Arterial Chemoembolization for the Treatment of Desmoid Fibromatosis

Desmoid Clinical Trial

— Embodesmo  

Official title:

Chemioembolizzazione Arteriosa Per il Trattamento Della Fibromatosi Desmoide: Studio Osservazionale Prospettico

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06268457
• Other study ID #: CE-AVEC 789/2023/Oss/IOR
• Status: Recruiting
• Start date: February 20, 2024
• Est. completion date: February 1, 2028

Study information

• Verified date: March 2024
• Source: Istituto Ortopedico Rizzoli
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Desmoid fibromatoses are rare (1-2 cases/million per year) and locally aggressive mesenchymal tumors. For asymptomatic disease, current guidelines suggest an initial period of active surveillance.

The current scientific evidence regarding the efficacy and safety of the treatment of desmoid fibromatosis by arterial embolization is constituted by several retrospective and prospective studies. Embolization of desmoid tumors alone, without chemotherapy, on the contrary, has been shown to be inefficient. Using Doxorubicin in desmoid fibromatosis is effective but associated with systemic toxicity. Consequently, this drug is reserved for symptomatic, nonresponsive, rapidly growing or life-threatening tumors. The intrinsic hypervascularity of desmoid tissue can be exploited as a conduit to achieve local distribution of Doxorubicin by navigation of a catheter endovascular.

Description:

Desmoid fibromatoses are rare (1-2 cases/million per year) 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.

The current scientific evidence regarding the efficacy and safety of the treatment of desmoid fibromatosis by arterial embolization is constituted by several retrospective and prospective studies. These studies report promising results through the use of chemoembolization, that is, arterial embolization using particles loaded with chemotherapy. Embolization of desmoid tumors alone, without chemotherapy, on the contrary, has been shown to be inefficient. Doxorubicin is routinely used in the treatment of soft tissue sarcomas and other mesenchymal malignancies. Its use against desmoid fibromatosis is effective but associated with hematologic, gastrointestinal, and cardiac toxicity. Consequently, this drug is reserved for symptomatic, nonresponsive, rapidly growing or life-threatening tumors. The intrinsic hypervascularity of desmoid tissue can be exploited as a conduit to achieve local distribution of Doxorubicin by navigation of a catheter endovascular. Doxorubicin contains a protonated amine group which can establish an ionic bond with the sulfonate present on the surface of microbeads of hydrogel, ensuring embolization and elution of the drug. This process allows high concentrations of Doxorubicin in the target tissue and low concentrations in the systemic circulation.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: February 1, 2028
• Est. primary completion date: February 1, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• FIbromatosis demsoid symptomatic and in active phase (documented growth at last follow-ups)

• Patients who are not candidates for surgery or cryoablation

Exclusion Criteria:

• Patients with life expectancy <3 months or severely impaired status functional status (ASA 4)

• Patients with fibromatosis not in active phase, documented clinically and by investigations imaging (MRI, CT)

• Patients with coagulation deficiency or plateletopenic disease

• Patients with documented active infection

Related Conditions & MeSH terms

• Desmoid

Intervention

Procedure:
• Chemoembolization with Doxorubicin
Identification of the main vessels afferent to the metastasis to be treated. Selective and super-selective catheterization of pathological arterial branches, responsible for the blood supply to the neoplasm, through the use of micro-catheters. Injection of embolizing material (hydrogel microspheres with doxorubicin) Control of technical success: the embolization will be followed by diagnostic arteriography that will evaluate the success of the procedure (occlusion of more than 90 percent of the pathologic vasculature).

Locations

Country	Name	City	State
Italy	Istituto Ortopedico Rizzoli	Bologna	Emilia Romagna

Sponsors (1)

Lead Sponsor	Collaborator
Istituto Ortopedico Rizzoli

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Volume reduction in cm	Reduction of the lesion evaluated with MRI, measuring the diameter of the lesion in cm.	1 year
Primary	Reduction 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
Primary	Reduction of antalgic therapy (mg)	Mean reduction in the use of pain medications evaluated in mg of active ingredient taken daily before the treatment and at follow-up.	1 year
Secondary	Improvement in quality of life (EORTC QLQ-C15-PAL questionnaire)	Clinical assessment regarding quality of life by EORTC QLQ-C15-PAL questionnaire. The questionnaire consists of several scales, covering different aspects related to palliation. The maximum and minimum scores for each scale of the questionnaire can range from 0 to 100. The interpretation of scores depends on the specific scale in the questionnaire, and a higher value may indicate either greater negative impact (e.g., more symptoms or more suffering) or greater positive impact (e.g., better quality of life or less symptomatology). The overall assessment requires a detailed analysis of individual scores and related scales.	1 year
Secondary	Improvement in quality of life (EORTC QLQ - BM22)	Clinical assessment regarding quality of life by EORTC QLQ - BM22 questionnaire. The questionnaire consists of several scales, covering different aspects related to palliation. The maximum and minimum scores for each scale of the questionnaire can range from 0 to 100. The interpretation of scores depends on the specific scale in the questionnaire, and a higher value may indicate either greater negative impact (e.g., more symptoms or more suffering) or greater positive impact (e.g., better quality of life or less symptomatology). The overall assessment requires a detailed analysis of individual scores and related scales.	1 year