Therapy Detail

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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).

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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).

Filtering

  • Case insensitive filtering will display rows where any text in any cell matches the filter term
  • Simple literal full or partial string matches
  • Separate multiple filter terms with a spaces, order doesn't matter (a b c and c b a are equivalent )
  • Filtering will only apply to rows that are already loaded on the page, filtering has no impact on query parameters
  • Use quotes to match a longer phrase which contains spaces "mtor c1483f"

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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

Filtering

  • Case insensitive filtering will display rows where any text in any cell matches the filter term
  • Simple literal full or partial string matches
  • Separate multiple filter terms with a spaces, order doesn't matter (a b c and c b a are equivalent )
  • Filtering will only apply to rows that are already loaded on the page, filtering has no impact on query parameters
  • Use quotes to match a longer phrase which contains spaces "mtor c1483f"

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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


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