Missing content? – Request curation!
Request curation for specific Genes, variants, or PubMed publications.
Have questions, comments or suggestions? - Let us know!
Email us at : ckbsupport@jax.org
Therapy Name | PF-03084014 |
Synonyms | |
Therapy Description |
Nirogacestat (PF-03084014) is a small molecule that selectively binds and inhibits Gamma-secretase inhibiting its proteolytic activity on downstream targets including NOTCH receptor proteins thus blocking pathway activation in cancer (NCI Drug Dictionary). |
Drug Name | Trade Name | Synonyms | Drug Classes | Drug Description |
---|---|---|---|---|
PF-03084014 | Nirogacestat|PF 03084014|PF03084014 | Gamma secretase inhibitor 12 | Nirogacestat (PF-03084014) is a small molecule that selectively binds and inhibits Gamma-secretase inhibiting its proteolytic activity on downstream targets including NOTCH receptor proteins thus blocking pathway activation in cancer (PMID: 28887726, PMID: 31211955). |
Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|
NOTCH1 mutant | triple-receptor negative breast cancer | sensitive | PF-03084014 | Preclinical - Pdx | Actionable | In a preclinical study, PF-03084014 treatment resulted in tumor regression in patient-derived xenograft models of triple receptor negative breast cancer harboring NOTCH1 mutations (PMID: 25564152). | 25564152 |
NOTCH1 S2449fs | triple-receptor negative breast cancer | sensitive | PF-03084014 | Preclinical - Pdx | Actionable | In a preclinical study, PF-03084014 treatment resulted in tumor regression in patient derived xenograft animal models of triple receptor negative breast cancer harboring NOTCH1 S2449fs (PMID: 25564152). | 25564152 |
NOTCH1 act mut | colorectal cancer | sensitive | PF-03084014 | Preclinical - Cell culture | Actionable | In a preclinical study, the gamma secretase inhibitor PF-03084014 reduced Notch1 cleavage, decreased activation of Notch targets, and increased apoptosis in colorectal cancer cell lines exhibiting increased Notch activation (PMID: 23868008). | 23868008 |
Clinical Trial | Phase | Therapies | Title | Recruitment Status | Covered Countries | Other Countries |
---|---|---|---|---|---|---|
NCT03785964 | Phase III | PF-03084014 | Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (DeFi) | Active, not recruiting | USA | CAN | 5 |
NCT02137564 | Phase II | PF-03084014 | Gamma Secretase Inhibitor PF-03084014 in Treating Patients With AIDS-Associated Kaposi Sarcoma | Withdrawn | 0 | |
NCT02109445 | Phase II | Gemcitabine + Nab-paclitaxel PF-03084014 | Study Of PF-03084014 In Combination With Gemcitabine And Nab-Paclitaxel In Patients With Metastatic Pancreatic Adenocarcinoma Not Previously Treated With Anticancer Therapies | Terminated | USA | 0 |
NCT01981551 | Phase II | PF-03084014 | Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis | Active, not recruiting | USA | 0 |
NCT04195399 | Phase II | PF-03084014 | A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery | Recruiting | USA | 1 |
NCT02299635 | Phase II | PF-03084014 | A Study Evaluating PF-03084014 In Patients With Advanced Breast Cancer With Or Without Notch Alterations | Terminated | USA | 5 |
NCT02955446 | Expanded access | PF-03084014 | Compassionate Use Protocol for PF-03084014 in Patients With Advanced Solid Tumor Malignancies | No longer available | USA | 0 |