A phase Ib/II first-in-human imaging study of anti-GD2-800CW in patients with neuroblastoma.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Prinses Maxima Centrum voor Kinderoncologie B.V.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Analytical,Diagnostic,Therapeutic Techniques and Equipment [E]-Surgical Procedures, Operative [E04], Diseases [C] - Neoplasms [C04]

Trial duration

5 Nov 2025 → ongoing

Decision date (initial)

2025-04-22

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

KWF

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

- To establish the recommended phase 2 dose (RP2D), based on efficacy and safety, of the anti-GD2-800CW fluorescent imaging agent in pediatric patients with neuroblastoma.
- To investigate safety of anti-GD2-800CW based on treatment related new AEs.

Secondary objectives 3

1. To assess the pharmacodynamic and pharmacokinetic characteristics of an i.v. injection of anti-GD2-800CW.
2. To correlate clinical imaging data with histopathology data to determine the accuracy for depicting GD-2 expressing vital tumor cells.
3. To assess the intra-operative usability of anti-GD2-800CW to distinguish tumor tissue from surrounding tissue on eyesight of the surgeons.

Conditions and MedDRA coding

Neuroblastoma

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Patients with the diagnosis of neuroblastoma as defined by histopathology (confirmed by the PMC Department of Pathology), who will be operated for NB as standard of care procedure
2. Patients older than 1 year of age and not older than 18 years.
3. Written informed consent from patients and/or from parents or legal guardians, according to local law and regulations

Exclusion criteria 5

1. Previous treatment with Dinutuximab-beta, either alone or in combination with chemotherapy.
2. Pregnancy or positive pregnancy test (urine or serum) in females of childbearing potential. Pregnancy test must be performed within during the screening period and before the administration of the IMP.
3. Breast feeding.
4. Sexually active participants not willing to use highly effective contraceptive method (pearl index <1) as defined in CTFG HMA 2020 (Appendix I) during trial participation and until 6 months after end of protocol therapy.
5. Patients that received prior treatment with chimeric antibodies.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Anti-GD2-800CW related adverse events not to be expected from Dinutuximab-alone infusion
2. Tumor to background ratio (TBR) of at least 2.0 ex vivo and sufficient for detection during surgery. TBR is established by dividing the mean fluorescence signal of the tumor by the mean fluorescence signal of the background. Background is determined by fluorescence intensity in healthy kidney, liver and surrounding background tissue.

Secondary endpoints 3

1. Pharmacokinetics of anti-GD2-800CW; blood samples will be taken at t=0 (just before infusion), t=8h (directly after infusion), t=24h (after infusion), t=72h (after infusion), t=96h (immediately before surgery), t=120h (24h after surgery), t=144h (48h after surgery) and t=192h (4 days after surgery).
2. The sensitivity and specificity of anti-GD2-800CW to detect GD2-expressing, vital tumor cells. Pathologic status of removed fluorescent tissue and of removed fluorescent tissue will be compared to fluorescent status.
3. Clinical visibility: score of ≥ 4.0 on a Likert scale of 1-5 based on the judgement of three surgeons. They will grade the intra-operative visibility of the fluorescence and whether a clear distinction can be made on eyesight between tumor and surrounding tissue based on fluorescence.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Prinses Maxima Centrum voor Kinderoncologie B.V.

Sponsor organisation

Prinses Maxima Centrum voor Kinderoncologie B.V.

Address

Heidelberglaan 25

City

Utrecht

Postcode

3584 CS

Country

Netherlands

Scientific contact point

Organisation

Prinses Maxima Centrum voor Kinderoncologie B.V.

Contact name

Lideke van der Steeg

Public contact point

Organisation

Prinses Maxima Centrum voor Kinderoncologie B.V.

Contact name

TDC Secretary

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

Results and documents

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

Documents 16 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