Clinical Trials

Paxalisib (GDC-0084) is under evaluation across several Phase I through Phase III clinical trials targeting central nervous system disorders and advanced malignancies, including adult glioblastoma, pediatric brain tumors, primary CNS lymphoma, and focal cortical dysplasia. Spanning completed, active, suspended, and recruiting statuses, these studies range from early pharmacokinetic trials in healthy subjects to adaptive oncology evaluations. Research is led by diverse sponsors, including Kazia Therapeutics, academic institutions such as the University of California, San Francisco, and pediatric centers like St. Jude Children's Research Hospital.

NCT Number Recruitment Conditions Sponsor/Collaborators Start Date Phases
NCT07447076 RECRUITING
Recurrent Atypical Teratoid/Rhabdoid Tumor; Atypical Teratoid/Rhabdoid Tumor (ATRT) of the CNS
Sabine Mueller, MD, PhD
 2026-06-17 PHASE2
NCT06208657 RECRUITING
Childhood Cancer; Childhood Solid Tumor; Childhood Brain Tumor; Recurrent Cancer; Refractory Cancer
Australian & New Zealand Children's Haematology/Oncology Group
 2024-07-10 PHASE1; PHASE2
NCT07391215 RECRUITING
Malignant Primary Gliomas; Glioblastoma Multiforme (GBM)
Institute of Cancer Research, United Kingdom
 2026-01-19 PHASE1; PHASE2
NCT03970447 RECRUITING
Glioblastoma
Global Coalition for Adaptive Research
 2019-07-30 PHASE2; PHASE3
NCT05009992 RECRUITING
Diffuse Intrinsic Pontine Glioma; Diffuse Midline Glioma, H3 K27M-Mutant; Recurrent Diffuse Intrinsic Pontine Glioma; Recurrent Diffuse Midline Glioma, H3 K27M-Mutant; Recurrent WHO Grade III Glioma; WHO Grade III Glioma
University of California, San Francisco
 2021-10-20 PHASE2
NCT07287202 RECRUITING
Focal Cortical Dysplasia; Tuberous Sclerosis Complex (TSC); Hemimegalencephaly
Sovargen
 2026-07-14 PHASE1; PHASE2
NCT04906096 RECRUITING
Primary Central Nervous System Lymphoma; Non-Hodgkin Lymphoma of Extranodal Site
Lakshmi Nayak, MD
 2021-06-01 PHASE2
NCT04192981 ACTIVE_NOT_RECRUITING
Brain Metastases; Leptomeningeal Metastasis
Memorial Sloan Kettering Cancer Center
 2019-12-06 PHASE1
NCT03994796 ACTIVE_NOT_RECRUITING
CDK Gene Mutation; Metastatic Malignant Neoplasm in the Brain; Metastatic Malignant Solid Neoplasm; NTRK Family Gene Mutation; PI3K Gene Mutation; ROS1 Gene Mutation; KRAS G12C Mutation
Alliance for Clinical Trials in Oncology
 2019-10-17 PHASE2
NCT05183204 SUSPENDED
Glioblastoma
Weill Medical College of Cornell University
 2022-02-14 PHASE2
NCT03765983 TERMINATED
Breast Cancer
Dana-Farber Cancer Institute
 2019-02-25 PHASE2
NCT03522298 COMPLETED
Glioblastoma, Adult
Kazia Therapeutics Limited
 2018-05-15 PHASE2
NCT05012670 COMPLETED
Healthy
Kazia Therapeutics Limited
 2021-08-18 PHASE1
NCT05012670 Completed
Healthy
Kazia Therapeutics Limited|Quotient Sciences
 2021-08-18 Phase 1
NCT03696355 COMPLETED
Brain and Central Nervous System Tumors
St. Jude Children's Research Hospital
 2018-11-19 PHASE1
NCT03696355 Completed
Brain and Central Nervous System Tumors
St. Jude Children''s Research Hospital|Kazia Therapeutics Limited
 2018-11-19 Phase 1
NCT01547546 COMPLETED
Glioblastoma, Glioma
Genentech, Inc.
 2012-04 PHASE1

(data from https://clinicaltrials.gov, updated on 2026-06-23)

Check the Paxalisib (GDC-0084) product page for in-depth specifications, including solubility, stock solutions, MOA, and working concentrations.

Compliance for Clinical Use

Mechanism and Biochemical Profile

Paxalisib (GDC-0084) is a potent, brain-penetrant dual inhibitor that binds to class I PI3K isoforms and mTOR, thereby blocking downstream phosphorylation within the oncogenic PI3K/Akt/mTOR signaling cascade to arrest cell growth and induce apoptosis. By effectively penetrating the blood-brain barrier and halting intracellular survival pathways, this dual inhibition offers therapeutic relevance in suppressing tumor growth across central nervous system malignancies, including glioblastoma and diffuse intrinsic pontine glioma.

Appendix RUO and cGMP Quality Standards

Quality Dimension RUO (Research Use Only) cGMP (Current Good Manufacturing Practice)
Clinical Applicability Prohibited in human clinical trials or medical diagnostics. Mandatory for human clinical trials (Phase I–III) and therapies.
Regulatory Status Non-regulated grade; exempt from drug manufacturing laws. Legally enforced by health authorities (e.g., FDA, EMA, NMPA).
Facility Environment Unclassified analytical or research laboratories. Validated Cleanrooms (ISO Class 5–8) with continuous monitoring.
Quality Control Basic purity and activity testing. Rigorous safety release testing (Sterility, Endotoxin, Mycoplasma).
Process Validation Basic equipment calibration; no process validation required. Full qualification (IQ/OQ/PQ) and complete batch records.
Quality Assurance Vendor self-declared without required formal QMS. Mandatory QA/QC unit, Change Control, CAPA, and vendor audits.
Regulatory Impact High risk of IND rejection if used as a critical raw material. Required for IND/NDA filings, supported by Drug Master Files (DMF).

Footnotes

Regulatory Note: Governed by FDA (21 CFR Parts 210/211/312), EMA (EudraLex Vol 4), ICH Guidelines (E6/Q7/Q9/Q10), and compendial standards (USP <71>/<85>/<1043>).

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Note: Technical data last updated: Sep 1, 2026.