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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 20 sites in 7 countries.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

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
20 sitesin 7 countries
Study dates
30 Mar 2026to Mar 2031
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

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

20 sites in 7 countries. Contact the study team or a site near you to ask about taking part.

20 sites in 7 countries

United States8 sites, 8 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

    Recruiting

Other trials in United States

France3 sites, 3 recruiting

  • La Timone Children's Hospital

    Marseille, 13005

    Gabriel Revon-Riviere, Dr, 04 91 38 68 22, gabriel.revon-riviere@ap-hm.fr

    Recruiting

  • Institut Curie

    Paris, 75005

    Amaury Leruste, Dr, +33 (0)1 44 32 40 15, amaury.leruste@curie.fr

    Recruiting

  • Gustave Roussy

    Villejuif, 94800

    Pablo Berlanga, Dr, (+33) 142 11 41 67, PABLO.BERLANGA@gustaveroussy.fr

    Recruiting

Other trials in France

Spain3 sites, 3 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

    Recruiting

  • Hospital Infantil Universitario Niño Jesús

    Madrid, 28009

    Beatriz Vergara Munoz, Dr, +34616531224, beatriz.vergara@salud.madrid.org

    Recruiting

Other trials in Spain

United Kingdom3 sites, 3 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

    Recruiting

Other trials in United Kingdom

Canada1 site, 1 recruiting

  • The Hospital for Sick Children

    Toronto, Ontario, M5G 1E8

    Daniel Morgenstern, DR, 416-813-7654 ext. 427565, daniel.morgenstern@sickkids.ca

    Recruiting

Other trials in Canada

Denmark1 site, 1 recruiting

  • Rigshospitalet

    Copenhagen, 2100

    Karsten Nysom, Dr, 35 45 08 09, karsten.nysom@regionh.dk

    Recruiting

Other trials in Denmark

Germany1 site, 1 recruiting

  • Charité - Universitätsmedizin Berlin

    Berlin, 13353

    Hedi Deubzer, Dr, +49 30 450 566 715, hedwig.deubzer@charite.de

    Recruiting

Other trials in Germany

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.

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 groups in NCT07549321
Study groupRoleWhat they receive
Higher doseOtherNot described
Receives: Drug: hu1418K322A + Temozolomide + Irinotecan
Lower doseOtherNot described
Receives: Drug: hu1418K322A + Temozolomide + Irinotecan

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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)

  2. 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)

  1. Overall survival (OS)

    Measured: Up to 3 years

  2. Overall Metastatic Response Rate (mORR)

    Measured: Assessed at end of treatment (up to 12 months)

  3. Duration of Response (DOR)

    Measured: From the time of first response until disease progression, discontinuation from study, start of new therapy, or death

  4. Progression-Free Survival (PFS)

    Measured: From start of Cycle 1 until the first occurrence of progression followed for up to 3 years

  5. 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.

  1. First posted24 Apr 2026
  2. Study start30 Mar 2026
  3. Primary completionMar 2031 (estimated)
  4. Study completionMar 2031 (estimated)

Resources

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)
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
3 Nov 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.

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing hu1418K322A + Temozolomide + Irinotecan in people with High-Risk Neuroblastoma, run by Renaissance Pharma Ltd. at 20 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 3 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in United States, France, Spain, United Kingdom, Canada and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall response rate (ORR) (time frame: Assessed at end of treatment (up to 12 months)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07549321.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2026.

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