Recurrent Stage IV High Risk Neuroblastoma
Conditions
Brief summary
A phase I observational study on the safety and efficacy of treatment of recurrent stage IV high risk neuroblastoma with Nivolumab
Interventions
DRUGSintilimab
Sintilimab is administered every 21 days until the disease progresses or treatment is terminated due to unacceptable toxicity. For patients with clinical and radiologic benefits, treatment can last up to 2 years. Dose: 2 mg / kg intravenously for 60 min (± 10 min window)
Sponsors
Shanghai Children's Medical Center
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Months to 12 Years
Healthy volunteers
No
Inclusion criteria
1. 12 Months to 12 years 2. Histologic verification of stage IV high risk neuroblastoma at relapse following lack of complete response to at least two lines of therapy 3. Fully recovered from the acute toxic effects of all prior anti-cancer treatment 4. At least 21 days after the last dose of myelosuppressive chemotherapy (42 days if prior nitrosourea) 5. At least 42 days after the completion of any type of immunotherapy, e.g. tumor vaccines 6. At least 56 days must have elapsed after transplant or stem cell infusion; patients with prior allogeneic transplants are not eligible 7. Blood counts recovery including White cell count \>= 750/mm\^3 and Platelet count \>= 50,000/mm\^3 8. Creatinine clearance ≥ 50ml/min 9. Liver function: Total bilirubin ≤ 2 mg/dl, Alanine aminotransferase or Aspartate aminotransferase ≤ 2.5 U/dl (or \< 5 in case of liver impairment) 10. Life expectancy of at least 4 months 11. Negative pregnancy test in women of childbearing potential 12. Use of an effective contraceptive method during the whole treatment and 13. up to 3 months after the completion of treatment in males and females 14. Prior informed consent signed
Exclusion criteria
1. Patients requiring daily systemic corticosteroids are not eligible; patients must not have received systemic corticosteroids within 7 days of enrollment on study 2. Patients who are currently receiving another investigational drug are not eligible 3. Patients who are currently receiving other anti-cancer agents are not eligible 4. Patients with a history of any grade autoimmune disorder are not eligible; asymptomatic laboratory abnormalities (e.g. antinuclear antibody (ANA), rheumatoid factor, altered thyroid function studies) will not render a patient ineligible in the absence of a diagnosis of an autoimmune disorder 5. Patients with \>= grade 2 hypothyroidism due to history of autoimmunity are not eligible; note: hypothyroidism due to previous irradiation on thyroidectomy will not impact eligibility 6. Patients who have an uncontrolled infection are not eligible. 7. Patients with active autoimmune disease. (any autoimmune state requiring medical treatment-including chronic medications)all immune modifying drugs should be stopped at least 7 days prior to enrollment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| response | 2 years after taking the drug | Complete response: (CR): the tumor shrunk more than 50%. Part of the reaction: (PR): the reduction of tumor body was more than 30%. No response: (NP): the tumor decreased by less than 30% or increased. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| side effect | 2 years after taking the drug | Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 |
Other
| Measure | Time frame | Description |
|---|---|---|
| Mutation | before taking the drug | Mutation load, high microsatellite instability (MSI-H) or mismatch repair defect (dMMR) |
| programmed death-ligand1 (PD-L1) expression | before taking the drug | immunohistochemistry (IHC) test for PD-L1 and cluster of differentiation 8 (CD8) expression in tumor tissue |
Countries
China
Contacts
Primary ContactSong Gu, MD
Outcome results
None listed