Clinical Study of Eflornithine After Immunotherapy for High-risk Neuroblastoma（CSHEIN）
Overview
- Phase
- Not Applicable
- Status
- Active, not recruiting
- Sponsor
- Shanghai Children's Hospital
- Enrollment
- 20
- Locations
- 1
- Primary Endpoint
- The event free survival rate after the completion of the first DFMO
Overview
Brief Summary
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.
Detailed Description
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 Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 1 Month to 18 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •According to the International Neuroblastoma Risk Group Classification, it has been histologically diagnosed as high-risk NB.
- •Under the age of
- •The disease assessment status is PR (via CT or MRI) and the bone marrow smear is negative.
- •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.
- •Qualified hematological parameters and organ function; Refer to the CI CTC 4.0 adverse reaction grading standard of grade 2 and below.
- •Eflornithine needs to be activated within 120 days after the completion of previous treatment.
Exclusion Criteria
- •According to the International Neuroblastoma Risk Group Classification, it has been histologically diagnosed as non high risk NB.
- •Prior to enrollment, the disease assessment status was either progressive disease (PD) or relapse (via CT or MRI).
- •Positive bone marrow smear.
- •Hematological parameters and organ function are not qualified, according to the CI CTC 4.0 adverse reaction grading standard of grade 3 or above.
- •The guardian does not agree to participate.
Outcomes
Primary Outcomes
The event free survival rate after the completion of the first DFMO
Time Frame: 5 years
The event free survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy.
overall survival rate after the completion of the first DFMO
Time Frame: 5 years
overall survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy.
Secondary Outcomes
- Incidence of Treatment-Emergent Adverse Events(5 years)