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"
Sorting
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- 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 |
|---|---|---|---|---|---|---|---|
| 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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| 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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