Doxorubicin Eluting Intra-arterial Embolization for Aggressive Desmoid Fibromatosis

NCT03966742 | Completed Phase 2

• 1 other identifier: RMC-0485-17
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

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

Outcome Measures

Primary Outcomes (3)

• Objective response rate of tumor size.

  Response to treatment. Measured by change in tumor size according to RECIST (Response Evaluation Criteria in Solid Tumors) version 1.1 criteria.

  At baseline ; 6-10 weeks after each treatment

• Objective response rate of tumor biological activity.

  Response to treatment. Tumor biological activity measured by change in MRI T2 signal intensity.

  At baseline ; 6-10 weeks after each treatment

• Patient reported outcomes.

  Change in clinical symptoms measured by standard clinical patient questionnaires - EORTC QLQ-C30. (Quality of Life Questionnaire). All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. Thus a high score for a functional scale represents a high level of functioning, a high score for the global health status / QoL represents a high QoL. Similarly, a high score for a symptom scale represents a high level of symptomatology.

  At baseline ; 6-10 weeks after each treatment

Secondary Outcomes (1)

• Adverse event profile (safety)

  At baseline ; 6-10 weeks

Other Outcomes (2)

• Pharmacokinetics of Doxorubicin

  5 minutes, 30 minutes, 1 hour, 12 hours, 24 hours after treatment procedure

• Exploratory biomarkers

  At baseline ; 6-10 weeks

Study Arms (1)

Treatment

EXPERIMENTAL

Doxorubin-eluting particle embolization for treatment of Desmoid Fibromatosis.

Procedure: Drug Eluting Bead Embolization (DEB) for Desmoid Fibromatosis

Interventions

Drug Eluting Bead Embolization (DEB) for Desmoid Fibromatosis PROCEDURE

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.

Treatment

Eligibility Criteria

• Age: 3 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Age 3-80 years.
• Histologically confirmed diagnosis of Desmoids Fibromatosis.
• After at least one systemic treatment line. Standard first line systemic treatment may include: Methotrexate, Vinblastine, Doxorubicin, Liposomal Doxorubicin (Doxil), NSAIDS or hormonal treatment. If first line treatment is renounced, this treatment decision must be documented. Considering the trend of avoiding surgical treatment, the documentation must include that the treatment decision is not associated to the resectability of the tumor.
• Karnofsky performance status (PS)\>50% for patients older than 16 years or Lansky PS \>50% for patients under 16 years.
• At least one measurable lesion, with a long diameter of at least 30mm, with an anatomical location accessible for endovascular treatment.
• T2 signal increase on MRI.
• No evidence of prior treatment toxicity, adequate washout period after prior treatment:
• days after myelosuppressive chemotherapy treatment.
• days after GCSF (Granulocyte colony-stimulating factor), 14 days after Neulastim.
• days after targeted/biologic treatment.
• Female patients of childbearing potential must be willing to use an adequate method of contraception (hormonal, barrier or abstinence) for the treatment period and up to 90 days after the treatment completion.
• Willing and able to provide written informed consent for the trial.

You may not qualify if:

• Participation in another interventional study.
• Congestive heart failure, characterised by LVEF (Left Ventricular Ejection Fraction) \< 50% or Shortening fracture \< 27%.
• Previous treatment with anthracycline of a accumulative dose of more than 360 mg/m2.
• History of allergic reaction attributed to Doxil or doxorubicin treatment.
• Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure (New York Heart Association Class III or IV), cardiac arrhythmia, or psychiatric illness, social situations that would limit compliance with study requirements.

Sponsors & Collaborators

• Rabin Medical Center: lead

Study Sites (1)

Rabin Medical Center

Petah Tikva, Israel

Location

Related Publications (1)

• Elnekave E, Atar E, Amar S, Bruckheimer E, Knizhnik M, Yaniv I, Dujovny T, Feinmesser M, Ash S. Doxorubicin-Eluting Intra-Arterial Therapy for Pediatric Extra-Abdominal Desmoid Fibromatoses: A Promising Approach for a Perplexing Disease. J Vasc Interv Radiol. 2018 Oct;29(10):1376-1382. doi: 10.1016/j.jvir.2018.04.009. Epub 2018 Jul 31.

  PMID: 30075974 BACKGROUND

MeSH Terms

Conditions

Desmoid Tumors; Desmoid disease, hereditary

Condition Hierarchy (Ancestors)

Fibroma; Neoplasms, Fibrous Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Clinic Interventional Oncology

Model Details: All patients

Study Record Dates

• First Submitted: April 8, 2019
• First Posted: May 29, 2019
• Study Start: October 11, 2018
• Primary Completion: December 18, 2020
• Study Completion: December 18, 2020
• Last Updated: March 25, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1)