CEB-01 treatment for children with resectable abdominal tumors

First-in-paediatrics, Open Label, Exploratory, Externally Controlled Clinical Trial to Evaluate Safety, Efficacy and Pharmacokinetics of 7-ethyl-10-hydroxy Camptothecin (SN-38) Formulated as a Biocompatible Polymeric Nanofiber Membrane (CEB-01) for Treatment, in Addition to Standard of Care, of Paediatric Patients From Birth to Less Than 18 Years of Age With a Locally Resectable Tumours in Comparison to Standard of Care

This study is testing a new treatment called CEB-01 for children with certain abdominal tumors to see if it helps them do better than just the usual care they receive.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Ages: N/A to 18 Years
• Sex: All
• Sponsor: Cebiotex (Industry-sponsored)
• Drugs / interventions: chemotherapy, immunotherapy
• Locations: 1 site (Esplugues de Llobregat, Barcelona)
• Trial ID: NCT06986811 on ClinicalTrials.gov

What this trial studies

This clinical trial evaluates the safety, efficacy, and pharmacokinetics of CEB-01, a novel biocompatible polymeric nanofiber membrane loaded with SN-38, in pediatric patients with locally resectable abdominal tumors. The trial is open-label and exploratory, comparing the outcomes of patients receiving CEB-01 alongside standard care to those receiving standard care alone. It aims to assess various endpoints including adverse events, progression-free survival, overall survival, and quality of life. The study involves three cohorts of participants and utilizes an external control group for comparison.

Who should consider this trial

Good fit: Ideal candidates include children aged 0 to 18 years with specific diagnoses such as neuroblastoma, soft tissue sarcoma, Wilms tumor, and other related tumors.

Not a fit: Patients with tumors that are not locally resectable or those who do not meet the specific inclusion criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could improve outcomes for pediatric patients with locally resectable abdominal tumors.

How similar studies have performed: While this approach is novel in pediatrics, similar studies in adult populations have shown promising results with localized chemotherapy delivery methods.

Eligibility criteria

Inclusion Criteria:

To be eligible to participate in this trial, an individual must meet all the following criteria:

1. ≤ 18 years.
2. Participants must have a diagnosis of:

   1. De novo or recurrent abdominal soft-tissue sarcoma.
   2. 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.
   3. 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.
3. 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.
4. Participants previously treated with irinotecan will be eligible if they have not had documented progressive disease during treatment.
5. Participants might have more than one surgically removable lesion.
6. Adequate liver, renal, haematological, and cardiac function.
7. 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.
8. Landky or Karnosfsky functional performance status score ≥ 50 at screening.
9. Female participants of childbearing potential must have a negative urine betahuman chorionic gonadotropin (beta-hCG) pregnancy test at time of screening.
10. Female and male participants of childbearing potential must be willing to use adequate contraception throughout the study and for 6 months after surgery.
11. Life expectancy greater than 6 months.
12. The participant legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained prior to any protocol screening procedures.

Exclusion Criteria

An individual who meets any of the following criteria will be excluded from participation in this trial:

1. Metastatic lesions.
2. 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.
3. Active bacterial, viral or fungal infection.
4. 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.
5. Impossibility of ensuring adequate follow-up.
6. Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
7. Contraindication to computed tomography (CT) scan.
8. 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.
9. Other relevant concomitant illnesses.
10. Participant' status post-allogeneic stem cell transplant are not eligible.
11. Participants with disease of any major organ system that would compromise their ability to withstand therapy.
12. Patients with tumour size requiring CEB-01 implant that exceeds the maximum implantable surface area based on Body Surface Area (BSA) correction.
13. Patients with known hypersensitivity to SN-38 or any of the CEB-01 excipients.
14. Pregnancy or lactation. Pregnant women are excluded from this study; if the patient is a lactating mother, breastfeeding should be discontinued.

Where this trial is running

Esplugues de Llobregat, Barcelona

• Hospital Sant Joan de Déu — Esplugues de Llobregat, Barcelona, Spain (Recruiting)

Study contacts

• Principal investigator: Marta Pilar Martin Gimenez, M.D.; Ph.D. — Hospital Sant Joan de Deu
• Study coordinator: A Responsible Person Designated by the Sponsor

Conditions Locally Resectable Paediatric Tumours; CEB-01; SN-38; Locally resectable paediatrics tumours