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 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1) (NCT05103358), a non-randomized phase 2 study. The primary outcome is Overall Response Rate (ORR).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT05103358 |
| Phase | Phase 2 |
| Condition | Tumor, Tumor, Solid, Metastasis |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 128 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Aadi Bioscience, Inc. |
| Results posted | Yes |
2. Clinical Question
PRECISION 1 asks whether nab-sirolimus is associated with confirmed tumor responses in adults and adolescents with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2. Its primary endpoint is overall response rate (ORR), defined as the percentage of participants whose best overall response is a confirmed partial response or complete response, assessed using RECIST v1.1 by independent radiographic review.
Population
Adults and adolescents with malignant solid tumors and pathogenic inactivating alterations in TSC1 or TSC2. The efficacy populations are described as enrolled participants who received nab-sirolimus, with the efficacy-evaluable set also requiring a post-baseline tumor scan.
Intervention
Nab-sirolimus, the study drug administered in this open-label trial. The dosing regimen and treatment duration are not reported in the registry record.
Comparator
There is no randomized control group. The registry describes separate cohorts for TSC1 and TSC2 alterations, both receiving nab-sirolimus; these cohorts are not a like-for-like control comparison.
Primary question
What proportion of the defined analysis population has a confirmed complete or partial response by independent radiographic review? The registry record states the endpoint and assessment schedule but does not report an ORR estimate or its confidence interval.
This is a treatment-response question in a molecularly selected, heterogeneous tumor population, rather than a randomized comparison of nab-sirolimus against another treatment or usual care. The design can describe responses observed among treated participants, but by itself it cannot separate treatment-associated change from other explanations for the observed outcomes.
3. Trial Design
PRECISION 1 is a Phase 2, multicenter, open-label basket trial. The registry classifies it as non-randomized and single-group. It records enrollment of 128 participants and a completed status. The recorded study start is 2022-03-10, and the primary completion date is 2024-12-23.
A basket design groups participants by a shared biological feature even when their cancers arise in different tissues. In this trial, eligibility centers on pathogenic inactivating alterations in TSC1 or TSC2. The registry describes Arm A for participants with TSC1 alterations and Arm B for participants with TSC2 alterations. Both cohorts receive nab-sirolimus; there is no randomized treatment assignment or untreated comparator in the record.
Open-label means that treatment masking is not used. The registry lists no masking. This can matter for outcomes involving clinical judgment or participant reports, although the primary tumor-response assessment is specified as an independent radiographic review. The registry does not describe the review procedures beyond that designation.
Response assessment begins after treatment initiation. The first assessment is scheduled 6 weeks (±7 days) after the first treatment, with assessments every 6 weeks (±7 days) during the first year, then every 12 weeks (±7 days) thereafter until disease progression, up to 30 months. The primary endpoint is based on CT or MRI scans from treatment initiation until progression.
The registry lists duration of response, disease control rate, time to response, progression-free survival, overall survival, patient-reported outcome, and treatment-emergent and treatment-related adverse events among secondary outcomes. It also lists 8 outcome measures. The registry record does not report treatment dose, eligibility details beyond the stated disease and genetic features, or an allocation procedure that would assign participants between treatment groups.
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: To avoid over-enrolling in any one tumor type, as defined by organ and histology, enrollment was capped at 15 patients for each organ-histology in each Arm.
Overall Study
| Stage | Arm A: Pathogenic Inactivating TSC1 Alterations | Arm B: Pathogenic Inactivating TSC2 Alterations |
|---|---|---|
| STARTED | 67 | 61 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 67 | 61 |
Reasons for not completing this period:
| Reason | Arm A: Pathogenic Inactivating TSC1 Alterations | Arm B: Pathogenic Inactivating TSC2 Alterations |
|---|---|---|
| Withdrawal by Subject | 13 | 8 |
| Study terminated by sponsor | 26 | 23 |
| Lost to Follow-up | 0 | 2 |
| Death | 26 | 26 |
| Sponsor has closed the study. disease progression | 1 | 0 |
| Disease progression | 1 | 0 |
| Symptoms | 0 | 1 |
| Discontinue nab-sirolimus | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: The Full Analysis Set included all enrolled patients who received at least 1 dose of nab-sirolimus. The Efficacy Evaluable Set included all enrolled patients who received at least one dose of nab-sirolimus and had at least one post-baseline scan for tumor response evaluation.
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.
| Characteristic | Unit | Arm A: Pathogenic Inactivating TSC1 Alterations | Arm B: Pathogenic Inactivating TSC2 Alterations | Total |
|---|---|---|---|---|
| Participants analysed | 67 | 61 | 128 | |
| Age, Customized: Age – <65 years | Participants | 34 | 34 | 68 |
| Age, Customized: Age – 65-74 years | Participants | 23 | 18 | 41 |
| Age, Customized: Age – > = 75 years | Participants | 10 | 9 | 19 |
| Sex: Female, Male: Female | Participants | 41 | 36 | 77 |
| Sex: Female, Male: Male | Participants | 26 | 25 | 51 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 4 | 8 | 12 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 61 | 53 | 114 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 2 | 0 | 2 |
| Race/Ethnicity, Customized: Race – White | Participants | 46 | 45 | 91 |
| Race/Ethnicity, Customized: Race – Asian | Participants | 13 | 13 | 26 |
| Race/Ethnicity, Customized: Race – Not Applicable | Participants | 4 | 2 | 6 |
| Race/Ethnicity, Customized: Race – Black or African American | Participants | 3 | 1 | 4 |
| Race/Ethnicity, Customized: Race – Other | Participants | 1 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Response Rate (ORR), measured over The first response assessment is done 6 weeks (±7 days) after first treatment and should be repeated every 6 weeks (±7 days) for the first year, then every 12 weeks (±7 days) thereafter until disease progression , up to 30 months..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | Percent of participants |
| Endpoint classification | Binary Endpoint |
Overall response rate measures the proportion of participants whose best overall tumor response is a confirmed complete response or partial response. In this trial, responses are assessed against RECIST v1.1 using CT or MRI scans, with independent radiographic review. A complete response requires disappearance of all target lesions. A partial response requires at least a 30% decrease from baseline in the sum of target-lesion diameters, with no new lesions and no progression of non-target lesions.
ORR is useful in a tumor-response study because it expresses how often a predefined degree of tumor shrinkage is documented. Requiring confirmation helps distinguish a response sustained at assessment from an unconfirmed observation. Independent review can reduce reliance on the treating team’s assessment, although the details of review procedures are not reported in the registry record.
The stated assessment schedule begins 6 weeks (±7 days) after first treatment and continues at the stated intervals until progression, up to 30 months. The record defines the Full Analysis Set as all enrolled participants who received at least 1 dose of nab-sirolimus. It defines the Efficacy Evaluable Set as those participants who received at least one dose and had at least one post-baseline scan for tumor-response evaluation.
The registry record does not report the number or percentage of participants with confirmed responses, nor a 95% confidence interval for ORR. Therefore, the endpoint definition and assessment plan can be described, but the magnitude and precision of the observed response rate cannot be interpreted from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a Phase 2 study of nab-sirolimus in participants selected for pathogenic inactivating TSC1 or TSC2 alterations. Its primary outcome is ORR by independent radiographic review. However, the record does not report the observed ORR, the number of confirmed responses, or a confidence interval. Without those results, no quantitative conclusion about the frequency or precision of response can be drawn from the registry entry.
Enrollment was 128, with separate TSC1 and TSC2 cohorts. The adverse-event record lists 67 participants at risk in the TSC1 cohort and 61 in the TSC2 cohort for serious adverse events. It lists 35 affected participants in the TSC1 cohort and 29 in the TSC2 cohort. These safety counts do not supply the missing efficacy endpoint, and they should not be used as a substitute for response results.
The two genetic cohorts are not randomized comparators. Differences between their outcomes, if outcomes were available, would not by themselves establish that one alteration predicts a different treatment effect: the cohorts could differ in tumor types, clinical characteristics, or other factors. The registry record does not report a comparative analysis plan or adjusted comparison between them.
In general, a confidence interval communicates uncertainty around an estimate, not a guarantee that the true value lies within a particular range. The primary outcome is specified with a 95% confidence interval, but the interval itself is not reported in the registry record. As a result, neither the response estimate nor its statistical precision can be assessed.
The study’s open-label, non-randomized design is suited to describing outcomes in a selected group receiving the study drug, but it lacks a randomized control group for estimating a comparative treatment effect. The independent radiographic review is a feature of the primary endpoint assessment; it does not remove the limitations that arise from the absence of randomization or a control group.
The registry lists additional outcomes, including progression-free survival, overall survival, duration of response, and patient-reported outcome. Their definitions and results are not included in the record details available for interpretation. The record also lists no analyses. This means the statistical comparisons or other analyses cannot be characterized from the registry entry; it does not establish that no analyses were conducted outside the record.
Overall, the record supports a description of the study question, design, endpoint definition, assessment schedule, and selected safety counts. It does not provide the primary response result needed to judge the observed response frequency or its precision, and it does not support a controlled comparison of nab-sirolimus with another treatment.
9. Statistical Methods Explained
The statistical interpretation begins with the estimand implied by the endpoint: the proportion of participants in a defined analysis population who achieve a confirmed complete or partial response. The registry specifies a Full Analysis Set and an Efficacy Evaluable Set. The former includes enrolled participants who received at least 1 dose of nab-sirolimus; the latter additionally requires at least one post-baseline scan for tumor-response evaluation. Those definitions determine who contributes to an ORR calculation, but the registry record does not report the response counts or the population used for a posted ORR result.
What does a response proportion convey?
As a general statistical concept, a proportion describes the share of people in a defined group who meet a stated outcome. For ORR, the outcome is a confirmed complete or partial response under the stated RECIST v1.1 criteria and independent radiographic review. A proportion is interpretable only alongside its analysis population and the method used to handle participants whose response status cannot be evaluated. The registry defines two populations but does not report how missing scans, unevaluable assessments, or withdrawals were handled for the primary analysis.
The response definition is based on tumor measurements: complete response means disappearance of all target lesions; partial response requires at least a 30% decrease in the sum of target-lesion diameters from baseline, with no new lesions and no progression of non-target lesions. Confirmation is part of the endpoint definition. The record does not give further operational details about confirmation timing or the independent review process.
How should a confidence interval be read?
In general, a confidence interval describes statistical uncertainty around an estimated quantity under the assumptions of the method used. A narrower interval indicates greater precision than a wider interval, while neither width nor location alone establishes clinical importance. The primary endpoint is specified with a 95% confidence interval, but the registry record does not report the interval or its corresponding ORR estimate. Consequently, no assessment of precision is possible from the record.
The ORR is a binary outcome at the participant level: a participant either meets the confirmed-response definition or does not. General methods for such outcomes estimate a proportion and may attach an interval reflecting sampling uncertainty. The registry entries do not identify the interval calculation method, so a particular statistical procedure should not be assumed.
What does the assessment schedule contribute?
The first response assessment is scheduled 6 weeks (±7 days) after first treatment. Assessments are repeated every 6 weeks (±7 days) during the first year, then every 12 weeks (±7 days) until disease progression, up to 30 months. This schedule defines when imaging is planned, but does not establish that every participant completed every assessment. In general, assessment timing can affect which response is recorded as best overall response and how much follow-up is available. The registry does not report actual scan completion, deviations from schedule, or the handling of assessments after discontinuation.
Independent radiographic review is specified for the primary endpoint, which can help separate endpoint assessment from the treating clinician’s view. That feature does not itself resolve missing data or establish a causal treatment effect. The registry record does not state whether the review was blinded to treatment or provide other review-process details.
Why does the design matter for inference?
The trial is non-randomized, open-label, and classified as single-group. The two arms are defined by TSC1 or TSC2 alteration, not by assignment to different treatments. General statistical reasoning therefore treats these cohorts as descriptive groups rather than randomized control and treatment groups. Any difference between cohorts could reflect differences in their composition as well as differences associated with the genetic alteration. The record does not report an adjusted comparison or a prespecified method for comparing the cohorts.
Randomization is generally used to balance measured and unmeasured prognostic factors across comparison groups, on average. Without it, a single-group study can describe outcomes among participants who received treatment but cannot by itself identify what would have happened to the same participants under a different treatment or no study treatment. Independent endpoint review may strengthen measurement procedures, but it cannot create a concurrent control group.
What can planning illustrations tell readers?
General sample-size planning uses assumptions about the outcome, target effect, allocation, significance level, and desired power to illustrate how much information a future study might require. Such illustrations are planning exercises, not observed trial results and not evidence that the completed study had a particular probability of detecting an effect. The stated planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects expressed as fractions of an observed effect. Time-to-event planning is described in total events; other planning is described as participants per arm.
These planning assumptions do not describe the trial’s own design justification. The registry record does not report a protocol-based sample-size justification, target power, or a planned effect size. Numerical planning outputs are not reported in the registry record. They therefore cannot be used to infer the actual number required for this trial or to claim that a treatment effect was detected.
The record lists no analyses. It does not report a statistical analysis plan, adjustment strategy, subgroup analysis procedure, interim-analysis plan, or approach to multiplicity. Absence of those details from the registry record should be read as missing documentation, not proof that such procedures were absent from study conduct.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several secondary outcomes in addition to ORR: duration of response, disease control rate, time to response, progression-free survival, overall survival, patient-reported outcome, and treatment-emergent and treatment-related adverse events. Considering several endpoints can raise a multiplicity question: when many outcomes are tested, the chance of at least one apparently notable result can increase. In general, statistical plans may address this through a prespecified hierarchy, adjustment, or by treating secondary findings as descriptive. The registry record does not report a multiplicity strategy or the order in which outcomes were intended to be tested.
The registry lists 8 outcome measures and no analyses. The analysis methods, any subgroup procedures, and any interim analyses are not reported in the registry record. It is not possible to determine from that information whether outcome testing was adjusted or whether the listed secondary outcomes were intended as formal hypothesis tests.
Missing-data handling for the primary response endpoint is also not reported in the registry record. The record defines the Full Analysis Set as enrolled participants who received at least 1 dose, and the Efficacy Evaluable Set as those who received at least one dose and had at least one post-baseline scan. It does not state how participants without an assessable post-baseline scan were treated in an ORR analysis, or how missed scans and early discontinuation were addressed.
These distinctions matter because the population used for an estimate and the treatment of unavailable assessments can affect how a response proportion is interpreted. The registry definitions identify candidate populations but do not provide the analysis population actually used for posted results. No ORR result or corresponding confidence interval is 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 |
|---|---|---|---|
| Arm A: Pathogenic Inactivating TSC1 Alterations | 35 | 67 | 52.2% |
| Arm B: Pathogenic Inactivating TSC2 Alterations | 29 | 61 | 47.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Arm A: Pathogenic Inactivating TSC1 Alterations | 26 of 67 |
| Arm B: Pathogenic Inactivating TSC2 Alterations | 26 of 61 |
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 |
|---|---|
| Arm A: Pathogenic Inactivating TSC1 Alterations | 67 of 67 |
| Arm B: Pathogenic Inactivating TSC2 Alterations | 61 of 61 |
Most frequent serious adverse event terms
| Event term | Organ system | Arm A: Pathogenic Inactivating TSC1 Alterations | Arm B: Pathogenic Inactivating TSC2 Alterations |
|---|---|---|---|
| Acute kidney injury | Renal and urinary disorders | 3 of 67 | 4 of 61 |
| Pneumonia | Infections and infestations | 3 of 67 | 2 of 61 |
| Sepsis | Infections and infestations | 3 of 67 | 2 of 61 |
| Urinary tract infection | Infections and infestations | 5 of 67 | 0 of 61 |
| Pleural effusion | Respiratory, thoracic and mediastinal disorders | 3 of 67 | 1 of 61 |
| Abdominal pain | Gastrointestinal disorders | 2 of 67 | 1 of 61 |
| Nausea | Gastrointestinal disorders | 1 of 67 | 2 of 61 |
| Dehydration | Metabolism and nutrition disorders | 2 of 67 | 1 of 61 |
| Confusional state | Psychiatric disorders | 2 of 67 | 1 of 61 |
| COVID-19 pneumonia | Infections and infestations | 2 of 67 | 0 of 61 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Arm A: Pathogenic Inactivating TSC1 Alterations | Arm B: Pathogenic Inactivating TSC2 Alterations |
|---|---|---|---|
| Fatigue | General disorders | 30 of 67 | 24 of 61 |
| Nausea | Gastrointestinal disorders | 25 of 67 | 19 of 61 |
| Anaemia | Blood and lymphatic system disorders | 32 of 67 | 10 of 61 |
| Diarrhoea | Gastrointestinal disorders | 24 of 67 | 17 of 61 |
| Decreased appetite | Metabolism and nutrition disorders | 28 of 67 | 12 of 61 |
| Stomatitis | Gastrointestinal disorders | 21 of 67 | 18 of 61 |
| Hypokalaemia | Metabolism and nutrition disorders | 21 of 67 | 13 of 61 |
| Constipation | Gastrointestinal disorders | 17 of 67 | 14 of 61 |
| Platelet count decreased | Investigations | 16 of 67 | 13 of 61 |
| Weight decreased | Investigations | 18 of 67 | 11 of 61 |
Serious events with the highest arm-level rate: urinary tract infection (7.5%), acute kidney injury (6.6%), pneumonia (4.5%), sepsis (4.5%), pleural effusion (4.5%).
The registry’s serious adverse-event summary separates participants by alteration cohort. In the TSC1 cohort, 67 participants are listed as at risk and 35 as affected. In the TSC2 cohort, 61 are listed as at risk and 29 as affected. The record also lists 26 deaths in each cohort. These are descriptive safety counts; the record does not establish from these figures alone whether an event was caused by nab-sirolimus.
The listed serious-event terms and frequencies are urinary tract infection, 7.5%; acute kidney injury, 6.6%; pneumonia, 4.5%; sepsis, 4.5%; pleural effusion, 4.5%; and nausea, 3.3%. The record does not specify in the available details which cohort each term frequency refers to, so those percentages should not be assigned to a particular cohort. Nor should a term frequency be assumed to represent the percentage with any serious adverse event.
In general, safety events can be counted by affected participants or by event occurrences, and one participant may experience more than one event. The registry entries do not describe the counting convention for the listed terms or provide additional event-level context. The safety figures should therefore be read as the registry’s reported summary, not as evidence of causation or a comparison against another treatment.
12. What the Result Does, and Does Not, Mean
The registry record establishes the setting and question of PRECISION 1: a Phase 2, open-label basket trial of nab-sirolimus in adults and adolescents with malignant solid tumors harboring pathogenic inactivating TSC1 or TSC2 alterations. It defines ORR as the primary endpoint and specifies assessment using RECIST v1.1 with independent radiographic review. It also provides the response-assessment schedule and definitions of two efficacy populations.
The key limitation for interpreting efficacy is that the record does not report the ORR estimate, response count, or 95% confidence interval. The endpoint definition alone does not show how many participants responded or how precisely a response rate was estimated. The listed safety figures do not fill that gap, because adverse-event counts measure a different set of outcomes.
Even if response results were available, the non-randomized, single-group design would chiefly support a description of outcomes among treated participants. It would not by itself show what would have happened to comparable participants receiving another treatment. The TSC1 and TSC2 cohorts are not randomized comparators, so contrasts between them would not provide a controlled estimate of treatment effect.
General statistical interpretation also distinguishes a response observed during treatment from a causal effect attributable to treatment. A causal comparison requires a credible counterfactual—an estimate of outcomes under an alternative condition for otherwise comparable participants. The registry record describes no randomized control group. Independent radiographic review can support consistent outcome assessment, but it cannot supply that counterfactual.
The registry lists secondary outcomes, but their numerical results and analysis details are not reported in the record. Thus, conclusions about duration of response, disease control, time to response, progression-free survival, overall survival, or patient-reported outcome cannot be drawn from the listed outcome names alone. The appropriate reading is limited to the design, endpoint definitions, assessment plan, and reported safety summaries, while recognizing that the primary efficacy result is not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable for studying a basket population selected by genetic alteration across malignant solid tumors. A shared molecular eligibility feature can create a coherent research question across otherwise different tumor types, but it also means the enrolled population may include clinically diverse diseases. The registry record does not give the tumor-type distribution, so the degree of clinical heterogeneity cannot be characterized from the listed details.
The two alteration cohorts make the structure visible: participants are grouped by TSC1 or TSC2 status, while the intervention is nab-sirolimus in both. This is not a randomized comparison of treatments. Distinguishing a cohort description from a controlled comparison is essential when interpreting any outcome estimates that may be reported elsewhere.
The endpoint also illustrates how a trial’s statistical question depends on operational definitions. ORR is not simply any observed tumor change: it requires a confirmed complete or partial response according to RECIST v1.1, with independent radiographic review. The scheduled imaging intervals and analysis-population definitions give context to what the response measure is intended to capture.
At the same time, registry reporting is necessary for readers to assess results. The endpoint is specified with a 95% confidence interval, but neither the estimate nor interval is reported in the registry record. That absence prevents evaluation of the observed response frequency and precision. It also highlights the difference between a clearly defined outcome and an available outcome result.
Finally, the trial emphasizes the boundary between descriptive evidence and comparative inference. A single-group study can document outcomes in a selected cohort and contribute information about a rare molecularly defined setting. Without a randomized control group, however, it cannot establish a comparative treatment effect through its design alone. Statistical interpretation should preserve both the value of the defined research question and the limits of the evidence recorded.
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.
Several limitations specific to this trial affect what can be concluded from the registry record. The study is non-randomized, open-label, and classified as single-group, with the two arms formed by TSC1 and TSC2 alteration status. The design does not provide a randomized control group, so treatment outcomes cannot be directly compared with outcomes under an alternative treatment within the trial.
The enrolled population is defined by genetic alteration and malignant solid tumor, but the registry entries do not report the distribution of tumor types or other participant characteristics. The degree to which results might vary across tumor types or clinical subgroups cannot be assessed from the record. The dosing regimen and treatment duration are also not reported in the registry entries.
The primary endpoint is described, but the ORR estimate, response count, and confidence interval are not reported in the registry record. The record defines two efficacy populations without stating which population was used for a reported primary result. It does not describe how missing scans, unevaluable responses, missed assessments, or discontinuations were handled.
The record lists secondary outcomes but does not report their results or analysis procedures. It does not report a multiplicity approach, a sample-size justification, interim-analysis procedures, or the statistical analysis plan. These details cannot be inferred from the endpoint list or the study’s enrollment.
Safety information is also limited to the listed counts and terms. The registry entries do not establish causality for the events, specify the cohort attribution of the listed term percentages, or provide enough context to compare safety with another treatment. These limitations concern the information recorded and the design; they do not establish that unreported analyses or data do not exist elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1) — NCT05103358.
- Registry record last updated 2026-10-28; status last verified 2026-09.
- Results first posted 2026-09-28.
- 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
PRECISION 1 (NCT05103358) is a completed Phase 2, multicenter, open-label, non-randomized basket trial of nab-sirolimus. The registry records enrollment of 128 participants and describes cohorts with pathogenic inactivating TSC1 or TSC2 alterations. The recorded study start is 2022-03-10, and the primary completion date is 2024-12-23.
The primary endpoint is ORR, defined as confirmed complete or partial response by RECIST v1.1 on CT or MRI, determined by independent radiographic review. The first response assessment is scheduled 6 weeks (±7 days) after first treatment, with later assessments at the stated intervals until progression, up to 30 months.
The registry record does not report the primary ORR result or its 95% confidence interval. It lists secondary outcomes and safety summaries, but does not report analysis methods, missing-data handling, or a multiplicity strategy. Because the two cohorts are not randomized comparators, the design describes outcomes among treated participants rather than providing a controlled treatment comparison.