NCT03966742

Doxorubicin Eluting Intra-arterial Embolization for Aggressive Desmoid Fibromatosis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Rabin Medical Center · Academic / Other
• Sex: All
• Age: 3 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

In this study Drug-eluting microbeads (DEB) loaded with Doxorubicin will be delivered into the target Desmoid Fibromatoses (DF) tissue via selective arterial embolization by angiographic technique. The objective of the study is to demonstrate the safety and efficacy of this treatment.

Detailed description

Desmoid Fibromatoses (DF) are locally aggressive lesions associated with substantial morbidity and potentially mortality, due to invasion of adjacent neurovascular structures and vital organs. They have no potential for metastasis. Histologically, they are characterised by mature fibroblasts within a matrix of abundant fibrous stroma. While 5-15% of cases are seen in patients with Familial Adenomatous Polyposis (FAP) syndrome, the vast majority arise sporadically. The etiology of Desmoids remains poorly understood and the therapeutic approaches in their management remain very diverse. For resectable lesions, surgery is recommended but reported cure rates range broadly from 12-80%. Systemic treatments range from non-steroidal anti-inflammatories and anti-estrogenic therapy to targeted tyrosine kinase inhibitors and cytotoxic chemotherapy, most commonly methotrexate, vinblastine and doxorubicin. Doxorubicin is an anthracycline with demonstrated efficacy in treating desmoids at systemic IV doses of 50- 75mg/m2 over 3-4 week cycles. Extended use is limited by dose - dependent cardiotoxicity which can be seen in up to 36% of patients receiving doses in excess of 550mg/m2. Delayed cardiotoxicity is particularly common and less predictable among pediatric cancer survivors. Selective trans-arterial chemo-embolization (TACE) is a method to achieve high tissue drug concentration with minimal systemic toxicity. Historically, this has been achieved by mixing doxorubicin with embolic agents such as lipiodol or gelatin sponge in the treatment of hepatocellular carcinoma (HCC). Drug-eluting microbeads (DEB) ionically loaded with doxorubicin have shown sustained release in TACE target tissues with substantially lower serum drug concentrations when compared to lipiodol TACE. The present study utilizes DEB's loaded with Doxorubicin delivered into the target DF tissue via selective arterial embolization by angiographic technique. This study follows a successful feasibility study.

Conditions

• Desmoid
• Desmoid Fibromatosis of Skin
• Desmoid Neoplasm of Chest Wall
• Desmoid Tumor Caused by Somatic Mutation
• Aggressive Fibromatoses
• Fibromatosis Desmoid
• Desmoid Fibromatosis

Interventions

Type	Name	Description
PROCEDURE	Drug Eluting Bead Embolization (DEB) for Desmoid Fibromatosis	Delivery of Doxorubicin selectively into Desmoid Fibromatosis utilizing its vascular supply as a conduit, and ionic loading the doxorubicin onto embolized particles as the drug delivery vehicle. Maximal dose is 75 mg/m2 and at least 50 mg. The bead size is 75-300 Mµ in a 2 CC syringe.

Timeline

• Start date: 2018-10-11
• Primary completion: 2020-12-18
• Completion: 2020-12-18
• First posted: 2019-05-29
• Last updated: 2022-03-25

Locations

1 site across 1 country: Israel