pralsetinib BID Dosing Schedule | Pralsetinib All Doses |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | 5 of 37 | 87 of 540 | 1 of 9 | 94 of 590 |
| Anaemia | Blood and lymphatic system disorders | 1 of 37 | 32 of 540 | 1 of 9 | 34 of 590 |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | 2 of 37 | 27 of 540 | 0 of 9 | 29 of 590 |
| Lung neoplasm malignant | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 37 | 26 of 540 | 0 of 9 | 27 of 590 |
| Urinary tract infection | Infections and infestations | 2 of 37 | 23 of 540 | 1 of 9 | 26 of 590 |
| Sepsis | Infections and infestations | 2 of 37 | 20 of 540 | 3 of 9 | 25 of 590 |
| COVID-19 | Infections and infestations | 1 of 37 | 16 of 540 | 0 of 9 | 17 of 590 |
| Pyrexia | General disorders | 0 of 37 | 15 of 540 | 0 of 9 | 15 of 590 |
| COVID-19 pneumonia | Infections and infestations | 1 of 37 | 13 of 540 | 0 of 9 | 14 of 590 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 2 of 37 | 11 of 540 | 1 of 9 | 14 of 590 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Pralsetinib ≤ 300 mg QD | Pralsetinib 400 mg QD | Pralsetinib BID Dosing Schedule | Pralsetinib All Doses |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | 16 of 37 | 285 of 540 | 6 of 9 | 310 of 590 |
| Aspartate aminotransferase increased | Investigations | 17 of 37 | 273 of 540 | 3 of 9 | 295 of 590 |
| Constipation | Gastrointestinal disorders | 12 of 37 | 238 of 540 | 2 of 9 | 255 of 590 |
| Alanine aminotransferase increased | Investigations | 16 of 37 | 212 of 540 | 3 of 9 | 232 of 590 |
| Diarrhoea | Gastrointestinal disorders | 10 of 37 | 189 of 540 | 4 of 9 | 207 of 590 |
| Hypertension | Vascular disorders | 9 of 37 | 189 of 540 | 4 of 9 | 206 of 590 |
| Pyrexia | General disorders | 7 of 37 | 164 of 540 | 3 of 9 | 176 of 590 |
| White blood cell count decreased | Investigations | 14 of 37 | 157 of 540 | 2 of 9 | 175 of 590 |
| Fatigue | General disorders | 12 of 37 | 154 of 540 | 2 of 9 | 169 of 590 |
| Cough | Respiratory, thoracic and mediastinal disorders | 9 of 37 | 149 of 540 | 2 of 9 | 161 of 590 |
Serious events with the highest arm-level rate: sepsis (33.3%), pneumonia (16.1%), anaemia (11.1%), cardiac arrest (11.1%), abdominal pain (11.1%).
For each dose group, the SAE figures give the number of participants with an SAE and the number at risk; the other-AE figures use the same format. In the all-doses group, 416 of 590 participants had an SAE, while 585 of 590 had other AEs. The ≤ 300 mg QD group records 26 of 37 with an SAE and 36 of 37 with other AEs. The 400 mg QD group records 381 of 540 and 536 of 540, respectively. The BID dosing group records 7 of 9 and 9 of 9.
These are participant counts over the stated safety follow-up, not event totals or rates per unit of time. A participant may have had more than one event, but those event totals are not reported. SAEs meet seriousness criteria; they are not necessarily treatment-related. The record does not provide a randomized comparator, confidence intervals, or a basis for causal comparisons across dose groups. The serious-event term percentages also lack a reported denominator, so their population-level meaning cannot be established from the registry record alone.
12. What the Result Does, and Does Not, Mean
The primary safety outcome describes the number of participants recorded with AEs and SAEs after receiving pralsetinib. The all-doses results show that AEs were recorded for 585 of 590 participants and SAEs for 416 of 590. The dose-specific figures provide additional descriptive detail, but allocation was non-randomized. They cannot establish that one dose or schedule caused more or fewer events than another.
The AE definition allows events to be counted whether or not they are considered related to the drug. Therefore, event occurrence and treatment causation are different questions. The SAE classification indicates seriousness under the stated criteria; it does not itself assign cause. Likewise, recorded deaths should not be interpreted as treatment-related deaths in the absence of an attribution reported in the registry record.
The study also listed tumor-response and disease-control outcomes as secondary measures. The safety counts do not answer those efficacy questions. Nor do the registry entries establish comparative effectiveness, since there was no randomized control group. No confidence intervals or primary-outcome hypothesis tests are reported, limiting assessment of statistical precision.
The results are most appropriately understood as a description of adverse-event experience among the defined safety population during the stated follow-up. They do not by themselves establish a causal safety effect, compare pralsetinib with another treatment, or determine the benefit-risk balance for an individual. The registry record does not report enough analysis detail to resolve those broader questions.
13. Why This Trial Matters Statistically
This study’s statistical structure matters because its primary endpoint is a safety description in a first-in-human Phase 1/2 study, not a randomized comparison. Dose escalation and dose expansion serve different study roles: multiple doses were used during escalation, while the expansion dose was selected during escalation. Dose groups formed in this context should not be interpreted like randomized arms, even when the same endpoint is recorded for each.
The event counts also illustrate the importance of naming the analysis population and observation period. The safety population includes participants who received at least 1 dose, and the endpoint covers treatment and follow-up through 30 days after the final dose. Event percentages are meaningful only in relation to their denominator and follow-up context. For the serious-event term percentages, the registry does not report the denominator, which limits interpretation.
Finally, a count of participants with an event is not the same as a causal estimate, an event rate, or an estimate of tumor response. Separating descriptive safety findings from controlled treatment effects helps avoid over-reading the data. The absence of reported confidence intervals, hypothesis tests, missing-data methods and detailed analysis populations further defines what can be concluded from the registry record.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The study was open-label and non-randomized, with no randomized comparator identified in the registry record. Its dose groups therefore do not support like-for-like causal comparisons. The posted primary outcome is a safety measure and does not establish antitumor activity. Although response and disease-control outcomes are listed as secondary outcomes, their results are not included in the registry entries.
The record does not report confidence intervals or hypothesis-test results for the primary safety outcome. It also does not report methods for missing data, detailed handling of follow-up duration, or the denominator and counting convention for the serious-event term percentages. The definitions of populations for secondary efficacy analyses are not reported in the registry record.
The safety endpoint counts whether participants experienced events, rather than the total number, duration or recurrence of events. The event definition does not require drug causation, and the death counts are not accompanied by attribution information. Detailed sample-size justification, interim analyses and statistical analysis methods are not reported in the registry record. These limitations constrain precision, causal interpretation and comparison across dose groups.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: 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 — NCT03037385.
- Registry record last updated 2025-05-31; status last verified 2025-05.
- Results first posted 2025-05-31.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The registry record describes a completed, open-label, non-randomized Phase 1/2 study of oral pralsetinib in participants with thyroid cancer, non-small cell lung cancer and other advanced solid tumors. Enrollment is reported as 590. The primary endpoint counts participants with AEs and SAEs in a safety population defined by receipt of at least 1 dose.
In the all-doses group, 416 of 590 participants had an SAE and 585 of 590 had other AEs. The dose groups are descriptive rather than randomized comparisons. The record also lists serious-event terms, deaths, and secondary tumor-response outcomes, but does not report enough statistical detail to support causal comparisons or assess uncertainty for the primary safety results.