Study on Sintilimab in Stage IV High Risk Neuroblastoma (SCMC-S-2020)

Clinical Study on the Treatment of Recurrent Stage IV High Risk Neuroblastoma With Sintilimab

A phase I observational study on the safety and efficacy of treatment of recurrent stage IV high risk neuroblastoma with Nivolumab

Study Overview

• Status: Not yet recruiting
• Conditions: Recurrent Stage IV High Risk Neuroblastoma
• Intervention / Treatment: Drug: Sintilimab

• Study Type: Interventional
• Enrollment (Anticipated): 10
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Song Gu, MD; Phone Number: 18930830716; Email: gusong@shsmu.edu.cn

Study Locations

• China
  • Shanghai
    • Shanghai, Shanghai, China, 200127
      • Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine
      • Contact: Song Gu, Doctor; Phone Number: 18930830716; Email: gusong@shsmu.edu.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Sintilimab; 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)	Drug: Sintilimab; 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)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
response	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.	2 years after taking the drug

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
side effect	Number of participants with treatment-related adverse events as assessed by CTCAE v5.0	2 years after taking the drug

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mutation	Mutation load, high microsatellite instability (MSI-H) or mismatch repair defect (dMMR)	before taking the drug
programmed death-ligand1 (PD-L1) expression	immunohistochemistry (IHC) test for PD-L1 and cluster of differentiation 8 (CD8) expression in tumor tissue	before taking the drug

Collaborators and Investigators

• Sponsor: Shanghai Children's Medical Center
• Investigators: Principal Investigator: Min Xu, MD, Shanghai Children's Medical Center

Study record dates

Study Major Dates

• Study Start (Anticipated): September 1, 2020
• Primary Completion (Anticipated): December 31, 2022
• Study Completion (Anticipated): June 30, 2023

Study Registration Dates

• First Submitted: May 27, 2020
• First Submitted That Met QC Criteria: June 1, 2020
• First Posted (Actual): June 2, 2020

Study Record Updates

• Last Update Posted (Actual): August 11, 2020
• Last Update Submitted That Met QC Criteria: August 8, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Neuroectodermal Tumors, Primitive; Neuroectodermal Tumors, Primitive, Peripheral; Neuroblastoma
• Other Study ID Numbers: SCMC-Sintilimab-2020

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No