Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Nirogacestat Hydrobromide · 1 trial · 3 indications
Will be estimated using the Kaplan-Meier method with the 95% confidence interval estimated by the Peto-Peto method.
Will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. All grade 3 or above toxicities deemed related to study drug will be summarized. All grade 1 and 2 toxicities observed in \> 5% of participants and deemed related to study drug will be reported.
PK parameters of nirogacestat will be defined to quantify systemic exposure, drug clearance, terminal half-life and other pharmacokinetic characteristics. These PK parameters will be summarized with descriptive statistics, including means, medians, ranges, and standard deviations.
PK parameters of nirogacestat will be defined to quantify systemic exposure, drug clearance, terminal half-life and other pharmacokinetic characteristics. These PK parameters will be summarized with descriptive statistics, including means, medians, ranges, and standard deviations.
PK parameters of nirogacestat will be defined to quantify systemic exposure, drug clearance, terminal half-life and other pharmacokinetic characteristics. These PK parameters will be summarized with descriptive statistics, including means, medians, ranges, and standard deviations.
| Arm | Type | Description |
|---|---|---|
| Treatment (nirogacestat) | EXPERIMENTAL | Patients receive nirogacestat PO BID on days 1-28. Cycles repeats every 28 days in the absence of disease progression or unacceptable toxicity. Patients undergo ECHO and CT or MRI on study. Patients may also undergo x-ray imaging and blood sample collection on study. |
| Name | Type | Description |
|---|---|---|
| Biospecimen Collection | PROCEDURE | Undergo blood sample collection |
| Computed Tomography | PROCEDURE | Undergo CT |
| Echocardiography Test | PROCEDURE | Undergo ECHO |
| Magnetic Resonance Imaging | PROCEDURE | Undergo MRI |
| Nirogacestat Hydrobromide | DRUG | Given PO |
| Quality-of-Life Assessment | OTHER | Ancillary studies |
| Questionnaire Administration | OTHER | Ancillary studies |
| X-Ray Imaging | PROCEDURE | Undergo x-ray |
Inclusion Criteria: * Patients must be \> 12 months and \< 18 years of age at the time of enrollment * Patients must have a body surface area of \> 0.3 m\^2 at the time of enrollment * Existing or recurrent desmoid tumor that is deemed not amenable to surgery without significant morbidity and progr...
Nirogacestat Hydrobromide is an investigational small molecule being developed for the treatment of desmoid fibromatosis, including recurrent and unresectable desmoid tumors. It is currently in Phase 2 clinical development and has not been approved by the FDA.
Nirogacestat Hydrobromide is an inhibitor that targets multiple components of the gamma-secretase complex, including APH1A, PSENEN, APH1B, PSEN1, NCSTN, and PSEN2. By inhibiting these targets, it is being studied for its potential effect on desmoid fibromatosis.
Nirogacestat Hydrobromide is being developed by SpringWorks Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol SWTX.
Nirogacestat Hydrobromide is currently in Phase 2 clinical development. It has received several FDA designations, including Priority Review, Fast Track, Orphan Drug, and Breakthrough Therapy, but it remains investigational and is not yet FDA approved.
Nirogacestat Hydrobromide is being studied in a Phase 2 clinical trial with the identifier NCT04195399. This trial is active but not recruiting, with an enrollment of 31 participants, and is evaluating the drug for treating desmoid tumors that cannot be removed by surgery.