Informations générales (source: ClinicalTrials.gov)
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
Interventional
Phase 2/Phase 3
Renaissance Pharma Ltd. (Voir sur ClinicalTrials)
mars 2026
mars 2031
27 octobre 2026
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.
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| CLCC INSTITUT GUSTAVE ROUSSY | Pablo BERLANGA | 27/10/2026 14:35:02 | Contacter | ||
Critères
Tous
Inclusion Criteria
1. Are ≥18 months to <18 years of age at time of informed consent or assent.
2. Are initially diagnosed with histologically proven HRNB with metastatic disease.
3. Have evaluable or measurable disease per International Neuroblastoma Response
Criteria (INRC)
4. Have a Lansky performance status of ≥50 (≤16 years ) or Karnofsky performance status
≥50% (for >16 years).
5. Have recovered from the toxic effects of prior chemotherapies
6. Are at least 2 weeks beyond any major tumor surgery
7. Meet the following organ function criteria, as measured within 1 week prior to
Investigational Medicinal Product (IMP) dosing:
1. BM function: i. Platelets ≥50 × 109/L ii. Absolute neutrophil count (ANC) ≥0.50
× 109/L
2. 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.
3. Liver function: Aspartate aminotransferase (AST) or alanine aminotransferase
(ALT) ≤3 × ULN and total bilirubin ≤1.5 × ULN.
4. 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.
5. Lung function: i. Pulse oximetry considered normal in room air. No evidence of
dyspnea at rest.
6. 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.
8. 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 .
9. 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
10. 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.
11. 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.
Exclusion Criteria
1. 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).
2. Any contraindications to any of the study treatments.
3. Patients with >Grade 2 diarrhea.
4. Patients with disease of any major organ system that would compromise their ability
to withstand chemoimmunotherapy.
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 while receiving treatment on this study except to manage allergic
reactions
8. Patients on any other immunosuppressive medications
9. Patients who have received enzyme-inducing anticonvulsants including phenytoin,
phenobarbital, or carbamazepine 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 participating in or planning to participate in another study that is either
blinded or involves an IMP
14. 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
15. Do not meet the following required washout periods prior to the administration of
the first dose of hu14.18K322A:
1. 14 days from prior systemic myelosuppressive chemotherapy.
2. 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).
3. 14 days from systemic steroids.
4. ≥12 weeks from large field radiation therapy (i.e., total body irradiation,
whole abdominal, total lung, ≥50% pelvis).
5. 6 weeks from prior craniospinal radiotherapy or
Iodine-131-metaiodobenzylguanidine (131I-MIBG) therapy.
6. 2 weeks from radiotherapy to the primary tumor bed.
7. ≥7 days from small treatment field radiation.
8. 14 days or 5 half-lives (whichever is longer) from last administration of an
IMP.
9. >7 days prior to study enrollment for drugs that are strong inducers or
inhibitors of Cytochrome P450 3A4 (CYP3A4).
10. ≥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).
16. Ongoing need for any medication known or suspected to interfere with study
treatment.
1. Are ≥18 months to <18 years of age at time of informed consent or assent.
2. Are initially diagnosed with histologically proven HRNB with metastatic disease.
3. Have evaluable or measurable disease per International Neuroblastoma Response
Criteria (INRC)
4. Have a Lansky performance status of ≥50 (≤16 years ) or Karnofsky performance status
≥50% (for >16 years).
5. Have recovered from the toxic effects of prior chemotherapies
6. Are at least 2 weeks beyond any major tumor surgery
7. Meet the following organ function criteria, as measured within 1 week prior to
Investigational Medicinal Product (IMP) dosing:
1. BM function: i. Platelets ≥50 × 109/L ii. Absolute neutrophil count (ANC) ≥0.50
× 109/L
2. 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.
3. Liver function: Aspartate aminotransferase (AST) or alanine aminotransferase
(ALT) ≤3 × ULN and total bilirubin ≤1.5 × ULN.
4. 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.
5. Lung function: i. Pulse oximetry considered normal in room air. No evidence of
dyspnea at rest.
6. 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.
8. 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 .
9. 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
10. 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.
11. 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.
Exclusion Criteria
1. 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).
2. Any contraindications to any of the study treatments.
3. Patients with >Grade 2 diarrhea.
4. Patients with disease of any major organ system that would compromise their ability
to withstand chemoimmunotherapy.
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 while receiving treatment on this study except to manage allergic
reactions
8. Patients on any other immunosuppressive medications
9. Patients who have received enzyme-inducing anticonvulsants including phenytoin,
phenobarbital, or carbamazepine 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 participating in or planning to participate in another study that is either
blinded or involves an IMP
14. 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
15. Do not meet the following required washout periods prior to the administration of
the first dose of hu14.18K322A:
1. 14 days from prior systemic myelosuppressive chemotherapy.
2. 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).
3. 14 days from systemic steroids.
4. ≥12 weeks from large field radiation therapy (i.e., total body irradiation,
whole abdominal, total lung, ≥50% pelvis).
5. 6 weeks from prior craniospinal radiotherapy or
Iodine-131-metaiodobenzylguanidine (131I-MIBG) therapy.
6. 2 weeks from radiotherapy to the primary tumor bed.
7. ≥7 days from small treatment field radiation.
8. 14 days or 5 half-lives (whichever is longer) from last administration of an
IMP.
9. >7 days prior to study enrollment for drugs that are strong inducers or
inhibitors of Cytochrome P450 3A4 (CYP3A4).
10. ≥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).
16. Ongoing need for any medication known or suspected to interfere with study
treatment.