Electrochemotherapy for Desmoid Tumors (ECT-DESMO)

Treatment of Desmoid Fibromatosis With Electrochemotherapy. Prospective Observational Study

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.

Study Overview

• Status: Withdrawn
• Conditions: Desmoid Fibromatosis
• Intervention / Treatment: Procedure: Electrochemotherapy with bleomycin

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 Type: Observational

Contacts and Locations

Study Locations

• Italy
  • Bologna, Italy, 40136
    • IRCCS Istituto Ortopedico Rizzoli

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with diagnosis of desmoid fibromatosis with an indication for electrochemotherapy treatment

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Performed electrochemotherapy; Patients diagnosed with desmoid fibromatosis unable to perform or failed other treatments who are treated with electrochemotherapy

Procedure: Electrochemotherapy with bleomycin; Electrochemotherapy (ECT) with bleomycin is a localized treatment approach used for managing desmoid fibromatosis, a benign but locally aggressive tumor. This technique combines electrical pulses with the administration of bleomycin, a chemotherapeutic agent, to enhance drug uptake into tumor cells. The electrical pulses create temporary pores in the cell membranes, allowing bleomycin to enter more effectively, leading to increased cytotoxicity. ECT is typically performed on patients who have not responded to conventional therapies or for whom surgery is not feasible. ECT can lead to significant tumor shrinkage and improvement in symptoms, making it a promising option in the multidisciplinary management of desmoid fibromatosis.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Volume reduction in cm	Change of the lesion evaluated with MRI, measuring the diameter of the lesion in cm.	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of the role of inflammatory cytokines	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	3 months
Change of pain (VAS score)	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.	1 year
Change in quality of life ( EuroQol-5D questionnaire)	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.	1 year
Feasibility of in vitro viability analysis of biopsy material	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.	1 year

Collaborators and Investigators

• Sponsor: Istituto Ortopedico Rizzoli
• Investigators: Principal Investigator: Giancarlo Facchini, Medicine and Surgery, IRCCS Istituto Ortopedico Rizzoli

Study record dates

Study Major Dates

• Study Start (Actual): October 17, 2024
• Primary Completion (Actual): August 31, 2025
• Study Completion (Actual): August 31, 2025

Study Registration Dates

• First Submitted: October 24, 2024
• First Submitted That Met QC Criteria: October 24, 2024
• First Posted (Actual): October 26, 2024

Study Record Updates

• Last Update Posted (Actual): May 14, 2026
• Last Update Submitted That Met QC Criteria: May 11, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: Electrochemotherapy; Interventional radiology; Desmoid tumor
• Additional Relevant MeSH Terms: Neoplasms; Neoplasms by Histologic Type; Neoplasms, Connective and Soft Tissue; Neoplasms, Connective Tissue; Neoplasms, Fibrous Tissue; Fibroma; Desmoid Tumors; Peptides; Amino Acids, Peptides, and Proteins; Investigative Techniques; Therapeutics; Clinical Laboratory Techniques; Drug Therapy; Cytological Techniques; Carbohydrates; Glycoconjugates; Electrochemical Techniques; Glycopeptides; Electroporation Therapies; Electroporation; Bleomycin; Electrochemotherapy
• Other Study ID Numbers: CE AVEC: 418/2024/Oss/IOR

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Privacy concerns

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No