Study of Cryoablation and Nirogacestat for Desmoid Tumor
Detailed Description:
The primary purpose of this protocol is treatment. Systemic therapy with oral study agent, nirogacestat, will be given for 3 cycles (1 cycle = 28 days) followed by a single cryoablation procedure between Cycles 3 and 4, and then continued nirogacestat for Cycles 4 through 26. Treatment with nirogacestat may continue via this study for up to 24 months (26 cycles of treatment), unless there is progression of disease, intolerance, subject withdraws their consent, start of a new anticancer therapy, or until Subject Study Completion or Termination occurs.
Primary Outcome Measures :
Clinical Benefit per RECIST v1.1 [ Time Frame: 1 year ]
Clinical benefit (CB) is defined as the number of participants assessed with a complete response (CR), partial response (PR), or stable disease (SD) within 1 year and with no evidence of loss of response nor progression within that year. Response & progression will be assessed according to the Revised Response Evaluation Criteria in Solid Tumors (RECIST)v1.1, as follows:
RECIST v1.1:
CR=Disappearance of all lesions
PR=≥30% decrease in diameter of target lesions
Progressive disease (PD)=20% increase in diameter of target lesion; progression of non-target lesion; or ≥1 new lesion(s)
- For the overall outcome:
SD=Changes not meeting above criteria
Overall Response (OR)=CR+PR
CB=CR+PR+SD
Secondary Outcome Measures :
Clinical Benefit per mRECIST [ Time Frame: 1 year ]
Clinical benefit (CB) is defined as the number of participants assessed with a complete response (CR), partial response (PR), or stable disease (SD) within
1 year and with no evidence of loss of response nor progression within that year.
Response & progression will be assessed according to the modified Revised Response Evaluation Criteria in Solid Tumors (mRECIST), as follows.
CR=Disappearance of intratumoral enhancement
PR=≥ 30% decrease in the diameter of target lesion(s)
PD=An increase of >20% in the diameter of target lesion. The outcome is the number of participants alive after 1 year and without PD per mRECIST, a number without dispersion
Progression-free Survival (PFS) [ Time Frame: 1 year ]
Progressive-free survival (PFS) is an assessment of the number of participants at a specific time who remain alive and without tumor progression. Progressive disease (PD) will be assessed according to Revised Response Evaluation Criteria in Solid Tumors (RECIST)v1.1, or, for tumors assessed with MRI enhancement, modified RECIST (mRECIST) defined as follows:
RECIST v1.1:
PD=20% increase in diameter of target lesion; progression of non-target lesion; or ≥1 new lesion(s) mRECIST:
PD=An increase of >20% in the diameter of target lesion. The outcome is the number of participants alive after 1 year and without PD per RECIST or mRECIST, a number without dispersion.
Objective Response [ Time Frame: 1 year ]
Objective response (OR) is defined as the number of participants assessed with a complete response (CR) or partial response (PR) within 1 year and with no evidence of loss of response nor progression within that year. Response & progression will be assessed according to the Revised Response Evaluation Criteria in Solid Tumors (RECIST)v1.1, or, for assessed with MRI enhancement, modified RECIST (mRECIST).
Time-to-Response (TTR) [ Time Frame: 1 year ]
Time-to-response (TTR) is an assessment of the length of time from the start of treatment until clinical response (complete response, CR; or partial response, PR) is documented.in accordance with the Revised Response Evaluation Criteria in Solid Tumors (RECIST)v1.1, or, for assessed with MRI enhancement, modified RECIST (mRECIST) defined as follows:’