CEB-01 in Paediatrics With Locally Resectable Abdominal Tumours

NCT06986811 | Recruiting Phase 2 DMC

• 2 other identifiers: CEB-01-RLP01-CT2024-520054-38-00
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

The CEB-01 implant is a membrane containing SN-38, the active metabolite of irinotecan, an already authorized chemotherapeutic agent. After surgical removal of the abdominal cancer tumor, CEB-01 will be placed in the surgical bed for a local and sustained release of the chemotherapy. This is expected to delay or prevent local recurrence of abdominal tumors after surgery, while keeping a tolerable toxicity profile. The study aims to assess the safety, tolerability, pharmacokinetics, and efficacy of CEB-01 in pediatric patients with locally resectable abdominal tumors including Soft Tissue Sarcoma (STS), high-risk Neuroblastoma (NB), Wilms tumour (WT), germ cell tumors (GCT), extracranial malignant rhabdoid tumour (eMRT), synovial sarcoma (SS), desmoplastic small round cell tumour (DSRCT) and fibrolamellar hepatocellular carcinoma (FL-HCC

Conditions

Locally Resectable Paediatric Tumours

Keywords

CEB-01; SN-38; Locally resectable paediatrics tumours

Outcome Measures

Primary Outcomes (1)

• Frequency of Adverse Events (AEs) (Safety)

  Adverse Events (AE), serious and non-serious, with their frequency, severity, and relatedness to study drug and coded according to the most updated version of the Common Terminology Criteria for Adverse Events (CTCAE).

  Through study completion, average 3 years

Secondary Outcomes (8)

• Progression-free survival (PFS)

  Through study completion, average 3 years

• Overall survival (OS).

  Through study completion, average 3 years

• Local recurrence-free survival (LRFS)

  Through study completion, average 3 years

• New lesions either distant or metastatic by RECIST 1.1 (Response Evaluation Criteria in Solid Tumors, version 1.1)

  Through study completion, average 3 years

• Area under the concentration-time curve (AUC0-inf) of SN-38 ( 7-ethyl-10-hydroxy Camptothecin)

  During 60 days

Study Arms (2)

Standard surgery and CEB-01 implant after surgery

EXPERIMENTAL

Procedure/Surgery: Standard surgery The location and size of the tumor determine the type of surgery. Drug: CEB-01 It is novel formulation for local release of chemotherapy. It consists of a biocompatible and biodegradable nanofiber membrane made of poly(lactic-co-glycolic acid) (PLGA), which is loaded with the anti-tumor drug SN-38 and implanted in the surgical bed after tumor removal.

Procedure: Standard surgery; Drug: CEB-01

Active Comparator: Arm 2 standard surgery

ACTIVE COMPARATOR

Procedure/Surgery: Standard surgery The location and size of the tumor determine the type of surgery.

Procedure: Standard surgery

Interventions

Standard surgery PROCEDURE

The location and size of the tumor determine the type of surgery.

Active Comparator: Arm 2 standard surgery; Standard surgery and CEB-01 implant after surgery

CEB-01 DRUG

It is novel formulation for local release of chemotherapy. It consists of a biocompatible and biodegradable nanofiber membrane made of poly(lactic-co-glycolic acid) (PLGA), which is loaded with the anti-tumor drug SN-38 and implanted in the surgical bed after tumor removal.

Standard surgery and CEB-01 implant after surgery

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• To be eligible to participate in this trial, an individual must meet all the following criteria:
• ≤ 18 years.
• Participants must have a diagnosis of:
• De novo or recurrent abdominal soft-tissue sarcoma.
• De novo or recurrent high-risk neuroblastoma according to Children's Oncology Group (COG) risk classification, regardless of response to frontline therapy, diagnosed either by a former histologic verification of neuroblastoma and/or former demonstration of tumour cells in the bone marrow with increased urinary catecholamines at the time of study enrolment. Participants who were initially considered low or intermediate risk but were then reclassified as high risk are also eligible.
• Other tumours: recurrent Wilms tumour, de novo or recurrent Germ cell tumour, de novo or recurrent extracranial malignant rhabdoid tumour, de novo or recurrent synovial sarcoma, de novo or recurrent fibrolamellar hepatocellular carcinoma, and de novo or recurrent desmoplastic small round cell tumour.
• A histology assessment is required for enrolment of de novo cases. A new histology assessment is not required for enrolment of the recurrent cases, but it will be obtained from the resected tumour to assess whether the histology is identical to the original tumour.
• Participants previously treated with irinotecan will be eligible if they have not had documented progressive disease during treatment.
• Participants might have more than one surgically removable lesion.
• Adequate liver, renal, haematological, and cardiac function.
• Participants must have fully recovered from the acute toxic effects (Grade 3 or above) of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this trial.
• Landky or Karnosfsky functional performance status score ≥ 50 at screening.
• Female participants of childbearing potential must have a negative urine betahuman chorionic gonadotropin (beta-hCG) pregnancy test at time of screening.
• Female and male participants of childbearing potential must be willing to use adequate contraception throughout the study and for 6 months after surgery.
• Life expectancy greater than 6 months.

You may not qualify if:

• An individual who meets any of the following criteria will be excluded from participation in this trial:
• Metastatic lesions.
• Other malignancies within past 2 years except for in-situ cancers or basal/squamous cell skin cancer. Subjects with other malignancies are eligible if they are disease-free for at least 24 months or have a clinically stable concurrent malignancy not requiring tumour-directed treatment.
• Active bacterial, viral or fungal infection.
• Known history of active human immunodeficiency virus (HIV) infection, hepatitis B, hepatitis C or chronic liver disease. Testing is not required in the absence of clinical findings or suspicion.
• Impossibility of ensuring adequate follow-up.
• Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
• Contraindication to computed tomography (CT) scan.
• Major surgery within 14 days prior to the implant of study drug or still in recovery after experiencing surgical complications; neither tumour biopsy nor central line insertion are considered a major surgery.
• Other relevant concomitant illnesses.
• Participant' status post-allogeneic stem cell transplant are not eligible.
• Participants with disease of any major organ system that would compromise their ability to withstand therapy.
• Patients with tumour size requiring CEB-01 implant that exceeds the maximum implantable surface area based on Body Surface Area (BSA) correction.
• Patients with known hypersensitivity to SN-38 or any of the CEB-01 excipients.
• Pregnancy or lactation. Pregnant women are excluded from this study; if the patient is a lactating mother, breastfeeding should be discontinued.

Sponsors & Collaborators

• CEBIOTEX: lead

Study Sites (1)

Hospital Sant Joan de Déu

Esplugues de Llobregat, Barcelona, 08950, Spain

RECRUITING

Study Officials

• Marta Pilar Martin Gimenez, M.D.; Ph.D.

  Hospital Sant Joan de Deu

  PRINCIPAL INVESTIGATOR

Central Study Contacts

A Responsible Person Designated by the Sponsor

CONTACT

+34 93 434 44 12; investigacion@mfar.net

Anna Huguet, Ph.D.

CONTACT

ahuguet@cebiotex.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Masking Details: This is a single-blind trial in which investigators, and investigators' staff, will be aware of the treatment allocation (CEB-01 or not, but only once the tumour/ s removal has/have been completed). The participants, medical staff of the sponsor or data analysts will remain blinded to each participant's assigned study treatment from the time of randomization until database lock.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 60 participants, open label treatment arm CEB-01 plus standard of care (consisting of surgery with/without radiotherapy and/or chemotherapy), compared with a well-matched population of approximately 10 patients, from the same participating sites, including historical controls (e.g. within the last 10 years) or contemporary controls (e.g. subjects that fulfil inclusion and exclusion criteria, but do not desire to receive experimental treatment) treated with standard of care (consisting of surgery with/without radiotherapy and/or chemotherapy) will be the external control arm.

Study Record Dates

• First Submitted: May 15, 2025
• First Posted: May 23, 2025
• Study Start: May 6, 2025
• Primary Completion (Estimated): May 1, 2029
• Study Completion (Estimated): May 1, 2029
• Last Updated: May 31, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)