An experimental trial to learn about hu14.18K322A treatment in children and adolescents with high-risk neuroblastoma.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Renaissance Pharma Limited

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Decision date (initial)

2026-06-30

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Renaissance Pharma Ltd.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Safety, Others, Efficacy

To evaluate the efficacy of hu14.18K322A treatment given in combination with chemotherapy in participants with high-risk neuroblastoma.
Sub Protocol 1: To evaluate the efficacy of hu14.18K322A treatment given in combination with chemotherapy in participants with HRNB relapsed on treatment
Sub Protocol 2: To evaluate the efficacy of hu14.18K322A treatment on metastatic disease when given in combination with chemotherapy in participants with HRNB refractory to treatment

Secondary objectives 5

1. To evaluate the safety and tolerability of hu14.18K322A treatment given in combination with chemotherapy in participants with high-risk neuroblastoma
2. To evaluate the patient reported outcomes or observer reported outcomes of hu14.18K322A treatment given in combination with chemotherapy in participants with high-risk neuroblastoma
3. To assess immunogenicity of hu14.18K322A treatment given in combination with chemotherapy in participants with high-risk neuroblastoma
4. To explore the predictive potential of selected biomarkers measured in samples collected in response to hu14.18K322A
5. To characterize pharmacokinetic parameters of hu14.18K322A treatment given in combination with chemotherapy in participants with high-risk neuroblastoma

Conditions and MedDRA coding

Participants with relapsed HRNB and participants with HRNB refractory to treatment The clinical trial will recruit participants aged ≥18 months to <18 years, and therefore all participants will be minors at the time of consent.

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration, Medicines And Healthcare Products Regulatory Agency

Plan to share IPD

Yes

IPD plan description

Individual Patient Data (IPD) will be collected for all study participants and securely stored in the sponsor’s clinical database. All datasets will be pseudonymised to protect subject confidentiality and handled in accordance with applicable data protection requirements, including GDPR. The IPD may be made available to competent authorities and regulatory bodies for review, verification, or inspection purposes upon request. Access will be granted under controlled conditions and only for legitimate regulatory assessment, ensuring that personal identifiers are removed or replaced and that data processing complies with ethical and legal standards

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1. ≥18 months to <18 years 2. Initially diagnosed with histologically proven HRNB with metastatic disease 3. Have evaluable or measurable disease per INRC, (Park, 2017) 4. Have a Lansky performance status of ≥50 or Karnofsky performance status ≥50% 5. Have recovered from the toxic effects of prior chemotherapies as per institutional guidelines 6. Are at least 2 weeks beyond any major tumor surgery and fully recovered from any post-surgical complications 7. Meet the following organ function criteria, as measured within 1 week prior to IMP dosing: a. BM function: i. Platelets ≥50 × 109/L ii. Absolute neutrophil count (ANC) ≥0.50 × 109/L b. Renal function: i. Age-adjusted serum creatinine ≤1.5 × ULN for age ii. eGFR at least 60 mL/min using the Schwartz formula c. Liver function: AST or ALT ≤3 × ULN and total bilirubin ≤1.5 × ULN. In case of liver metastases, AST or ALT ≤5 × ULN and total bilirubin ≤1.5 × ULN d. Cardiac function: i. Shortening fraction ≥27% or ejection fraction of ≥50% on echocardiogram ii. QTc on ECG using QTcF ≤ 450 msec e. Lung function: i. Pulse oximetry considered normal in room air, per Investigator judgment. No evidence of dyspnea at rest. f. CNS function: i. No clinical or radiological evidence of active CNS disease at the time of study enrollment ii. Participants with seizure disorders may be enrolled if seizures are well controlled on anti-seizure medications iii. No active CNS toxicity 8. Consent to pregnancy testing and use of an acceptable contraception method if applicable 9. If a fertile and sexually active male, agree to use condoms 10. Are willing and able to provide voluntary written informed assent 11. Parent or legal guardian is willing and able to provide and has provided voluntary written informed consent Sub Protocol A: a. Patients experiencing their first or second relapse having achieved disease stabilization or an initial response (SD, MR, PR or CR) to previous therapy b. If received prior anti-GD2 therapy, last dose administered at least 14 days previously and no ongoing toxicities c. If applicable, at least 6 weeks from AHCT following myeloablative therapy Sub Protocol B: a. HRNB participants who are first line refractory following a minimum of 4 cycles of induction chemotherapy with no history of PD, where refractory is defined as SD, MR or PR with at least one metastatic lesion remaining b. Identification of primary tumor documented c. Measurable disease by cross sectional imaging INRC, (Park, 2017).

Exclusion criteria 1

1. 1. Any active uncontrolled infection at the time of enrollment. Any known history of infection with HIV, or active or chronic infection with HCV/HBV 2. Any contraindications to any of the study treatments. 3. Patients with >Grade 2 diarrhea. 4. Patients with disease of any major organ system. 5. Patients who have undergone a prior allogeneic stem cell transplant < 6 months ago or have undergone a solid organ transplant. 6. Patients who are on hemodialysis. 7. Patients who require or are likely to require pharmacologic doses of systemic corticosteroids. 8. Patients on any other immunosuppressive medications 9. Patients who have received enzyme-inducing anticonvulsants for at least 7 days prior to study enrollment. 10. Patients who have been diagnosed with any malignancy other than neuroblastoma. 11. Patients with symptoms of congestive heart failure. 12. Patients with a history of Grade 4 allergic reactions to anti-GD2 antibodies or reactions that required permanent discontinuation of the anti-GD2 therapy. 13. Patients involved in another blinded study or an IMP study within 14 days or 5 half-lives before Screening or during the study. 14. Females who are breastfeeding, pregnant or planning pregnancy. 15. Failure to meet the required washout periods before the first dose of hu14.18K322A 16. Ongoing need for any medication known or suspected to interfere with study treatment. 17. Any underlying condition that would jeopardise the safety of the participant Sub Protocol A: a. Patients with refractory only disease. b. Patients who progress during induction. c. Patients who failed to stabilize or respond to prior anti-GD2 mAb in combination with chemotherapy treatment. Sub Protocol B: a. Evidence of PD in the primary tumor, in metastatic soft tissue and skeletal site b. Initiation of consolidation or maintenance treatment. c. Prior anti-GD2 mAb therapy.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Primary efficacy endpoint defined in Sub Protocol, Sub Protocol A: ORR defined as the proportion of participants treated with hu14.18K322A in combination with chemotherapy who achieve a CR or PR according to the INRC Sub Protocol B: mCRR defined as the proportion of participants treated with hu14.18K322A in combination with chemotherapy who achieve an overall mCR response

Secondary endpoints 1

1. Multiple secondary endpoints as described in Protocol Section M.2, A2 and B2.

Investigational products

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

Test 3

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Renaissance Pharma Limited

Sponsor organisation

Renaissance Pharma Limited

Address

8a Crabtree Road, Thorpe Industrial Estate Thorpe Industrial Estate

City

Egham

Postcode

TW20 8RN

Country

United Kingdom

Scientific contact point

Organisation

Renaissance Pharma Limited

Contact name

Patricia Marosics

Public contact point

Organisation

Renaissance Pharma Limited

Contact name

Clinical Trial Information Desk

Third parties 13

Locations

3 EU/EEA countries · 7 investigational sites

By country

Investigational sites

Results and documents

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

Documents 119 files

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

Application history

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