Electrochemotherapy for Desmoid Tumors

Recruiting

• Conditions: Desmoid Fibromatosis

• Registration Number: NCT06660121
• Lead Sponsor: Istituto Ortopedico Rizzoli

Brief Summary

The goal of this observational study is to learn about the effects of Electrochemotherapy (ECT) in patients with Desmoid Fibromatosis. The main question it aims to answer is:

Is ECT effective in reducing the size of the lesion and improving patients' symptomatology?

Patients will be followed with MRI and Quality of Life questionnaires at 3, 6 and 12 months after the intervention.

Detailed Description

Desmoid fibromatoses are rare and locally aggressive, characterized histologically by monoclonal myoblasts present in abundant stromal tissue.The current therapeutic strategy has abandoned primary resection, as recurrences after resection are common and often their phenotype is more infiltrative. Nonsurgical approaches remain suboptimal. For asymptomatic disease, current guidelines suggest an initial period of active surveillance.

Other minimally invasive local treatments such as cryoablation have been shown to be effective; however, certain patients are not candidates for such treatment because of lesion location/characteristics.

An additional minimally invasive treatment with currently little evidence in the literature is arterial embolization. However, this procedure cannot be performed in the case of superficial lesions, nor in the presence of shared vasculature between the lesion and other healthy tissues or organs. In the absence of other treatments in use, electrochemotherapy treatment can be performed, which has proven effective on several other types of soft tissue and bone tumors, malignant or benign, treated at our Institute.

Electrochemotherapy is based on the principle whereby reversible electroporation causes the application of short, high-voltage electrical pulses to temporarily align water molecules on the hydrolipid layer of the cell membrane, leading to a reversible local increase in permeability by increasing the intracellular concentration of Bleomycin and its toxicity on tumor cells by a factor of up to several thousand.

Study Timeline

• Study Start: 2024-10-17
• Study First Submitted: 2024-10-24
• Study First Submitted QC: 2024-10-24
• Study First Posted: 2024-10-26
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-05
• Last Update Posted: 2024-11-07
• Primary Completion: 2028-08-01
• Study Completion: 2029-08-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

• Symptomatic Desmoid Fibromatosis and in active phase (documented growth at last follow-up)
• Patients not eligible for surgery, cryoablation, or embolization
• Signature of study-specific informed consent.

Exclusion Criteria

• Patients with fibromatosis not in active phase, documented clinically and with imaging investigations (MRI, CT)
• Patients with documented active infection
• Previous treatment for < 30 days
• Pregnancy and lactation status
• Patients of childbearing age without contraceptive use for at least 3 months
• Presence of metal synthetic media at the site of treatment
• COPD with FiO2 < 30 mmHg
• Impaired renal function with eGFR

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Volume reduction in cm	1 year	Change of the lesion evaluated with MRI, measuring the diameter of the lesion in cm.

Secondary Outcome Measures

Name	Time	Method
Change of pain (VAS score)	1 year	Clinical assessment regarding pain by Visual Analogue Scale (VAS) score (0-100 mm), in which 0 represents no pain, and 100 represents maximum pain imaginable.
Change in quality of life ( EuroQol-5D questionnaire)	1 year	Clinical assessment regarding quality of life by EuroQol-5D questionnaire. EQ-5D score can range from -0.594 to 1.0.; The minimum value represents the worst possible health condition. The maximum value of 1.0 represents the best possible health condition. A value of 1.0 indicates that the patient has no problem in any of the five dimensions assessed by the questionnaire.
Feasibility of in vitro viability analysis of biopsy material	1 year	Evaluation of the feasibility of in vitro viability analysis of biopsy material for culturing explants and stabilizing primary cultures. Success is defined as the establishment of primary culture in 70% of samples.; Because desmoid tumors are not aggressive, it may not be easy to maintain them in culture in vitro.
Evaluation of the role of inflammatory cytokines	3 months	Evaluation of the prognostic or predictive role of inflammatory cytokines detected in the blood of patients collected on the day of treatment and after 3 months

Trial Locations

Locations (1)

IRCCS Istituto Ortopedico Rizzoli; 🇮🇹 Bologna, Italy