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

Official title

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

Overview

The primary objective of the study is to confirm safety and technical success 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.

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.

Interventions

• Device BioPearl™
  TACE

Primary outcome measures

• Safety: by evaluating all procedural or study device related grade 3-4-5 adverse events (AEs) [Time frame: Day 29]
• Technical success [Time frame: Day 1]

Secondary outcome measures (6)

• Tumor response rate [Time frame: 18 months]
• Progression Free Survival (PFS) [Time frame: 18 months]
• Time to progression [Time frame: 18 months]
• Duration of response [Time frame: 18 months]
• Best response [Time frame: 18 months]
• Survival Rate [Time frame: 18 months]

Eligibility criteria

Inclusion criteria

• Subject is at least 18 years old
• 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
• 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 ≤ 7.0
• Subject with BCLC (Barcelona Clinic Liver Cancer) Stage A or B classification who is not a candidate for curative treatment at the time of study inclusion
• Subject with treatment failure/recurrence after a prior resection/ablation is permitted, with the exception of recurrence in the segment of a prior thermal ablation
• WHO (World Health Organization) or ECOG (Eastern Cooperative Oncology Group) performance status 0 or 1
• Subject deemed treatable in one session for initial treatment
• Normal liver or compensated cirrhosis with preserved liver function (Child-Pugh Class A)
• Total bilirubin ≤ 2.0 mg/dl
• Adequate bone marrow function: Hemoglobin ≥ 9g/dl, absolute neutrophil count ≥ 1.0 x 109/L, platelet count ≥ 75 x 109/L
• Subject with no ascites or with minor ascites controlled by sodium dietary restrictions (subject receiving diuretic treatment or paracentesis is not eligible)
• Adequate renal function: serum creatinine < 1.5 X ULN (Upper Limit of Normal)
• Subject has provided written informed consent
• Subjects of childbearing/reproductive potential should use adequate birth control measures, during the study treatment period until survival follow-up

Exclusion criteria

• Subject previously treated with any systemic therapy for HCC
• Subject previously treated with intra-arterial loco-regional therapy for HCC
• Eligible for curative treatment at the time of study inclusion
• Recurrence in the segment of a prior thermal ablation
• Advanced liver disease: Child-Pugh's B-C class or active gastrointestinal bleeding, encephalopathy
• Advanced tumoral disease: BCLC class C or D (vascular invasion - even segmental, extra-hepatic spread or cancer-related symptoms performance status >1)
• History of another primary tumor. Exceptions include:

A. Malignancy treated with curative intent ≥ 5 years before inclusion and with no known active disease

B. Malignancy which occurred < 5 years before, not active and not expected to recur or be clinically relevant in the next 5 years

• Subject with history of biliary tree disease or biliary dilatation
• Portal vein thrombosis, porto-systemic shunt, hepatofugal blood flow or absent portal blood flow in the liver area to be treated
• Contraindication to multiphasic CT and MRI (e.g. allergy to contrast media)
• Any other contraindication for embolization procedure or Doxorubicin treatment
• 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

• 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
• Pregnant or breast-feeding woman

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

France · 3 centers

• Beaujon Hospital — Clichy
• CHU Grenobles (Hôpital Michallon) — La Tronche
• Paul-Brousse Hospital — Villejuif

Italy · 3 centers

• Fondazione IRCCS Instituto Tumori — Milan
• Fondazione Policlinico Universitario A. Gemelli — Rome
• Città della Salute e della Scienza di Torino — Turin

Belgium · 2 centers

• CUB Hôpital Erasme — Brussels
• UZ Leuven — Leuven

Germany · 2 centers

• SLK-Kliniken Heilbronn — Heilbronn
• University Hospital Tübingen — Tübingen

Publications

• Verset G, Iezzi R, Bargellini I, Bucalau AM, Pereira P, Groezinger G, Spreafico C, Maleux G. BioPearl doxorubicin microspheres for unresectable HCC: a prospective, single-arm, multicenter study: BIOPEARL-ONE. Future Oncol. 2025 Feb;21(5):557-564. doi: 10.1080/14796694.2024.2446137. Epub 2024 Dec 30. PMID 39972606

Identifiers

NCT: NCT05911633 · T144E3

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗