Clinical Study of Eflornithine After Immunotherapy for High-risk Neuroblastoma（CSHEIN）

N/AActive Not RecruitingNCT07278674

Shanghai Children's Hospital20 enrolled

Overview

Evaluate the impact of maintenance therapy with eflornithine on event-free survival and overall survival in high-risk neuroblastoma (NB) children after immunotherapy, and assess its safety.

The investigators plan to recruit 20 participants and divide them into Group A and Group B based on their clinical status. The inclusion criteria for Group A are: newly diagnosed high-risk neuroblastoma (NB) patients who have completed the standard treatment plan (including immunotherapy) and whose disease has reached complete response (CR) or very good partial response (VGPR). The inclusion criteria for Group B are: recurrent or refractory NB patients who have completed any treatment for recurrent disease, or who have achieved disease stability after any salvage or intensification therapy for primary refractory disease, with at least a partial response (PR) as assessed by CT or MRI and negative bone marrow aspiration. There are no restrictions regarding gender or geographic region. Participants must be under 18 years of age. Eligible subjects will be enrolled into the study after receiving the first oral dose of eflornithine, with a total treatment course of 2 years. Follow-up will continue for up to 3 years after completion of eflornithine therapy.

Study Type

OBSERVATIONAL

Enrollment

Conditions

High Risk Neuroblastoma

Interventions

Oral administration of eflornithine for the prevention of high-risk neuroblastomaDRUG

This study is a single arm clinical observational study and does not involve control interventions

Eligibility

Sex: ALLMin age: 1 MonthMax age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. According to the International Neuroblastoma Risk Group Classification, it has been histologically diagnosed as high-risk NB. 2. Under the age of 18. 3. The disease assessment status is PR (via CT or MRI) and the bone marrow smear is negative. 4. If the residual mass is MIBG negative or MIBG positive and lacks FDG-PET affinity, it is considered evidence that the mass does not represent active disease. Subjects with stable residual tumor mass visible on CT/MRI will be included in the study. 5. Qualified hematological parameters and organ function; Refer to the CI CTC 4.0 adverse reaction grading standard of grade 2 and below. 6. Eflornithine needs to be activated within 120 days after the completion of previous treatment. Exclusion Criteria: 1. According to the International Neuroblastoma Risk Group Classification, it has been histologically diagnosed as non high risk NB. 2. Prior to enrollment, the disease assessment status was either progressive disease (PD) or relapse (via CT or MRI). 3. Positive bone marrow smear. 4. Hematological parameters and organ function are not qualified, according to the CI CTC 4.0 adverse reaction grading standard of grade 3 or above. 5. The guardian does not agree to participate.

Locations (1)

Ruijin Hospital Hainan Branch, affiliated with Shanghai Jiao Tong University School of Medicine and Children's Hospital of Shanghai, affiliated with Shanghai Jiao Tong University School of Medicine

Hainan, China

Outcomes

Primary Outcomes

The event free survival rate after the completion of the first DFMO

The event free survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy.

Time frame: 5 years

overall survival rate after the completion of the first DFMO

overall survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy.

Time frame: 5 years

Secondary Outcomes

Incidence of Treatment-Emergent Adverse Events

This study will comprehensively evaluate the safety and tolerability of eflornithine. The primary assessments include the incidence and severity of all treatment-emergent adverse events (graded per NCI CTCAE v5.0)

Time frame: 5 years

Data from ClinicalTrials.gov

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