Study Evaluating the Efficacy and Safety With CAR-T for Relapsed or Refractory Neuroblastoma in Children

NCT02919046 | Unknown

• 1 other identifier: WM-CART-07
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 2

Brief Summary

This single-arm, multicenter clinical study will treat the patient who have relapsed or refractory neuroblastoma with an infusion of the patient's own T cells that have been genetically modified to express a chimeric antigen receptor(CAR)that will bind to tumour cells modified to express the GD2 protein on the cell surface. The study will determine if these modified T cells help the body's immune system eliminate tumour cells .The trial will also study the safety of treatment for CAR-T, how long CAR-T cells stay in the patient's body and the impact on this treatment for survival.

Conditions

Relapsed or Refractory Neuroblastoma

Keywords

neuroblastoma; CAR-T

Outcome Measures

Primary Outcomes (1)

• The overall efficiency of patients with neuroblastoma after autologous CAR-T cell therapy

  The overall efficiency will be determined by the evaluation of CT/MRI scans and bone marrow biopsy. Assessment of tumor remission rate according to International Neuroblastoma Response Criteria. The overall efficiency = (complete remission (CR) number + the number of very good partial remission (VGPR) number + partial response (PR) number + mixed reaction (MR) number + no response (NR) number) / total number of cases receiving treatment.

  28d,56d,90d

Secondary Outcomes (4)

• Progression free survival

  3 years

• Overall survival

  3 years

• Patients-based Quality of Life Evaluation

  3 years

• 3°or above incidence rate of serious adverse reaction related to treatment

  3 years

Study Arms (1)

single arm

EXPERIMENTAL

Name:The Chimeric Antigen Receptor T Cell Immunotherapy (CAR-T) Dosage form：injection Dosage:100ml/time Frequency:0 days,the first day,the second day,29 days,30 days Duration:Total five times

Biological: GD2-targeted CAR-T cells

Interventions

GD2-targeted CAR-T cells BIOLOGICAL

This study have only one arm that is CAR-T experimental arm. Firstly all participators will be attended the screening, who passed the screening for the treatment of CAR-T cells, the CAR-GD2-modified T cells can recognize and kill tumor cells in the body，follow-up 35 months.

single arm

Eligibility Criteria

• Age: 1 Year - 14 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Up to diagnostic criteria for relapsed or refractory neuroblastoma or high-risk patients，including:
• Relapsed neuroblastoma : Children diagnosed with neuroblastoma who after standard treatment and remission, present lesions again and cannot reach complete remission with surgery.
• Refractory neuroblastoma : ① Untreated patients that do not have to reach completes remission after 4 courses of chemotherapy in accordance with standard regimens nor reach complete remission with surgery. ② High-risk patients : Who have cell genetic variation, such as MYCN amplification or bone marrow metastasis.
• Relapsed or Refractory Neuroblastoma: Target, of which expression may be intervened , discovered with Immunohistochemistry can be selected (GD2 +) (more than 50% of tumor cells is at least 2+ , adopting anti-GD2-mAb14G2a ).
• Age: 1\~14 years old of age at the time of enrollment, male or female.
• Physical condition is good: ECOG score reaches 0 to 2 points.
• Body weights greater than or equal to 10 kg.
• White blood cell counts acuity≥ 1.0 x10\^9 / L.
• Estimated survival times \> 90 days.
• Voluntary participation, good compliance, can cooperate with the experimental observation and signed an informed consent form.

You may not qualify if:

• Positive pregnancy tests.
• Uncontrolled infection.
• HIV infection, hepatitis B or C activity period.
• Patients who need long-term immunosuppressive therapy (Such as allergies, autoimmune diseases, GVHD, etc.)
• Combined activity of the central nervous system malignant tumor invasion.
• Abnormal coagulation function, patients with severe thrombosis.
• Organ failure
• Heart：class Ⅱ or above.
• Liver：class Ⅱ or above( Refer to Classification of Wuhan Conference (1983)).
• Kidney: The second stage of renal insufficiency or above.
• Lung: class Ⅱdecreased slightly or above.
• Brain: The central nervous system transfer or have active lesions.
• Patients who have participated in other clinical trials or other clinical trials in the past 30 days.
• The researchers believe that the patient is not suitable to participate in the study.

Sponsors & Collaborators

• Sinobioway Cell Therapy Co., Ltd.: lead
• Nanjing Children's Hospital: collaborator
• Children's Hospital of Fudan University: collaborator

Study Sites (2)

Nanjing Children's Hospital

Nanjing, Jiangsu, 210008, China

RECRUITING

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

RECRUITING

Related Publications (1)

• Thomas S, Straathof K, Himoudi N, Anderson J, Pule M. An Optimized GD2-Targeting Retroviral Cassette for More Potent and Safer Cellular Therapy of Neuroblastoma and Other Cancers. PLoS One. 2016 Mar 31;11(3):e0152196. doi: 10.1371/journal.pone.0152196. eCollection 2016.

  PMID: 27030986 BACKGROUND

Related Links

• An Optimized GD2-Targeting Retroviral Cassette for More Potent and Safer Cellular Therapy of Neuroblastoma and Other Cancers

MeSH Terms

Conditions

Recurrence; Neuroblastoma

Condition Hierarchy (Ancestors)

Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Study Officials

• Yongjun Fang, Ph.D

  Nanjing Children's Hospital

  PRINCIPAL INVESTIGATOR

• Kuiran Dong, Ph.D

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yongjun Fang, Ph.D

CONTACT

18951769586; fyj322@189.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 26, 2016
• First Posted: September 29, 2016
• Study Start: September 1, 2016
• Primary Completion: September 1, 2020
• Study Completion: September 1, 2020
• Last Updated: March 14, 2017

Record last verified: 2017-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)