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 tumors 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 the safety and effectiveness of FOG-001 in people with advanced or metastatic solid tumors and familial adenomatous polyposis FAP, a genetic condition affecting the Wnt pathway. This Phase 12 trial studies FOG-001 alone and combined with other anticancer drugs in patients with tumors that have Wnt pathway activating mutations or related colorectal cancers. The trial aims to understand how the drug behaves in the body, its safety, and its ability to control or reduce tumors. Participants receive FOG-001 through intravenous or subcutaneous administration in continuous 28-day cycles. Some groups also receive combinations of FOG-001 with other cancer treatments like mFOLFOX-6, Nivolumab, Trifluridinetipiracil, or Bevacizumab. The study includes dose escalation and dose expansion phases to find the recommended dose and evaluate effects across different tumor types and genetic profiles. Different subgroups focus on specific cancers such as colorectal cancer, hepatocellular carcinoma, desmoid tumors, prostate cancer, and FAP. During the trial, participants undergo regular assessments including scans, biopsies, and blood tests to monitor safety, drug levels, and tumor response. Researchers measure side effects, tumor shrinkage, disease control, and specific cancer markers over about 10 months. The study tracks overall response using RECIST criteria and evaluates pharmacokinetics and pharmacodynamics. Safety is closely monitored, and participants may have follow-up evaluations after treatment cycles. The total duration varies by participant depending on treatment and response.

Researchers are observing the use and performance of Galvanize Pulsed Electric Field PEF technology in treating soft tissue lesions in a real-world setting. This multi-center registry study aims to answer key questions about how PEF technology is used, its performance, and safety outcomes to help develop future device generations. Up to 200 patients who undergo or have undergone PEF procedures will be enrolled across the United States. Participants receive the PEF ablation procedure following their institutions standard care practices. The study includes both prospective enrollment, where patients will be followed for 2 years after the PEF procedure, and retrospective enrollment, where patients who already had the procedure are followed longitudinally. This observational registry does not involve experimental treatment but monitors routine clinical use of the device. During the study, participants will be monitored according to their usual care schedules, with follow-up visits as determined by their physicians. Researchers will collect information on anesthesia type used during the PEF procedure, target location and size of the ablation, and track any serious or unexpected adverse events within 30 days after treatment. The total follow-up duration for participants is 2 years, allowing long-term assessment of safety and device performance.

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.

Researchers are evaluating how nirogacestat may affect ovarian function in adult premenopausal women who have progressing desmoid tumors or aggressive fibromatosis. Desmoid tumors are rare, slow-growing soft tissue tumors that do not spread to other parts of the body but can cause serious health problems. This open-label phase 4 study aims to understand the incidence and recovery rate of ovarian toxicity caused by nirogacestat while also assessing the drugs safety and tolerability in this population. Participants will take nirogacestat tablets orally at a dose of 150 mg twice daily. The study includes up to 24 treatment cycles, each lasting 28 days, followed by a clinical follow-up period of up to 2 years. During this time, researchers will monitor ovarian function recovery and other health effects related to ovarian toxicity and drug safety. Throughout the study, participants will undergo regular evaluations including blood tests to measure hormone levels, completion of menstrual diaries, and electronic patient-reported outcomes. Researchers will track ovarian function recovery rates, the occurrence and timing of ovarian toxicity events, and any adverse events using standardized criteria. The total participation time may extend up to 2 years after treatment for thorough monitoring and safety assessments.

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 Dupuytrens 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.