Paxalisib

also known as GDC-0084 · G-02441729 · RG 7666 · Sim-0395 · SVG103

Small molecule targets MTOR via inhibition

Developed for: glioblastoma · metastatic malignant neoplasm in the brain · adult glioblastoma · atypical teratoid rhabdoid tumor · brain cancer · breast cancer · central nervous system neoplasm · childhood malignant neoplasm
+9 more
· diffuse midline glioma, H3 K27M-mutant · grade III glioma · hemimegalencephaly · isolated focal cortical dysplasia type II · neoplastic meningitis · non-Hodgkin lymphoma · primary central nervous system lymphoma · refractory malignant neoplasm · tuberous sclerosis
Investigated by: Kazia Therapeutics Limited · Genentech, Inc. · Alliance for Clinical Trials in Oncology · AstraZeneca · Australian & New Zealand Children's Haematology/Oncology Group · Bayer
+39 more
· Biohaven Pharmaceuticals, Inc. · Bristol-Myers Squibb · C17 Council · CV Biomanufacturing · Cambridge University Hospitals NHS Foundation Trust · Cancer Research UK · Dana-Farber Cancer Institute · Eli Lilly and Company · Global Coalition for Adaptive Research · Institute of Cancer Research, United Kingdom · Jazz Pharmaceuticals · Kintara Therapeutics, Inc. · Knoa Pharma LLC · Lakshmi Nayak, MD · Medical Research Future Fund · Memorial Sloan Kettering Cancer Center · Minderoo Foundation · Mirati Therapeutics Inc. · Mithil Prasad Foundation · National Cancer Institute (NCI) · National Institute of Neurological Disorders and Stroke (NINDS) · Neeve Kolte and Brave Ronil Foundation · Pacific Pediatric Neuro-Oncology Consortium · Polaris Group · Quotient Sciences · Rally Foundation · Royal Marsden NHS Foundation Trust · Sabine Mueller, MD, PhD · Sovargen · St. Jude Children's Research Hospital · Stand Up To Cancer · Storm the Heavens Fund · The ChadTough Defeat DIPG Foundation · The Hospital for Sick Children · Tough2gether Foundation · University of California, San Francisco · Vigeo Therapeutics, Inc. · Weill Medical College of Cornell University · Will Meeker Foundation

Trials 15 · a red edge is where a trial was stopped

today NCT07447076 · Ph 2 NCT07287202 · Ph 1/2 NCT07391215 · Ph 1/2 NCT06208657 · Ph 1/2 NCT05183204 · Ph 2 NCT05009992 · Ph 2 NCT05012670 · Ph 1 NCT04906096 · Ph 2 NCT04192981 · Ph 1 NCT03994796 · Ph 2 NCT03970447 · Ph 2/3 NCT03765983 · Ph 2 NCT03696355 · Ph 1 NCT03522298 · Ph 2 NCT01547546 · Ph 1

2012201320142015201620172018201920202021202220232024202520262027202820292030203120322033203420352036

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 14 trials
Phase 1	NCT05012670	Aug 2021 → Mar 2023	—	Kazia Therapeutics Limited	Completed	No outcome recorded
Phase 1	NCT04192981	Dec 2019 → Dec 2026 expected	metastatic malignant neoplasm in the brain, neoplastic meningitis	Memorial Sloan Kettering Cancer Center	Active not recruiting	No outcome recorded
Phase 1	NCT03696355	Nov 2018 → Apr 2021	central nervous system neoplasm	St. Jude Children's Research Hospital	Completed	No outcome recorded
Phase 1	NCT01547546	Apr 2012 → Jan 2015	glioblastoma	Genentech, Inc.	Completed	No outcome recorded
Phase 1/23 trials
Phase 1/2	NCT07287202	Mar → Dec 2026 expected	hemimegalencephaly, isolated focal cortical dysplasia type II, tuberous sclerosis	Sovargen	Not yet recruiting	No outcome recorded
Phase 1/2	NCT07391215	Jan 2026 → Jan 2029 expected	glioblastoma	Institute of Cancer Research, United Kingdom	Recruiting	No outcome recorded
Phase 1/2	NCT06208657	Jul 2024 → Dec 2030 expected	brain cancer, childhood malignant neoplasm, refractory malignant neoplasm	Australian & New Zealand Children's Haematology/Oncology Group	Recruiting	No outcome recorded
Phase 27 trials
Phase 2	NCT07447076	Jun 2026 → Dec 2035 expected	atypical teratoid rhabdoid tumor	Sabine Mueller, MD, PhD	Recruiting	No outcome recorded
Phase 2	NCT05183204	Feb 2022 → Jun 2026	glioblastoma	Weill Medical College of Cornell University	Suspended	No outcome recorded
Phase 2	NCT05009992	Oct 2021 → Dec 2027 expected	diffuse midline glioma, H3 K27M-mutant, grade III glioma	University of California, San Francisco	Recruiting	No outcome recorded
Phase 2	NCT04906096	Jun 2021 → Dec 2026 expected	non-Hodgkin lymphoma, primary central nervous system lymphoma	Lakshmi Nayak, MD	Recruiting	No outcome recorded
Phase 2	NCT03994796	Oct 2019 → Oct 2026 expected	NTRK fusion positive cancer, metastatic malignant neoplasm in the brain	Alliance for Clinical Trials in Oncology	Active not recruiting	No outcome recorded
Phase 2	NCT03765983	Feb 2019 → Apr 2024	breast cancer	Dana-Farber Cancer Institute	Terminated	No outcome recorded
Phase 2	NCT03522298	May 2018 → Mar 2023	adult glioblastoma	Kazia Therapeutics Limited	Completed	No outcome recorded
Phase 2/31 trial
Phase 2/3	NCT03970447	Jul 2019 → Jun 2028 expected	glioblastoma	Global Coalition for Adaptive Research	Recruiting	No outcome recorded

Evidence & citations 10 cited values

Every value below carries the sentence it was read from. 18 sources stand behind the page.

Field	Value	Cited text
Known as	Paxalisib	ClinicalTrials.gov intervention name — accepted as the source's own label NCT04906096 ↗ 9 NCT06208657 ↗ NCT07447076 ↗ NCT05009992 ↗ NCT03522298 ↗ NCT07391215 ↗ NCT03970447 ↗ NCT03994796 ↗ NCT05183204 ↗ NCT05012670 ↗
Known as	G-02441729	ChEMBL registry synonym — accepted as the source's own label CHEMBL3813842 ↗
Known as	GDC-0084	“Patients will be dosed orally with paxalisib (GDC-0084) capsules (15-mg each) at the dose and schedule to which they are assigned.” NCT03522298 ↗ 4 NCT04192981 ↗ NCT03765983 ↗ NCT03696355 ↗ NCT01547546 ↗
Known as	RG 7666	ChEMBL registry synonym — accepted as the source's own label CHEMBL3813842 ↗
Known as	Sim-0395	ChEMBL registry synonym — accepted as the source's own label CHEMBL3813842 ↗
Known as	SVG103	ClinicalTrials.gov intervention name — accepted as the source's own label NCT07287202 ↗
Action	Inhibit	“inhibitor of PI3K and mTOR” PMID 32269051 ↗ Apr 2020
Modality	Small molecule	“small-molecule inhibitor” PMID 32269051 ↗ Apr 2020 1 CHEMBL3813842 ↗
Route	Oral	“GDC-0084 was administered orally, once daily” PMID 31937616 ↗ Jan 2020
Target	MTOR	“inhibitor of PI3K and mTOR” PMID 32269051 ↗ Apr 2020