View clinical trials related to Desmoid Tumors.
Filter by:Desmoid tumors, also called aggressive fibromatosis, are rare, locally invasive tumors with no potential for metastasis. The incidence is approximately 2 to 4 per million per year in the general population. The "watch and wait" policy is the standard of care for newly diagnosed patients in Europe. Patients who progress have first-line medical treatment, surgery is no longer the treatment because there is a high rate of recurrence (> 60%) and radiotherapy is not very suitable because the patients are often young and the tumors are large. Cryoablation seems to be an effective therapeutic option that should be evaluated.
Desmoid tumors (DT) are rare tumors (2-4 cases/million/year) that originate from musculoaponeurotic structures. Although they are benign tumors with no metastatic potential, DT are considered as locally aggressive tumors, with local invasiveness and tissue destruction, leading to pain, and disability. Surgery remains the keystone of therapy, but is limited by the anatomical situation of extra-abdominal desmoid (EAD) tumors (chest wall, root members). In patients where surgery is considered, negative-margin resection (R0) is recommended, but this frequently results in cosmetic/functional impairment. Moreover, prognostic impact of R0 resections remains controversial. The outcome after initial surgery depends upon several factors such as age, tumor site, and tumor size as demonstrated by recent data from the French Sarcoma Group. Alternative therapies to DT surgery for front-line or recurrence include NSAID's, anti-estrogens alone or in combination, -interferon, chemotherapy, targeted therapies or radiation therapy. All of these medical approaches however may fail to achieve long-term disease control and a number of patients suffer from irreducible pain, and disability from tumor volume. Cryoablation is a promising technique that is suitable for patients experiencing extra-abdominal DT. The procedure is based on repeated cycles of freezing/passive thawing of the tumor, leading to cell death. The technique has many advantages, among which: the accurate control of iceball under real-time MRI or CT-scan monitoring (that is not possible with other techniques such as radiofrequency), the lack of mutilation, the possibility of repeating the procedure. The cryoablation procedure has proven to be beneficial for the treatment of various tumors (liver metastases, breast, kidney). Recently, percutaneous cryotherapy has been reported in the treatment of EAD tumors poorly suited to surgery, with promising results. In the light of these encouraging data, it is believed that patients with extra-abdominal DT not amenable to surgery unless unacceptable surgical sequel and progressing after at least two lines of adequate medical therapy (tamoxifen, NSAID or chemotherapy), could benefit from the cryoablation procedure. Tumor cryotherapy-induced regression should allow symptoms relief, prolonged progression-free survival and a better quality of life.
Background: - Desmoid tumors (also known as aggressive fibromatosis), are rare, locally invasive, slow-growing soft-tissue tumors. The disease can be either asymptomatic or be associated with severe loss of organ function and significant morbidity. - Treatment with the selective small-molecule Gamma-secretase inhibitor PF-03084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early phase clinical trial. - The Notch pathway is a key regulator of cell differentiation, proliferation and apoptosis; aberrant signaling via the Notch pathway is associated with carcinogenesis. Objectives: - Primary: Determine the response rate (Complete Response (CR)+Partial Response (PR)) of PF-03084014 in patients with desmoid tumors/aggressive fibromatosis - Secondary: Assess symptom measures at baseline and on study; perform genotyping for germline and somatic mutations in adenomatous polyposis coli gene (APC) and catenin-beta 1 (CTNNB1) genes; correlate clinical response to therapy with genotyping data; and assess modulation of the Notch pathway by evaluating notch response genes in tumor biopsies at baseline and after drug administration Eligibility: - Age greater than or equal to18; histologically confirmed desmoid tumor not amenable to curative resection or definitive radiation therapy that has progressed after receiving at least one line of standard treatment; adequate organ function - Willingness to provide blood samples and 10 unstained slides or a tumor block for genetic research studies Study Design: - This is an open-label Phase II trial of PF-03084014; study drug will be administered orally at 150 mg twice a day in 21-day cycles - Optional tumor biopsies for research purposes will be performed at baseline prior to study treatment and at the beginning of cycle 7 (+/- one cycle) - Restaging scans (magnetic resonance imaging (MRI) with diffusion weighting) will be performed at baseline, at the end of cycles 1 and 6, and then every 6 cycles - Health-related quality of life (HRQOL)/symptom questionnaires will be administered at baseline and at each Clinical Center visit
The purpose of this study is to examine the response rate of desmoid tumors to hydroxyurea. The investigators hypothesize that hydroxyurea will be a safe, non-toxic alternative to aggressive surgery or chemotherapy for this difficult to treat tumor.