Clinical trial NCT06013618: Clinical Analysis of Naxitamab (hu3F8) in the Treatment of Pediatric High Risk or Refractory/ Relapsed Neuroblastoma
Official title: Clinical Analysis of Naxitamab (hu3F8) in the Treatment of Pediatric High Risk or Refractory/ Relapsed Neuroblastoma
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Neuroblastoma
Lead sponsor: Sun Yat-sen University
Sponsor class: OTHER
Collaborators: name: Hainan General Hospital
First posted: 2023-08-28
Enrollment: 120
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: 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.

Detailed 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.

Eligibility criteria: 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:

1. Significant organ toxicity
2. Known or suspected allergy or hypersensitivity to anti-GD2 antibodies or to GM-CSF or its s components.
3. Patient is pregnant, planning to become pregnant (while being treated with naxitamab) or is currently breastfeeding
4. 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
5. 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
6. 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
7. Left ventricular ejection fraction of \<50% by echocardiography OR other clinically relevant cardiac disorders at the discretion of the investigator
8. 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:
9. Patient has active progression of the NB disease
10. Patient has active NB disease at primary site or soft-tissue metastasis
11. Patient has known CNS metastases when initiating naxitamab treatment

Primary outcomes:
measure: ORR
timeFrame: from start of naxitamab treatment to 1.5 years after EOT
Secondary outcomes:
measure: DCR
timeFrame: from start of naxitamab treatment to 1.5 years after EOT
measure: EFS
timeFrame: from start of naxitamab treatment to 1.5 years after EOT
measure: OS
timeFrame: from start of naxitamab treatment to 1.5 years after EOT
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Sun Yat-sen University Cancer Center
state: Guangdong
Study officials:
affiliation: SunYat Sen University Cancer Center
name: Yizhuo Zhang
role: STUDY_CHAIR
References: