Clinical Analysis of Naxitamab (hu3F8) in the Treatment of Pediatric High Risk or Refractory/ Relapsed Neuroblastoma

Sun Yat-sen University

Status and phase

Enrolling

Phase 2

Conditions

Neuroblastoma

Treatments

Drug: GM-CSF

Drug: GM-CSF with combination regimen

Drug: Naxitamab in combination therapy

Drug: Sintilimab

Drug: Naxitamab monotherapy

Drug: Irinotecan

Drug: Temozolomide

Study type

Interventional

Funder types

Other

Identifiers

NCT06013618

3F8-RWS

Details and patient eligibility

About

This is an prospective study to evaluate the safety and efficacy of naxitamab monotherapy or combined with chemotherapy or combined with chemotherapy and checkpoint inhibitor in the treatment of pediatric high-risk and refractory/relapsed neuroblastoma in Sun Yat-sen University Cancer Center.

Full description

Patients with high risk neuroblastoma who obtain CR after chemotherapy combined with surgery, radiotherapy and/or hematopoietic stem cell transplantation received axitamab and GM-CSF. Patients with high-risk neuroblastoma treated by chemotherapy combined with surgery, radiotherapy and or hematopoietic stem cell transplantation patients with tumor residual or progression during treatment (refractory) received naxitamab and GM-CSF in combination with irinotecan and temozolomide or naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody.

Enrollment

120 estimated patients

Sex

All

Ages

12+ months old

Volunteers

No Healthy Volunteers

Inclusion criteria

1)Confirmed diagnosis of high-risk NB 2)1 year of age or above 3)Patient or parent/guardian must provide written informed consent to participate 4)If patient is sexually active, the patient agrees to use effective contraception 5)Confirmed negative urine pregnancy test for sexually active female of child-bearing potential (post-menarche)

Exclusion criteria

Significant organ toxicity
Known or suspected allergy or hypersensitivity to anti-GD2 antibodies or to GM-CSF or its s components.
Patient is pregnant, planning to become pregnant (while being treated with naxitamab) or is currently breastfeeding
Patient will undergo treatment with another investigational drug, whilst being treated with naxitamab or has received another investigational drug within the 4 weeks prior to commencing treatment with naxitamab
Patient is either eligible and able to participate in or is currently participating in an active interventional Y-mAbs sponsored clinical trial with naxitamab within the indication applied for
Patient is unable to comply with the naxitamab treatment or has a medical condition that would potentially increase the severity of the toxicities experienced from naxitamab treatment at the discretion of the treating physician
Left ventricular ejection fraction of <50% by echocardiography OR other clinically relevant cardiac disorders at the discretion of the investigator
Inadequate pulmonary function defined as evidence of dyspnea at rest, exercise intolerance, and/or chronic oxygen requirement. In addition, room air pulse oximetry < 94% and/or abnormal pulmonary function tests if these assessments are clinically indicated

Applicable for treatment with naxitamab in combination with GM-CSF only:
Patient has active progression of the NB disease
Patient has active NB disease at primary site or soft-tissue metastasis
Patient has known CNS metastases when initiating naxitamab treatment

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

120 participants in 3 patient groups

naxitamab and GM-CSF only

Other group

Description:

Suitable for patients with high risk neuroblastoma who obtain CR after chemotherapy combined with surgery, radiotherapy and/or hematopoietic stem cell transplantation. The treatment cycle is repeated every 4 weeks for a total of 5 courses, and discontinuation of nasetuzumab and GM-CSF should be considered if disease progression or unacceptable toxicity occurs.

Treatment:

Drug: Naxitamab monotherapy

Drug: GM-CSF

naxitamab and GM-CSF in combination with irinotecan and temozolomide

Other group

Description:

Suitable for high-risk group neuroblastoma treated by chemotherapy combined with surgery, radiotherapy and or hematopoietic stem cell transplantation patients with tumor residual or progression during treatment (refractory); Patients who relapse after initial treatment. Repeat every 3 weeks until tumor progression, patient withdrawal, or toxicity becomes intolerable, up to 8 procedures.

Treatment:

Drug: Temozolomide

Drug: Irinotecan

Drug: Naxitamab in combination therapy

Drug: GM-CSF with combination regimen

naxitamab and GM-CSF in combination with irinotecan and temozolomide and PD-1 antibody

Other group

Description:

Treatment:

Drug: Temozolomide

Drug: Irinotecan

Drug: Sintilimab