NCT06986811

CEB-01 in Paediatrics With Locally Resectable Abdominal Tumours

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: CEBIOTEX · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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

Detailed description

CEB-01-RLP01-CT trial is 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 de novo or recurrent, locally resectable, abdominal tumors in comparison to standard of care. The trial population will consist of 80 participants who fulfil all the inclusion and exclusion criteria, allocated in three cohorts: Cohort 1: 20 participants with abdominal STS, open label treatment arm consisting of CEB-01 plus the standard of care (which may include surgery, with or without radiotherapy and/or chemotherapy). The outcomes will be compared to a well-matched population of about 10 participants from the same participating sites, including historical controls or contemporary controls. Cohort 2: 20 participants with high-risk NB, open label treatment arm consisting of CEB-01 plus the standard of care (which may include surgery, with or without radiotherapy and/or chemotherapy). The outcomes will be compared to a well-matched population of about 10 participants from the same participating sites, including historical controls or contemporary controls. Cohort 3: 20 participants with rare abdominal tumors including WT, GCT, eMRT, SS, DSRCT and FL-HCC, open label uncontrolled treatment arm to obtain additional safety and efficacy data. For measurement of primary safety and efficacy endpoints, follow-up will consist of short-term evaluation at 365 ± 30 days and long-term evaluation at 1095 ± 30 days post-surgery as it is considered sufficient for the assessment of the therapeutic effect of CEB-01 regarding local recurrence. For pharmacokinetic assessment, blood samples will be collected at baseline and at 9 different time points until 56 ± 7 days post-surgery. For each participant the trial duration will be composed of a screening period for up to 28 days, one day for surgery and 1095 ± 30 days of follow-up. A Data Safety Monitoring Board (DSMB) will review data in an unblinded fashion; details are given in the DSMB charter. ScheduledThe scheduled review will be performed by the DSMB once 10 randomised and treated participants have completed 6 months of follow-up. The DSMB can be convened at the request of the sponsor should safety signals be detected

Conditions

• Locally Resectable Paediatric Tumours

Interventions

Type	Name	Description
PROCEDURE	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.

Timeline

• Start date: 2025-05-06
• Primary completion: 2029-05-01
• Completion: 2029-05-01
• First posted: 2025-05-23
• Last updated: 2025-05-31

Locations

1 site across 1 country: Spain