Actively Recruiting
Phase II Study of Cryoablation Combined with Oral Nirogacestat for Desmoid Tumor Treatment
Led by Nam Bui · Updated on 2025-09-08
28
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
Sponsors
N
Nam Bui
Lead Sponsor
S
SpringWorks Therapeutics, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
This research aims to evaluate the treatment of desmoid tumors using a combination of oral systemic therapy with the study drug nirogacestat and a single cryoablation procedure. The study involves participants with progressing or symptomatic desmoid tumors, focusing on the effectiveness of this combined approach in managing the condition over an extended period. It is a Phase II clinical trial assessing treatment outcomes over up to 24 months. Participants will receive nirogacestat orally at a dose of 150 mg twice daily continuously for three 28-day cycles. After the third cycle, they will undergo a single cryoablation procedure targeting one tumor mass. Following this procedure, nirogacestat treatment will continue from cycle 4 through cycle 26, potentially lasting up to 24 months, unless there is disease progression, intolerance, withdrawal of consent, or initiation of new anticancer therapy. During the study, participants will have regular assessments including clinical evaluations and imaging to monitor tumor response using RECIST criteria. Researchers will track outcomes such as clinical benefit, progression-free survival, objective response, time-to-response, duration of response, and any related adverse events for one year. Participants will also undergo laboratory tests and safety monitoring throughout the treatment period, which may last up to two years.
CONDITIONS
Brief Title
Study of Cryoablation and Nirogacestat for Desmoid Tumor
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Histologically confirmed diagnosis of desmoid tumor that is progressing or symptomatic
- Tumor is 50% to less than 75% suitable for cryoablation in a single session
- Completed any prior desmoid tumor therapy at least 28 days before starting study treatment with resolved toxicities
- Stable dose of chronic nonsteroidal anti-inflammatory drugs if used for conditions other than desmoid tumor
- Age 18 years or older
- ECOG performance status of 2 or less at screening
- Adequate organ and bone marrow function based on specified laboratory values
- Negative pregnancy test for women of childbearing potential at screening
- Ability to swallow tablets
- Ability to undergo MRI
- Willingness and ability to provide informed consent
- Use of contraception consistent with study requirements during and after treatment
You will not qualify if you...
- Prior or current treatment with GS inhibitors or anti-Notch antibody therapy
- Use of any desmoid tumor treatment including tyrosine kinase inhibitors, chronic NSAIDs (except as allowed), or investigational treatments within 28 days before study treatment
- Use or anticipated use of strong or moderate CYP3A4 inhibitors or inducers within 14 days before study treatment
- Known allergy to nirogacestat or its ingredients
- Active or chronic infection at consent or screening
- Known malabsorption syndrome or gastrointestinal conditions impairing drug absorption
- History of high-grade malignancy within the past 2 years, with exceptions
- Significant cardiac events or conditions within 6 months prior to consent
- Congenital long QT syndrome or risk factors for Torsades de pointes
- Current or chronic liver disease or biliary abnormalities (except specific exceptions)
- Participation in another investigational drug or device study within 28 days before study treatment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 24 months (26 cycles of 28 days each)
Participants receive oral nirogacestat continuously for up to 26 cycles (each cycle is 28 days). A single cryoablation procedure is performed between Cycles 3 and 4. Treatment may continue for up to 24 months unless disease progresses or other reasons for discontinuation occur.
Continuous oral medication with 1 cryoablation procedure between Cycles 3 and 4
Trial Site Locations
Total: 1 location
1
Stanford University
Palo Alto, California, United States, 94395
Actively Recruiting
Research Team
P
Priyanka Reddy
A
Amir Emami
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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