23 Participants Needed

Cryoablation + Nirogacestat for Desmoid Tumors

MA
BA
BP
AH
Overseen ByAmir H Emami
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Breakthrough TherapyThis drug has been fast-tracked for approval by the FDA given its high promise

Trial Summary

What is the purpose of this trial?

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

Do I need to stop my current medications to join the trial?

Yes, if you are currently taking any treatment for desmoid tumors, you must stop at least 28 days before starting the study treatment. If you are on chronic NSAIDs for other conditions, you need to be on a stable dose for at least 28 days before starting the study treatment.

What data supports the effectiveness of the treatment Cryoablation + Nirogacestat for Desmoid Tumors?

Research shows that cryoablation, a treatment that uses extreme cold to destroy abnormal tissue, is effective and safe for managing desmoid tumors, especially when surgery isn't an option. Multiple studies highlight its success in controlling tumor growth and providing pain relief.12345

Is cryoablation safe for treating desmoid tumors?

Research shows that cryoablation, a procedure that uses extreme cold to destroy tissue, has been studied for its safety in treating desmoid tumors. Studies indicate it is generally safe, with a focus on its tolerability and effectiveness in managing these tumors.12346

What makes the treatment with Nirogacestat for Desmoid Tumors unique?

Nirogacestat is a unique treatment for Desmoid Tumors because it is a gamma-secretase inhibitor, which targets a specific pathway involved in tumor growth, unlike traditional treatments that may not specifically target this pathway. Additionally, combining it with cryoablation (a procedure that freezes and destroys abnormal tissue) offers a novel approach that may enhance treatment effectiveness.7891011

Research Team

NB

Nam Bui, M.D.

Principal Investigator

Stanford University

Eligibility Criteria

Adults with a confirmed diagnosis of desmoid tumor that is either growing or causing symptoms, and who can swallow tablets. The tumor must be partially treatable by cryoablation without harming nearby critical structures. Participants need proper organ function, no severe cardiac issues or recent heart attacks, and cannot be on certain medications. Women must not be pregnant or breastfeeding and use contraception; men agree to use condoms.

Inclusion Criteria

I can swallow pills.
My desmoid tumor can be mostly treated with freezing.
Women of childbearing potential must have a negative serum pregnancy test at screening
See 10 more

Exclusion Criteria

I haven't taken strong or moderate CYP3A4 inhibitors or inducers in the last 14 days.
Current enrollment or recent enrollment in another clinical study with any investigational drug or device
Known hypersensitivity to nirogacestat
See 11 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Systemic therapy with oral study agent, nirogacestat, for 3 cycles (1 cycle = 28 days) followed by a single cryoablation procedure, then continued nirogacestat for Cycles 4 through 26

24 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 year

Treatment Details

Interventions

  • Cryoablation
  • Nirogacestat
Trial OverviewThe trial tests the effectiveness of nirogacestat taken orally followed by a single cryoablation procedure for treating desmoid tumors. Nirogacestat is given first to potentially shrink the tumor before freezing it with cryoablation to destroy remaining cancer cells.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Nirogacestat 150 mgExperimental Treatment2 Interventions
Patients will receive 3-cycle lead-in with systemic therapy with nirogacestat 150 mg po BID, given continuously.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Stanford University

Lead Sponsor

Trials
2,527
Recruited
17,430,000+

SpringWorks Therapeutics, Inc.

Industry Sponsor

Trials
14
Recruited
900+

Findings from Research

Percutaneous image-guided cryoablation is a safe and effective treatment for inoperable extra-abdominal desmoid tumors, with a major complication rate of only 5.8% and a disease-free survival rate of 82.3% over 24 months.
Although 47% of patients had residual tumors after treatment, there was no local tumor progression observed at 6, 12, or 24 months, indicating good local control of the disease.
Percutaneous image-guided cryoablation in inoperable extra-abdominal desmoid tumors: a study of tolerability and efficacy.Havez, M., Lippa, N., Al-Ammari, S., et al.[2020]
Cryoablation has been successfully performed on 10 patients with desmoid fibromatosis, showing a reproducible technique with low toxicity and significant symptomatic improvement in 8 out of 10 patients after a median follow-up of 353 days.
Larger tumors required more resources and had a higher chance of leaving residual tumor tissue, but complete ablation led to no recurrences or need for further treatment, indicating the importance of fully covering the tumor during the procedure.
Percutaneous cryoablation for desmoid fibromatosis: initial experience at a UK centre.Johnston, EW., Alves, A., Messiou, C., et al.[2022]
Cryoablation was found to be an effective treatment for desmoid tumors, with a significant reduction in viable tumor volume (43.7%) after the procedure, and 96.9% of patients experienced symptomatic relief.
The procedure was safe, with a technical success rate of 100% and only one major complication reported among 44 cryoablation procedures performed on 25 patients over a 10-year period.
A Single-Center 10-Year Retrospective Analysis of Cryoablation for the Management of Desmoid Tumors.Yan, YY., Walsh, JP., Munk, PL., et al.[2021]

References

Percutaneous image-guided cryoablation in inoperable extra-abdominal desmoid tumors: a study of tolerability and efficacy. [2020]
Percutaneous cryoablation for desmoid fibromatosis: initial experience at a UK centre. [2022]
A Single-Center 10-Year Retrospective Analysis of Cryoablation for the Management of Desmoid Tumors. [2021]
Percutaneous cryoablation for advanced and refractory extra-abdominal desmoid tumors. [2021]
Local Control and Analgesic Efficacy of Percutaneous Cryoablation for Desmoid Tumors. [2020]
Prognostic Factors for Local Recurrence after Cryoablation of Desmoid Tumors. [2023]
Locoregional regulatory peptide receptor targeting with the diffusible somatostatin analogue 90Y-labeled DOTA0-D-Phe1-Tyr3-octreotide (DOTATOC): a pilot study in human gliomas. [2018]
Temozolomide and radiotherapy as first-line treatment of high-grade gliomas. [2022]
Feasibility and Therapeutic Potential of Peptide Receptor Radionuclide Therapy for High-Grade Gliomas. [2023]
10.United Statespubmed.ncbi.nlm.nih.gov
Treatment of pituitary neoplasms with temozolomide: a review. [2018]
11.United Statespubmed.ncbi.nlm.nih.gov
Receptor radionuclide therapy with 90Y-DOTATOC in patients with medullary thyroid carcinomas. [2013]