High-Risk NeuroblastomaPhase 2/3SHINENCT07549321
A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma Patients
A Phase 2/3 study testing hu1418K322A + Temozolomide + Irinotecan in people with High-Risk Neuroblastoma, run by Renaissance Pharma Ltd. at 17 sites in 7 countries.
Trial summary
Neuroblastoma is the most common type of solid cancer found outside the brain in young children. Generally, it affects children younger than 5 years old, with the average age when it is found being just 2 years. Most patients have 'high-risk' disease, with spread of the disease to different sites (metastases). Read more about this study
- Who is it for?
- Ages 18 months to 18 years
- Conditions the study is for
- High-Risk Neuroblastoma
- What is being tested?
- hu1418K322A + Temozolomide + Irinotecan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 144 participants
- Where
- 17 sitesin 7 countries
- Study dates
- 30 Mar 2026to Mar 2031
- Phase
- Phase 2/3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Are ≥18 months to <18 years of age at time of informed consent or assent.
- Are initially diagnosed with histologically proven HRNB with metastatic disease.
- Have evaluable or measurable disease per International Neuroblastoma Response Criteria (INRC)
- Have a Lansky performance status of ≥50 (≤16 years ) or Karnofsky performance status ≥50% (for >16 years).
- Have recovered from the toxic effects of prior chemotherapies
- Are at least 2 weeks beyond any major tumor surgery
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- Meet the following organ function criteria, as measured within 1 week prior to Investigational Medicinal Product (IMP) dosing:
- BM function: i. Platelets ≥50 × 109/L ii. Absolute neutrophil count (ANC) ≥0.50 × 109/L
- Renal function: i. Age-adjusted serum creatinine ≤1.5 × upper limit of normal (ULN) for age. ii. Estimated glomerular filtration rate (eGFR) at least 60 mL/min using the Schwartz formula.
- Liver function: Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≤3 × ULN and total bilirubin ≤1.5 × ULN.
- Cardiac function: i. Shortening fraction ≥27% or ejection fraction of ≥50% on echocardiogram. ii. Corrected QT Interval on electrocardiogram (ECG) using the Fridericia formula (QTcF) ≤ 480 msec.
- Lung function: i. Pulse oximetry considered normal in room air. No evidence of dyspnea at rest.
- Central nervous system (CNS) function: i. Participants with a history of CNS disease must have no clinical or radiological evidence of active CNS disease at the time of study enrollment.
- If a fertile and sexually active woman of child-bearing potential (WOCBP), have a negative serum pregnancy test at Screening and then either a negative serum or urine test prior to each cycle and agree to use an acceptable and highly effective contraception method during the study and for at least 6 months after the last day of study treatment .
- If a fertile and sexually active male, agree to use condoms during the study and for at least 6 months after the last dose of study treatment
- If applicable based on age, are willing and able to provide voluntary written informed assent for participation in the study (as per local Institutional Review Board [IRB]/Independent Ethics Committee [IEC] requirements and if applicable based on regional age of consent) and to comply with all protocol requirements.
- Their parent or legal guardian (if applicable based on regional age of consent) is willing and able to provide voluntary written informed consent for the participant's involvement in the study.
Who cannot join
- Any active uncontrolled infection at the time of enrollment. Any known history of infection with human immunodeficiency virus (HIV), or active or chronic infection with hepatitis B virus (HBV) or hepatitis C virus (HCV).
- Any contraindications to any of the study treatments.
- Patients with >Grade 2 diarrhea.
- Patients with disease of any major organ system that would compromise their ability to withstand chemoimmunotherapy.
- Patients who have undergone a prior allogeneic stem cell transplant < 6 months ago or have undergone a solid organ transplant.
- Patients who are on hemodialysis.
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- Patients who require or are likely to require pharmacologic doses of systemic corticosteroids while receiving treatment on this study except to manage allergic reactions
- Patients on any other immunosuppressive medications
- Patients who have received enzyme-inducing anticonvulsants including phenytoin, phenobarbital, or carbamazepine for at least 7 days prior to study enrollment.
- Patients who have been diagnosed with any malignancy other than neuroblastoma.
- Patients with symptoms of congestive heart failure.
- Patients with a history of Grade 4 allergic reactions to anti-GD2 antibodies or reactions that required permanent discontinuation of the anti-GD2 therapy.
- Patients participating in or planning to participate in another study that is either blinded or involves an IMP
- If female, are breastfeeding, pregnant, or planning to become pregnant, or, if sexually active and of child-bearing potential, are unwilling to use an effective birth control method until 6 months after the last dose of study medication
- Do not meet the following required washout periods prior to the administration of the first dose of hu14.18K322A:
- 14 days from prior systemic myelosuppressive chemotherapy.
- 7 days from prior systemic biologic antineoplastic agents (e.g. anti-cancer agents not known to be myelosuppressive - not associated with reduced platelet or ANC counts).
- 14 days from systemic steroids.
- ≥12 weeks from large field radiation therapy (i.e., total body irradiation, whole abdominal, total lung, ≥50% pelvis).
- 6 weeks from prior craniospinal radiotherapy or Iodine-131-metaiodobenzylguanidine (131I-MIBG) therapy.
- 2 weeks from radiotherapy to the primary tumor bed.
- ≥7 days from small treatment field radiation.
- 14 days or 5 half-lives (whichever is longer) from last administration of an IMP.
- >7 days prior to study enrollment for drugs that are strong inducers or inhibitors of Cytochrome P450 3A4 (CYP3A4).
- ≥21 days and with recovery of all associated toxicities from receiving cellular therapy (e.g., modified T lymphocyte cells, Natural Killer (NK) cells, dendritic cells).
- Ongoing need for any medication known or suspected to interfere with study treatment.
Trial locations and contacts
17 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
17 sites in 7 countries
United States8 sites, 8 recruiting
- Recruiting
Children's Hospital Colorado Anschutz Medical
Aurora, Colorado, 80045
Navin Pinto MD, MD, 720-777-8134, navin.pinto@childrenscolorado.org
- Recruiting
The University of Chicago
Chicago, Illinois, 60637
Ami V Desai, Dr, 773-702-6808, adesai12@bsd.uchicago.edu
- Recruiting
University of Texas Southwestern
Dallas, Texas, 75390
Tanya C Watt, 6147955338, Tanya.Watt@utsouthwestern.edu
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27705
Jessica Sun, MD, 919-684-3401, jessica.sun@duke.edu
- Recruiting
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, 55454
Robin Williams, MD, 612-626-2778, will4877@umn.edu
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Rochelle Bagatell MD, +1 (215) 590-2299, bagatellr@chop.edu
- Recruiting
University of California San Francisco (UCSF)
San Francisco, California, 94143
Kieuhoa Vo, Dr, (415) 476-3831, Kieuhoa.Vo@ucsf.edu
- Recruiting
The Children's Hospital at Montefiore
The Bronx, New York, 10467
Alice Lee, MD, 718-741-2342, alee5@montefiore.org
United Kingdom3 sites, 3 recruiting
- Recruiting
Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
Quentin Campbell-Hewson, Dr, 0191 2821337, quentin.campbellhewson@nhs.net
- Recruiting
Southampton Children's Hospital
Southampton, Hampshire, SO16 6YD
Juliet Gray, Dr, +44-(0)23 8120 5213, juliet.gray@uhs.nhs.uk
- Recruiting
Royal Marsden NHS Foundation Trust
Sutton, SM2 5PT
Sucheta J Vaidya, Dr, 023 8120 6334, sucheta.vaidya@icr.ac.uk
Spain2 sites, 2 recruiting
- Recruiting
University Hospital Vall d'Hebron
Barcelona, 08035
Lucas Moreno Retortillo, Dr, 934 89 30 00 ext. 2887, lucas.moreno@vallhebron.cat
- Recruiting
Hospital Sant Joan de Deu
Barcelona, 08950
Jaume Mora, Dr, +34 93 600 97 33, jaume.mora@sjd.es
Canada1 site, 1 recruiting
- Recruiting
The Hospital for Sick Children
Toronto, Ontario, M5G 1E8
Daniel Morgenstern, DR, 416-813-7654 ext. 427565, daniel.morgenstern@sickkids.ca
Denmark1 site, 1 recruiting
- Recruiting
Rigshospitalet
Copenhagen, 2100
Karsten Nysom, Dr, 35 45 08 09, karsten.nysom@regionh.dk
France1 site, 1 recruiting
- Recruiting
Gustave Roussy
Villejuif, 94800
Pablo Berlanga, Dr, (+33) 142 11 41 67, PABLO.BERLANGA@gustaveroussy.fr
Germany1 site, 1 recruiting
- Recruiting
Charité - Universitätsmedizin Berlin
Berlin, 13353
Hedi Deubzer, Dr, +49 30 450 566 715, hedwig.deubzer@charite.de
About this study
Official title: A Phase 2/3 Study to Characterize and Evaluate the Efficacy, Safety, and Tolerability of hu14.18K322A Treatment Given in Combination With Chemotherapy in Participants With High-Risk Neuroblastoma
Neuroblastoma is the most common type of solid cancer found outside the brain in young children. Generally, it affects children younger than 5 years old, with the average age when it is found being just 2 years. Most patients have 'high-risk' disease, with spread of the disease to different sites (metastases). This multinational study aims to find out how effective and safe the treatment of a monoclonal anti-GD2 antibody hu14.18K322A (daretabart) is when used together with chemotherapy to treat children and young people who have high-risk neuroblastoma.
Read the detailed description
This is a Phase 2/3, open label, single arm study, evaluating how well hu14.18K322A works and how safe it is in combination with chemotherapy, in relapsed and refractory high-risk neuroblastoma (HRNB) patients.
The study is in 2 parts. The first part of the study will test two doses of hu14.18K322A to give with chemotherapy in 12 patients to confirm the best dose. Once the best dose is found, Part 2 of the study will recruit up to an additional 66 participants with relapsed disease and 66 participants with refractory disease.
Children and young people who take part in the study must be between 18 months and 18 years old at the time of consent and have confirmed HRNB that has spread to other parts of the body. It is expected that participants will receive up to 12 cycles of treatment. Once the treatment has finished, participants will be followed up to study long-term effects.
- Conditions
- High-Risk Neuroblastoma
- Being tested
- hu1418K322A + Temozolomide + Irinotecan
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hu1418K322A + Temozolomide + IrinotecanDrug
21-day cycle for a maximum of 12 cycles
| Study group | Role | What they receive |
|---|---|---|
| Higher dose | Other | Not described Receives: Drug: hu1418K322A + Temozolomide + Irinotecan |
| Lower dose | Other | Not described Receives: Drug: hu1418K322A + Temozolomide + Irinotecan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall response rate (ORR)
Proportion of participants treated with hu14.18K322A in combination with chemotherapy who achieve a complete response (CR) or partial response (PR) according to the International Neuroblastoma Response Criteria (INRC)
Measured: Assessed at end of treatment (up to 12 months)
Metastatic complete response rate (mCRR)
Proportion of participants treated with hu14.18K322A in combination with chemotherapy who achieve an overall metastatic complete response (mCR) response
Measured: Assessed at end of treatment (up to 12 months)
Secondary outcomes (5)
Overall survival (OS)
Measured: Up to 3 years
Overall Metastatic Response Rate (mORR)
Measured: Assessed at end of treatment (up to 12 months)
Duration of Response (DOR)
Measured: From the time of first response until disease progression, discontinuation from study, start of new therapy, or death
Progression-Free Survival (PFS)
Measured: From start of Cycle 1 until the first occurrence of progression followed for up to 3 years
Event-Free Survival (EFS)
Measured: From start of Cycle 1 until the first occurrence of progression, secondary malignancy, or death due to any cause followed for up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Apr 2026
- Study start30 Mar 2026
- Primary completionMar 2031 (estimated)
- Study completionMar 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for High-Risk NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Model description
- Two parts study. Part 1 (n=12) is randomised (open label). Part 2 (n=132) is single arm (open label) at selected dose.
- Masking
- None (open label)
- Enrolment
- 144 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Renaissance Pharma Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07549321
- Sponsor's protocol ID
- EPG-HU1418-201
- Other IDs
- 169169, 2025-524397-42-00, 1013435, Health Canada, 2025
- Acronym
- SHINE
- Status last verified
- Oct 2026
- First submitted
- 9 Apr 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: At the current time there is no plan to share data. We have not received a research proposal that would require utilization of IPD. In the event we receive a request to access IPD a full evaluation will be undertaken and the data will be shared as appropriate and in compliance with the applicable regulations.