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 | 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"
Sorting
- 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
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| Molecular Profile | Indication/Tumor Type | Response Type | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
|---|---|---|---|---|---|---|---|
| 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 |
| 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 |
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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- Click on any column header arrows to sort by that column
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| Clinical Trial | Phase | Therapies | Title | Recruitment Status |
|---|---|---|---|---|
| NCT02955446 | Expanded access | PF-03084014 | Compassionate Use Protocol for PF-03084014 in Patients With Advanced Solid Tumor Malignancies | No longer available |
| 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 |
| NCT03785964 | Phase III | PF-03084014 | Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (DeFi) | Active, not recruiting |
| NCT02137564 | Phase II | PF-03084014 | Gamma Secretase Inhibitor PF-03084014 in Treating Patients With AIDS-Associated Kaposi Sarcoma | Withdrawn |
| NCT02299635 | Phase II | PF-03084014 | A Study Evaluating PF-03084014 In Patients With Advanced Breast Cancer With Or Without Notch Alterations | Terminated |
| 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 |
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