BioPearl™ Microspheres Loaded With Doxorubicin for the Treatment of Unresectable Hepatocellular Carcinoma (HCC) (BIOPEARL-ONE)

BioPearl™ Microspheres Loaded With Doxorubicin for the Treatment of Unresectable Hepatocellular Carcinoma (HCC): Prospective, Single Arm, Multi-center, Post-Market Clinical Follow-up (PMCF) Study

The primary objective of the study is to confirm technical success and safety of BioPearl™ microspheres loaded with Doxorubicin in the treatment of unresectable hepatocellular carcinoma (HCC). The secondary objective of the study is to investigate the efficacy of BioPearl™ microspheres loaded with Doxorubicin in the treatment of subjects with unresectable HCC.

Study Overview

• Status: Recruiting
• Conditions: Hepatocellular Carcinoma Non-resectable
• Intervention / Treatment: Device: BioPearl™

Detailed Description

This is a prospective, single arm, multi-centre, post-market clinical follow-up study to further assess safety and efficacy in 50 subjects with unresectable HCC treated with BioPearl™ microspheres loaded with Doxorubicin. All subjects will undergo clinical follow-up until disease progression and/or next treatment option, after which subjects will be followed for survival. Subjects will be followed up to a maximum of 18 months. An intermediate analysis will take place during enrollment period on safety and technical success to support regulatory requirements.

• Study Type: Interventional
• Enrollment (Estimated): 50
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Belgium
  • Brussels, Belgium
    • Not yet recruiting
    • CUB Hôpital Erasme
    • Contact: Gotran Verset, MD
  • Leuven, Belgium
    • Recruiting
    • UZ Leuven
    • Contact: Geert Maleux, MD; Phone Number: 0032 16 34 75 78; Email: geert.maleux@uzleuven.be
• Germany
  • Heilbronn, Germany
    • Recruiting
    • SLK-Kliniken Heilbronn
    • Contact: Philippe Pereira, MD; Phone Number: 07131 49-466-63; Email: Philippe.Pereira@slk-kliniken.de
• Italy
  • Milan, Italy
    • Recruiting
    • Fondazione IRCCS Instituto Tumori
    • Contact: Carlo Spreafico, MD; Phone Number: 39 02 23901; Email: Carlo.Spreafico@istitutotumori.mi.it
  • Rome, Italy
    • Not yet recruiting
    • Fondazione Policlinico Universitario A. Gemelli
    • Contact: Roberto Iezzi, MD
  • Turin, Italy
    • Not yet recruiting
    • Città della Salute e della Scienza di Torino Presidio Molinette
    • Contact: Paolo Fonio, MD

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. Subject is at least 18 years old
2. Subject with HCC confirmed by histology or according to the latest applicable version of (EASL (the European Association for the Study of the Liver) criteria
3. Subject with tumor(s) < 5 cm and within the up to 7 criteria: the sum of the diameter of the largest tumor (in cm) and the number of tumors must be ≤ 7 (see appendix E)
4. BCLC (Barcelona Clinic Liver Cancer) B subject or BCLC A subject not a candidate for curative treatment at the time of study inclusion or who has failed/recurred after resection/ablation. Recurrence in the segment of RFA (Radiofrequency Ablation is not allowed
5. WHO (World Health Organization) or ECOG (Eastern Cooperative Oncology Group) performance status 0 or 1
6. Subject deemed treatable in one session for initial treatment
7. Normal liver or compensated cirrhosis with preserved liver function (Child-Pugh Class A)
8. Total bilirubin ≤ 2.0 mg/dl
9. Adequate bone marrow function: Hemoglobin ≥ 9g/dl, absolute neutrophil count ≥ 1.0 x 109/L, platelet count ≥ 75 x 109/L
10. Subject with no ascites or with minor ascites controlled by sodium dietary restrictions (subject receiving diuretic treatment or paracentesis is not eligible)
11. Adequate renal function: serum creatinine < 1.5 X ULN (Upper Limit of Normal)
12. Subject has provided written informed consent
13. Subjects of childbearing/reproductive potential should use adequate birth control measures, during the study treatment period until survival follow-up

Exclusion Criteria:

1. Subject previously treated with any systemic therapy for HCC
2. Subject previously treated with intra-arterial loco-regional therapy for HCC
3. Prior resection/ablation is allowed as per inclusion criteria 4
4. Eligible for curative treatment at the time of study inclusion
5. Advanced liver disease: Child-Pugh's B-C class or active gastrointestinal bleeding, encephalopathy
6. Advanced tumoral disease: BCLC class C or D (vascular invasion - even segmental, extra-hepatic spread or cancer-related symptoms performance status >1)
7. History of another primary tumor. Exceptions include: 1) Malignancy treated with curative intent ≥ 5 years before inclusion and with no known active disease / 2) Malignancy which occurred < 5 years before, not active and not expected to recur or be clinically relevant in the next 5 years
8. Subject with history of biliary tree disease or biliary dilatation
9. Portal vein thrombosis, porto-systemic shunt, hepatofugal blood flow or absent portal blood flow in the liver area to be treated
10. Contraindication to multiphasic CT and MRI (e.g. allergy to contrast media)
11. Any other contraindication for embolization procedure or Doxorubicin treatment
12. Subject is currently participating in an investigational drug or device study that has not completed the primary endpoint or that clinically interferes with the current study endpoints Note: Trials requiring extended follow-up for products that were investigational, but have become commercially available since then, are not considered investigational trials
13. In the Investigator's opinion subject has (a) co-morbid condition(s) that could limit the subject's ability to participate in the study, compliance with follow-up requirements or impact the scientific integrity of the study
14. Pregnant or breast-feeding woman

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Other: Single arm; Transarterial Chemo Embolization (TACE) with BioPearl™ microspheres loaded with Doxorubicin	Device: BioPearl™; TACE

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety: by evaluating all procedural or study device related grade 3-4-5 adverse events	Safety: by evaluating all procedural or study device related grade 3-4-5 adverse events (AEs) during a period of 4 weeks post initial treatment as per local investigator assessment	Day 29
Technical success	Ability to reach near stasis in the treated tumor feeding arteries during chemoembolization procedure	Day 1

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Tumor response rate	Assessed by mRECIST (modified Response Evaluation Criteria in Solid Tumors) criteria at 4 weeks and every 3 months as per local investigator assessment	18 months
Progression Free Survival (PFS	Defined as time from the treatment to disease progression according to mRECIST criteria or death from any cause, whichever occurs first as per local investigator assessment	18 months
Time to progression of treated tumor(s)	Defined as time from treatment to progression of the treated lesion according to mRECIST criteria as per local investigator assessment	18 months
Duration of response	According to mRECIST as per local investigator assessment	18 months
Best overall response	Defined as best response of treated tumor(s) recorded during the study according to mRECIST criteria as per local investigator assessment	18 months

Collaborators and Investigators

• Sponsor: Terumo Europe N.V.

Study record dates

Study Major Dates

• Study Start (Estimated): February 29, 2024
• Primary Completion (Estimated): June 30, 2026
• Study Completion (Estimated): November 30, 2027

Study Registration Dates

• First Submitted: May 26, 2023
• First Submitted That Met QC Criteria: June 20, 2023
• First Posted (Actual): June 22, 2023

Study Record Updates

• Last Update Posted (Actual): February 23, 2024
• Last Update Submitted That Met QC Criteria: February 22, 2024
• Last Verified: February 1, 2024

More Information

Terms related to this study

• Keywords: HCC; TACE; Chemoembolization; Hepatocellular Carcinoma Non-resectable
• Additional Relevant MeSH Terms: Digestive System Diseases; Neoplasms by Histologic Type; Neoplasms; Neoplasms by Site; Adenocarcinoma; Neoplasms, Glandular and Epithelial; Digestive System Neoplasms; Liver Diseases; Liver Neoplasms; Carcinoma; Carcinoma, Hepatocellular
• Other Study ID Numbers: T144E3

Plan for Individual participant data (IPD)

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

Drug and device information, study documents

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