Individualized Intrathecal Chemotherapy Based on Measurement of Brain Fluid Clearance in Pediatric Patients

2025-522453-20-00 Therapeutic exploratory (Phase II) Authorised, recruitment pending

Status Authorised, recruitment pending · 1 EU/EEA countries · 1 sites

Overview

Sponsor-declared trial summary

Phase Therapeutic exploratory (Phase II)
Status Authorised, recruitment pending
Participants planned 50
Countries 1
Sites 1

Acute lymphoblastic leukemia and lymphoma

Investigate/estimate brain fluid clearance (BFC) at the individual level in children aged 12-17 years undergoing clinically indicated intrathecal chemotherapy (methotrexate, and where applicable cytarabine) for blood or lymph cancer.

Key facts

Sponsor
Oslo University Hospital HF
Participant type
Pediatric, Patients
Age range
0-17 years
Gender
Male and Female
Therapeutic area
Diseases [C] - Nervous System Diseases [C10], Diseases [C] - Neoplasms [C04]
Decision date (initial)
2026-07-03
Transition trial
No
Low-intervention
No
Rare-disease indication
No
Vulnerable population
No

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Diagnosis, Pharmacokinetic

Investigate/estimate brain fluid clearance (BFC) at the individual level in children aged 12-17 years undergoing clinically indicated intrathecal chemotherapy (methotrexate, and where applicable cytarabine) for blood or lymph cancer.

Secondary objectives 3

  1. Evaluate safety/tolerability and explore the association between brain fluid clearance and Day 0-7 solicited AEs/AESIs.
  2. Explore association between brain fluid clearance and fatigue at 3 months.
  3. Explore association between brain fluid clearance and cognitive function at 3 months and 2 years after intrathecal chemotherapy.

Conditions and MedDRA coding

Acute lymphoblastic leukemia and lymphoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

  1. Age. Participant must be in the age range 12 to 17 years at the time of signing the informed consent.
  2. Type of Participant and Disease Characteristics. Individuals undergoing intrathecal chemotherapy with MTX for blood or lymph cancer and being treated at Oslo University Hospital.
  3. Weight. Body weight is above 50 kg.
  4. Sex and Contraceptive/Barrier Requirements. Both male and female. WOCBP may be included only if they meet protocol pregnancy-testing requirements, have a confirmed menstrual period, have a negative highly sensitive urine or serum pregnancy test before inclusion/intervention, and use highly effective contraception where applicable from inclusion through Day 7 safety follow-up. WOCBP means a post-menarche female participant who is not permanently sterile and not post-menopausal.
  5. Informed Consent. Documented informed consent/assent must be obtained before any study-specific procedure. Ages 12-15: consent from person(s) with parental responsibility plus age-adapted information and assent/participation according to maturity. Ages 16-17: participant informed consent plus consent from person(s) with parental responsibility. If a participant reaches 18 years before further protocol-defined procedures, express adult informed consent must be obtained.

Exclusion criteria 4

  1. Medical condition. Renal failure
  2. Medical condition. Hypersensitivity/Allergy to contrast agents
  3. Medical condition. Lowered seizure threshold
  4. Other. Pregnancy or breast feeding.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Exploratory model-derived BFC indices [absorption half-life (t½abs), time to maximal blood concentration (Tmax) of gadobutrol and maximum concentration (Cmax)] from the predefined gadobutrol popPK model using actual dose and sampling times.

Secondary endpoints 3

  1. Solicited AEs/AESIs Day 0-7, including headache, nausea, dizziness and other predefined symptoms; SAE/SUSAR occurrence; 30-minute post-dose observation findings.
  2. Mental Fatigue Scale score at 3 months.
  3. Change in neuropsychological test/questionnaire measures from baseline to 3 months and 2 years.

Investigational products

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

Test 1

Gadobutrol 1.0 mmol/mL solution for injection

PRD10809539 · Product

Active substance
Gadobutrol
Pharmaceutical form
SOLUTION FOR INJECTION
Route of administration
INTRATHECAL USE
Max daily dose
60 mg milligram(s)
Max total dose
60 mg milligram(s)
Max treatment duration
1 Day(s)
Authorisation status
Authorised
ATC code
V08CA09 — GADOBUTROL
Marketing authorisation
PL 00221/0400
MA holder
GE HEALTHCARE LIMITED
MA country
XI
Paediatric formulation
No
Orphan designation
No
Modified vs. Marketing Authorisation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Oslo University Hospital HF

Sponsor organisation
Oslo University Hospital HF
Address
Taarnbygget, Kirkeveien 166 Kirkeveien 166
City
Oslo
Postcode
0450
Country
Norway

Scientific contact point

Organisation
Oslo University Hospital HF
Contact name
Per Kristian Eide

Public contact point

Organisation
Oslo University Hospital HF
Contact name
Per Kristian Eide

Locations

1 EU/EEA country · 1 investigational sites

By country

CountryMS statusPlanned subjectsSites
Norway Authorised, recruitment pending 50 1
Rest of world 0

Investigational sites

Norway

1 site · Authorised, recruitment pending
Oslo University Hospital HF
Neurosurgery, Taarnbygget, Kirkeveien 166, Oslo

Results and documents

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

Documents 5 files

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

TypeTitleVersion
Protocol (for publication) D1_Protocol_EU CT 2025-522453-20-00_For publication 3
Recruitment arrangements (for publication) K1_Recruitment _EU CT 2025-522453-20-00 1
Subject information and informed consent form (for publication) L1_SIS-ICF_EU CT 2025-522453-20-00_Barn 12-15 1
Summary of Product Characteristics (SmPC) (for publication) E2_SmPC EU CT 2025-522453-20-00 1
Synopsis of the protocol (for publication) D1_NO_Synopsis_EU CT 2025-522453-20-00 2

Application history

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

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2026-03-13 Norway Acceptable
2026-06-29
2026-07-03