Aggressive Fibromatosis (Desmoid Tumor)

Researchers are evaluating the drug ES014 in adults with desmoid tumors in this open-label, multicenter, single-arm phase 2 clinical study. The study aims to assess the effectiveness, safety, tolerability, patient-reported outcomes, pharmacokinetics, pharmacodynamics, and immune response to ES014 in this patient population. Participants will receive ES014 through intravenous infusion at doses of either 700 mg or 1400 mg every 14 days. The study design includes two experimental arms with these dosing regimens. Treatment will be given over a period of 1 to 3 years while monitoring the patients closely for response and side effects. During the study, participants will undergo evaluations including tumor response assessments according to RECIST 1.1 criteria, safety and tolerability checks, pharmacokinetic testing, and immune response measurements. Researchers will track patient-reported outcomes and monitor for adverse effects throughout the study duration. The primary outcome is the objective response rate measured by an independent review committee over 1 to 3 years.

Desmoid fibromatoses are rare, locally aggressive tumors that arise from mesenchymal tissue. This research focuses on evaluating chemoembolization using Doxorubicin delivered through arterial embolization as a treatment for symptomatic and actively growing desmoid fibromatosis, especially in patients unsuitable for surgery or cryoablation. Current treatment guidelines favor active surveillance for asymptomatic cases and reserve chemotherapy for more severe presentations due to associated systemic risks. The study investigates chemoembolization, a procedure involving selective catheterization of arteries supplying the tumor and injecting hydrogel microspheres loaded with Doxorubicin. This approach aims to deliver high drug concentrations directly to the tumor site while minimizing systemic exposure. The procedure includes diagnostic arteriography to confirm successful embolization by occluding over 90% of the tumor's blood supply. Participants will be monitored over one year to assess outcomes such as tumor volume reduction, pain relief using VAS scores, and decreased need for pain medications. Quality of life improvements will also be evaluated using standardized questionnaires. Safety and procedural success will be tracked through imaging and clinical assessments throughout the study, with the total participation duration spanning one year.

Researchers are evaluating treatments for desmoid tumors (DT) that progress after an initial period of watchful waiting, which is currently the standard care. This phase II randomized trial compares cryoablation, a procedure that freezes the tumor, with medical therapy using chemotherapy drugs. The trial also includes a cross-over design, allowing patients to receive cryoablation if needed after initial treatment. Participants are assigned to one of two groups: one receives a single cryoablation procedure on Day 0, while the other receives chemotherapy chosen by the investigator. Chemotherapy options include methotrexate plus vinblastine given weekly by intravenous infusion for the first 6 months and less frequently afterward, or vinorelbine taken orally for 12 months, with doses adjusted for adults and adolescents. The study aims to assess treatment effects over several months, including the possibility of switching treatments. During the study, participants undergo regular assessments including MRI scans to measure tumor response, pain evaluations, quality of life questionnaires, and monitoring for side effects. Researchers will measure the rate of disease control at 12 months and track progression-free survival and treatment-related adverse events up to 39 months. Health economics and patient-reported outcomes are also evaluated, with ongoing follow-up to understand long-term effects.

Desmoid tumors are benign growths that can recur locally after surgery and have an unpredictable course. Because of this, treatment has shifted away from surgery toward careful observation or medical therapies such as hormonal treatments and low-dose chemotherapy. This study aims to evaluate cryotherapy as a minimally invasive treatment option to avoid chemotherapy, especially in younger patients with desmoid tumors. The treatment under study is cryotherapy, which involves using anesthesia and imaging-guided percutaneous probes to freeze the tumor tissue. The probes create ice spheres up to 3 cm in size to thoroughly treat the tumor. This procedure is done under CT control to ensure accuracy and safety. Participants will be monitored to assess their response to cryotherapy starting from the baseline before treatment. The study measures how the tumor responds to this treatment over time. Participants will undergo necessary evaluations and imaging to track treatment effects and safety. The total duration and follow-up details are based on individual patient progress and response.

Researchers are evaluating FOG-001 for safety, tolerability, pharmacokinetics, pharmacodynamics, and anti-tumor activity in people with locally advanced or metastatic solid tumors that likely or known to have mutations activating the Wnt pathway. This first-in-human, phase 1/2, multicenter study includes dose escalation and expansion phases to assess FOG-001 alone and in combination with other cancer drugs. FOG-001 is given either intravenously or subcutaneously in continuous 28-day cycles. Some participants receive FOG-001 alone, while others receive it combined with mFOLFOX-6, Nivolumab, Trifluridine/tipiracil, or Bevacizumab according to prescribing information. Multiple study parts include treatment of various cancers such as colorectal, hepatocellular carcinoma, desmoid tumors, and prostate cancer, based on specific mutation status. Participants will undergo safety evaluations including monitoring for adverse events and dose-limiting toxicities over approximately 10 months. Tumor response is assessed regularly using imaging criteria, along with specific measurements like PSA30 for prostate cancer. Pharmacokinetic and pharmacodynamic tests are done during early cycles. The study involves frequent visits for treatment administration, scans, lab tests, biopsies, and clinical assessments to track treatment effects and safety throughout the study period.

Researchers are observing the use and performance of the Galvanize Pulsed Electric Field (PEF) technology in real-world settings for patients with soft tissue lesions. The study aims to understand how often and how well the PEF technology is used, monitor safety outcomes, and provide information to improve future versions of the device. This observational registry includes patients who have undergone or are scheduled to undergo PEF ablation. Participants will receive PEF ablation treatment according to their institution's standard care practices. The study follows patients either prospectively or retrospectively, with a total follow-up period of two years starting from their PEF procedure date. Up to 200 patients across the US will be enrolled, and their treatments will be monitored but not altered by the study. During the study, participants will be followed according to their usual care plans, with researchers collecting information on the type of anesthesia used, the target location and size of the PEF treatment area, and any serious or unexpected adverse events within 30 days after the procedure. The study will track outcomes through imaging and standard follow-up visits as determined by the treating physician. Participants will be involved for up to two years to gather long-term safety and performance data on the device.

This research aims to collect detailed information on the use of cryoablation procedures in children with soft tissue and bone lesions. Cryoablation is a minimally invasive technique that uses image guidance to insert specialized needles or probes into lesions, applying cycles of freezing and thawing to destroy tissue. While this method is well established for adults, especially in treating kidney tumors, its use in children is still new and not well documented. The study will audit the range of applications, success rates, safety, complications, and effectiveness of cryoablation in the pediatric population. The study involves children undergoing cryoablation therapy for benign soft tissue lesions. Cryoablation uses a mechanized unit connected to probes that freeze and thaw the targeted tissue via highly pressurized gases. This process causes tissue destruction both mechanically and through immune responses. The registry collects data from multiple centers in the UK and Europe, gathering standardized information about each procedure. Data will be anonymized and entered into a web-based system for analysis. The study is open and non-controlled, focusing on repeat treatments as determined by clinical decisions. Participants will be followed for up to five years to monitor outcomes such as changes in lesion size, symptom changes, procedure failures, and any complications within two years after treatment. The registry aims to provide evidence on safety, efficacy, and best practice for pediatric cryoablation. Data will be analyzed collectively while remaining owned by individual hospitals. This will help improve treatment guidance and support future clinical trials if needed. The total participation duration varies depending on treatment and follow-up schedules.

This research aims to study how nirogacestat may impact ovarian function in adult premenopausal women who have progressing desmoid tumors or aggressive fibromatosis. These rare, slow-growing soft tissue tumors are locally invasive and, despite being benign, can cause serious health problems. Nirogacestat is a tumor inhibitor approved in the USA for adults with progressing desmoid tumors needing systemic treatment, and this open-label Phase 4 study evaluates its effects on ovarian toxicity and overall safety in this population. Participants will receive nirogacestat tablets taken by mouth at a dose of 150 mg twice daily. The study includes up to 24 cycles of treatment, with each cycle lasting 28 days, followed by a clinical follow-up period of up to 2 years. During this time, researchers will assess ovarian function recovery, incidence of ovarian toxicity events, and other adverse effects related to the treatment. Throughout the study, participants will regularly provide hormone level blood samples timed to their menstrual cycle and complete menstrual diaries and electronic patient-reported outcome questionnaires. Researchers will monitor ovarian toxicity events and treatment safety using standardized criteria over the treatment and follow-up periods. The total involvement may last up to nearly three years, allowing detailed evaluation of ovarian function recovery and treatment tolerability.

Researchers are observing people with desmoid-type fibromatosis, a condition involving abnormal growth of fibrous tissue, to understand its progression over a period of at least one month. The study includes individuals newly diagnosed or with newly recurrent disease, aiming to monitor the condition without immediate treatment intervention. This observational study is sponsored by Memorial Sloan Kettering Cancer Center and focuses on tracking disease changes over time. Participants will undergo cross-sectional imaging using MRI with gadolinium contrast as the preferred method. For those unable to have MRI, CT scans with intravenous contrast will be used instead. The study involves active observation, with imaging assessments carried out to measure tumor size and progression. This approach allows researchers to evaluate the natural course of desmoid fibromatosis while participants are managed without systemic treatment. During the study, participants will have baseline and follow-up imaging scans to monitor tumor size and disease progression. Researchers will assess progression-free survival at 12 months as the primary outcome. Participants must be able to undergo imaging tests and provide informed consent. The study will monitor safety and disease status throughout the observation period, which may extend beyond the initial month based on clinical follow-up and imaging results.

Researchers are evaluating the effectiveness of percutaneous needle fasciotomy with or without corticosteroid injection for treating Dupuytren's contracture affecting the metacarpophalangeal (MCP) joints. This randomized controlled trial aims to compare these two treatment options in patients aged 45 to 99 years with this joint condition. The study is led by Regionshospitalet Silkeborg and is designed as a Phase 4 clinical trial with double masking. The study includes two treatment groups: one group will receive percutaneous needle fasciotomy combined with corticosteroid injection (Depo-Medrol), and the other group will receive the same fasciotomy combined with a saline injection as a placebo comparator. The fasciotomy procedure follows the Lermusiaux and Debeyre technique. Participants must have sufficient correction of the MCP joint during treatment, with less than 20 degrees passive extension deficit. Participants will be monitored for outcomes including the recurrence rate of contracture after two years. Assessments will focus on the correction of joint contracture, with follow-up visits to measure the effectiveness and safety of the treatments. The trial will also evaluate secondary outcomes related to joint function and patient well-being over the study period ending in March 2030.