Therapy Detail

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Therapy Name Nirogacestat
Synonyms
Therapy Description

Nirogacestat (PF-03084014) 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).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Nirogacestat PF-03084014|PF 03084014|PF03084014 Gamma secretase inhibitor 13 Nirogacestat (PF-03084014) 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).

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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  • Generally, the default sort order for tables is set to be first column ascending, however, specific tables may set a different default sort order.
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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, Nirogacestat (PF-03084014) 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, Nirogacestat (PF-03084014) 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

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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  • Generally, the default sort order for tables is set to be first column ascending, however, specific tables may set a different default sort order.
  • Click on any column header arrows to sort by that column
  • Hold down the Shift key and click multiple columns to sort by more than one column, be sure to set ascending or descending order for a given column, before moving on to the next column.

Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT05348356 Phase II Nirogacestat Nirogacestat in Ovarian Granulosa Cell Tumors Recruiting USA | ESP | CAN 1
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
NCT03785964 Phase III Nirogacestat Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (DeFi) Active, not recruiting USA | ITA | DEU | CAN | BEL 2
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 3
NCT02137564 Phase II Nirogacestat Gamma Secretase Inhibitor PF-03084014 in Treating Patients With AIDS-Associated Kaposi Sarcoma Withdrawn 0
NCT02955446 Expanded access Nirogacestat Compassionate Use Protocol for PF-03084014 in Patients With Advanced Solid Tumor Malignancies No longer available USA 0
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
NCT02299635 Phase II Nirogacestat A Study Evaluating PF-03084014 In Patients With Advanced Breast Cancer With Or Without Notch Alterations Terminated USA | ITA | ESP 3


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