CEB-01 in paediatrics with locally resectable abdominal tumours.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Cebiotex S.L.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Trial duration

6 May 2025 → ongoing

Decision date (initial)

2025-04-23

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Therapy, Efficacy

To assess the safety and tolerability of CEB-01.

Secondary objectives 2

1. To assess the efficacy of CEB-01 in locally resectable recurrent abdominal neuroblastoma, soft-tissue sarcoma, Wilms, germ cell tumours and other tumours in paediatrics.
2. To characterize the pharmacokinetics of SN-38.

Conditions and MedDRA coding

Desmoplastic small round cell tumour

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-003588-PIP01-24

Plan to share IPD

No

IPD plan description

No individual participant data (IPD) will be provided.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 12

1. Age ≤18 years.
2. Participants must have a diagnosis of: a.De novo or recurrent abdominal soft-tissue sarcoma. b.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. c.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 cases, except for patients with high-risk neuroblastoma for which a radioiodine-labelled meta-iodo-benzyl-guanidine (MIBG) test is positive and excess of production od catecholamines is confirmed. 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 as defined by biochemical and haematological parameters as follows: haemoglobin (Hb) >9 g/dL (with preoperative transfusion), platelets >80.000/mm3 with intraoperative transfusion, absolute neutrophil count ≥ 1.500/mm3 or below this threshold if there is clinical indication that the patient is on hematological recovery, albumin >3.0 g/dl, alanine aminotransferase (ALT) and aspartate aminotransferase (AST) <2,5 times the upper limit of normality [ULN], bilirubin <2 times the ULN, creatinine <1,0 mg/dl or creatinine clearance > 60 ml/min.
7. Participants must have fully recovered from the acute toxic clinically relevant effects (Grade 3 or above) of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this trial.
8. Lansky or Karnofsky functional performance status score ≥ 50 at screening.
9. Female participants of childbearing potential must have a negative urine beta-human 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’s 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 14

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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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).

Secondary endpoints 9

1. Progression-free survival (PFS).
2. Overall survival (OS).
3. Local recurrence-free survival (LRFS).
4. New lesions either distant or metastatic by RECIST 1.1.
5. Paediatric Quality of Life Questionnaire (KIDSCREEN-27)
6. Area under the concentration-time curve (AUC0-inf) of SN-38.
7. Maximum concentration (Cmax) of SN-38.
8. Time of maximum plasma concentration (Tmax) of SN-38.
9. Terminal half-life (t1/2) of SN-38.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Cebiotex S.L.

Sponsor organisation

Cebiotex S.L.

Address

Tech Barcelona Pier 07 Entresuelo, Via Laietana 26 Via Laietana 26

City

Barcelona

Postcode

08003

Country

Spain

Scientific contact point

Organisation

Cebiotex S.L.

Contact name

Antonio Perez

Public contact point

Organisation

Cebiotex S.L.

Contact name

Anna Huguet

Third parties 4

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 1 · Art. 38 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 19 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

3 submissions — initial application plus substantial / non-substantial modifications