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Therapy Name Nirogacestat
Synonyms
Therapy Description

Ogsiveo (nirogacestat) is a small molecule that selectively binds to Gamma-secretase and inhibits its proteolytic activity on downstream targets including NOTCH receptor proteins, thus blocking NOTCH pathway activation in cancer (PMID: 28887726, PMID: 31211955). Ogsiveo (nirogacestat) is FDA-approved for use in patients with progressing desmoid tumors (FDA.gov).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Nirogacestat Ogsiveo PF-03084014|PF 03084014|PF03084014 Gamma secretase inhibitor 13 Ogsiveo (nirogacestat) is a small molecule that selectively binds to Gamma-secretase and inhibits its proteolytic activity on downstream targets including NOTCH receptor proteins, thus blocking NOTCH pathway activation in cancer (PMID: 28887726, PMID: 31211955). Ogsiveo (nirogacestat) is FDA-approved for use in patients with progressing desmoid tumors (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
APC R1450* APC LOH desmoid tumor predicted - sensitive Nirogacestat Case Reports/Case Series Actionable In a clinical case study, Ogsiveo (nirogacestat) resulted in tumor regression in a pediatric patient with a desmoid tumor harboring APC R1450* and loss of the APC heterozygosity who stayed on treatment for 6 months (PMID: 32762028). 32762028
APC inact mut desmoid tumor predicted - sensitive Nirogacestat Case Reports/Case Series Actionable In a clinical case study, Ogsiveo (nirogacestat) resulted in stable disease lasting 9 months in a patient with a patient with a desmoid tumor harboring an inactivating APC mutation (PMID: 32762028). 32762028

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT01981551 Phase II Nirogacestat Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis Active, not recruiting USA 0
NCT05348356 Phase II Nirogacestat Nirogacestat in Ovarian Granulosa Cell Tumors Active, not recruiting USA | CAN 1
NCT02955446 Expanded access Nirogacestat Compassionate Use Protocol for PF-03084014 in Patients With Advanced Solid Tumor Malignancies No longer available USA 0
NCT05949099 Phase II Nirogacestat Study of Cryoablation and Nirogacestat for Desmoid Tumor Recruiting USA 0
NCT03785964 Phase III Nirogacestat Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (DeFi) Active, not recruiting USA | ITA | GBR | DEU | CAN | BEL 1
NCT04195399 Phase II Nirogacestat A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery Active, not recruiting USA | CAN | AUS 2
NCT02137564 Phase II Nirogacestat Gamma Secretase Inhibitor PF-03084014 in Treating Patients With AIDS-Associated Kaposi Sarcoma Withdrawn 0
NCT05879146 Phase II Nirogacestat Evaluation of the Response and Non-response of Nirogacestat in Desmoid Tumors- Clinical Study Not yet recruiting USA 0
NCT02109445 Phase II Gemcitabine + Nab-paclitaxel Nirogacestat 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
NCT02299635 Phase II Nirogacestat A Study Evaluating PF-03084014 In Patients With Advanced Breast Cancer With Or Without Notch Alterations Terminated USA | ITA | GBR | ESP 2


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