A Safety and Efficacy Study of hu14 in High-Risk Neuroblastoma Patients

NCT07549321 | Recruiting Phase 2 DMC FDA Drug

• 5 other identifiers: EPG-HU1418-2011691692025-524397-42-001013435Health Canada
• Study Type: interventional
• Target: 144
• Locations: 1 country
• Sites: 2

Brief 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). 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.

Conditions

High-Risk Neuroblastoma

Outcome Measures

Primary Outcomes (2)

• 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)

  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

  Assessed at end of treatment (up to 12 months)

Secondary Outcomes (5)

• Overall survival (OS)

  Up to 3 years

• Overall Metastatic Response Rate (mORR)

  Assessed at end of treatment (up to 12 months)

• Duration of Response (DOR)

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

• Progression-Free Survival (PFS)

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

• Event-Free Survival (EFS)

  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

Study Arms (2)

Higher dose

OTHER

Drug: hu1418K322A + Temozolomide + Irinotecan

Lower dose

OTHER

Drug: hu1418K322A + Temozolomide + Irinotecan

Interventions

hu1418K322A + Temozolomide + Irinotecan DRUG

21-day cycle for a maximum of 12 cycles

Higher dose; Lower dose

Eligibility Criteria

• Age: 18 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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
• 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

You may not qualify if:

• 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.
• 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:

Sponsors & Collaborators

• Renaissance Pharma Ltd.: lead

Study Sites (2)

Children's Hospital Colorado Anschutz Medical

Aurora, Colorado, 80045, United States

RECRUITING

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

MeSH Terms

Interventions

Temozolomide; Irinotecan

Intervention Hierarchy (Ancestors)

Dacarbazine; Triazenes; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Camptothecin; Alkaloids

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Two parts study. Part 1 (n=12) is randomised (open label). Part 2 (n=132) is single arm (open label) at selected dose.

Study Record Dates

• First Submitted: April 9, 2026
• First Posted: April 24, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): March 1, 2031
• Study Completion (Estimated): March 1, 2031
• Last Updated: May 1, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)