NCT07447076 Paxalisib Rhabdoid Tumor of the CNS Clinical Landscape Report 2026: Design, Endpoints, Sponsor and Readout Outlook
Turn a newly registered trial into a decision-ready clinical landscape. This report examines NCT07447076 using PatSnap Clinical Trials MCP for protocol design and endpoint evidence, with Drug & Asset MCP and Company & Deal Intelligence MCP used as the companion asset and sponsor enrichment workflow. Explore PatSnap MCP Servers to reproduce the research sequence inside an AI workflow.
MCP evidence snapshot: 27 August 2026. Trial records, recruitment status and projected dates can change after the snapshot and should be rechecked before operational decisions.
Why NCT07447076 is a hot trial to watch
Rhabdoid Tumor of the CNS is being segmented by mechanism, treatment setting, geography and endpoint architecture. NCT07447076 is notable because it evaluates Paxalisib in a Phase 2 design sponsored by The University of California, San Francisco. The main development question is whether the protocol can translate its rationale into a clinically interpretable and operationally credible readout.
Trial landscape snapshot
| Field | Indexed detail |
|---|---|
| Registration | NCT07447076 |
| Official title | Study of Novel Therapies for Young People With Recurrent/Progressive Atypical Teratoid Rhabdoid Tumor (ATRT) |
| Phase / status | Phase 2 / Recruiting |
| Intervention | Paxalisib |
| Sponsor | The University of California, San Francisco |
| Geography | United States |
| Enrollment | 29 |
| Primary endpoint | Arm A (Phase I): Proportion of participants who experience dose-limiting toxicity (DLT) |
| Endpoint time frame | up to 28 days |
| Primary completion / readout proxy | 2035-12-31 |
Protocol design and endpoint interpretation
This is a multi-treatment arm study that will be conducted through the Pacific Pediatric Neuro-oncology Consortium (PNOC).The study will assess the safety and efficacy of novel therapies and combinatorial strategies for participants with recurrent or progressive ATRT.
Allocation is Non-Randomized, masking is None (Open Label), and the intervention model is Parallel Assignment. Planned enrollment of 29 participants across United States shapes statistical precision, execution risk and external validity. Interpretation should account for baseline risk, prior therapy, assessment schedule, missing-data handling and clinical relevance—not only statistical significance.
- Arm A (Phase I): Proportion of participants who experience dose-limiting toxicity (DLT) (up to 28 days) — Tolerability is defined as the proportion of participants receiving at least one dose of combination gemcitabine and paxalisib with a reported dose-limiting toxicity (DLT) during cycle 1 for all participants in Phase I.
- Arm A (Phase I): Recommended Phase 2 Dose (RP2D) (Phase I) (up to 28 days) — The confirmed RP2D of combination gemcitabine and paxalisib implemented for participants enrolled in Phase II will be reported.
- Arm A (Phase II): Rate of Clinical Benefit (up to 2 years (24 cycles)) — Assess the efficacy of combination gemcitabine and paxalisib for participants in Phase II. Clinical Benefit rate (CBR) is defined as complete response (CR) + partial response (PR) + stable disease (SD), where SD is sustained over 4 months.
Readout outlook and evidence gap
The current protocol points to 2035-12-31 as the best available readout proxy. Because this is an active or newly posted study, a trial-specific result citation is not included until a normalized clinical-trial-result record becomes available. That gap is itself operationally important: teams should monitor first result indexing, conference abstracts, protocol amendments and changes to the primary-completion date.
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Asset and sponsor context
Drug & Asset MCP profile: Paxalisib is indexed as No normalized modality returned, with target No normalized target returned, mechanism No normalized mechanism returned, and global highest development status No normalized global status returned.
Company & Deal Intelligence MCP profile: The University of California, San Francisco is resolved to a normalized organization record in SAN FRANCISCO COUNTY, United States. The organization workflow adds identity, location, portfolio and partnering context where available.
The sponsor profile matters because scientific rationale alone does not determine development value. Manufacturing readiness, portfolio fit, geographic reach, partnering capacity and the ability to fund confirmatory development can determine whether a positive signal becomes a competitive asset.
White space around this program
- Sharper patient selection: prospective biomarker or phenotype definitions that identify who is most likely to benefit.
- Clinically interpretable endpoints: outcomes connecting activity with function, symptoms, survival or treatment burden.
- Comparator relevance: evidence against the most decision-relevant contemporary standard of care.
- Broader external validity: evidence across additional geographies, demographic groups and real-world settings.
- Operational differentiation: a development path that closes the readout gap without sacrificing safety or durability.
What to monitor next
Monitor recruitment, enrollment changes, protocol amendments, endpoint hierarchy, primary-completion timing, first result indexing, asset ownership and sponsor partnerships. A change in endpoint, population or ownership can alter the probability of success before a headline data release.
Bottom line
NCT07447076 provides a focused lens on Rhabdoid Tumor of the CNS development. Its value will be determined by whether Paxalisib can convert the current Phase 2 design into evidence that is clinically meaningful, operationally credible and differentiated from competing programs.
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