Health TrialsbyHealthPanel

Study of Cryoablation and Nirogacestat for Desmoid Tumor

ConditionDesmoid Tumor
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorNam Bui

About this trial

The primary purpose of this protocol is Systemic therapy with oral study agent, nirogacestat, followed by a single cryoablation procedure.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Histologically-confirmed diagnosis of desmoid tumor (DT) that is progressing (by RECIST criteria over the past 12 months) or symptomatic (as defined by change in pain regimen or impairment of activities of daily living (ADLs), or at investigator discretion). Note: Must have diagnosis of desmoid tumor on pathology report.

Desmoid tumor is 50 to <75% cryoablatable.

Proximity (< 1 cm) to critical structures, such as nerves, vessels, bowel, or skin, at risk of injury during ablation, despite hydrodissection

Large size ( > 100 mL)

Disqualifiers

Participant previously received or is currently receiving therapy with GS inhibitors or anti-Notch antibody therapy.

Participant is currently using any treatment for DT including tyrosine kinase inhibitors (TKIs), NSAIDS (chronic daily use - except as in inclusion criterion 4) or any investigational treatment 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study treatment.

Participant is currently using or anticipates using food or drugs that are known strong or moderate cytochrome P450 3A4 (CYP3A4) inhibitors, or strong CYP3a inducers within 14 days prior to the first dose of study treatment.

Participant has known hypersensitivity to the active substance or to any of the excipients of nirogacestat.

Trial design

Design model

Single group

Treatments tested in this trial

  • Nirogacestat

    Drug

    Nirogacestat 150 mg by mouth twice-daily continuously for Cycles 1 through 3. Treatment with nirogacestat may continue via this study protocol for up to 24 months (26 cycles of treatment), unless there is progression of disease, intolerance, start of new anticancer therapy, or Subject Study Completion or Termination occurs.

  • Cryoablation

    Procedure/Surgery

    Cryoablation procedure (single session) of one tumor mass between Cycles 3 and 4 of nirogacestat treatment

Treatment groups

28 Participants
are divided into 1 treatment group
Group A: Nirogacestat 150 mgExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Clinical Benefit per RECIST v1.1

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

Time frame
1 year

Secondary outcomes

1

Clinical Benefit per mRECIST

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

Time frame
1 year
2

Progression-free Survival (PFS)

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.

Time frame
1 year
3

Objective Response

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 frame
1 year
4

Time-to-Response (TTR)

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:' 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) mRECIST: * 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. For either: • SD=Changes not meeting above criteria The outcome is expressed as the median TTR (by RECIST or mRECIST), with standard deviation.

Time frame
1 year

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Nam Bui

Lead sponsor

Stanford University

Sponsor institution

Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany

Collaborator

Source ClinicalTrials.gov
Apply now