Therapy Detail

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Therapy Name Nirogacestat
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
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).

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  • 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
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
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

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 Nirogacestat Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (DeFi) Active, not recruiting USA | ITA | DEU | CAN | BEL 2
NCT02955446 Expanded access Nirogacestat Compassionate Use Protocol for PF-03084014 in Patients With Advanced Solid Tumor Malignancies No longer available 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
NCT05348356 Phase II Nirogacestat Nirogacestat in Ovarian Granulosa Cell Tumors Recruiting USA | CAN 1
NCT04195399 Phase II Nirogacestat A Study of a New Drug, Nirogacestat, for Treating Desmoid Tumors That Cannot be Removed by Surgery Recruiting USA | CAN 3
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
NCT02137564 Phase II Nirogacestat Gamma Secretase Inhibitor PF-03084014 in Treating Patients With AIDS-Associated Kaposi Sarcoma Withdrawn 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


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