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

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ClinicalTrials.gov Identifier: NCT02919046
Recruitment Status : Recruiting
First Posted : September 29, 2016
Last Update Posted : March 14, 2017
Sponsor:
Collaborators:
Nanjing Children's Hospital
Children's Hospital of Fudan University
Information provided by (Responsible Party):
Sinobioway Cell Therapy Co., Ltd.

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.

Condition or disease Intervention/treatment Phase
Relapsed or Refractory Neuroblastoma Biological: GD2-targeted CAR-T cells Not Applicable

Detailed Description:
This is a single-arm, multicenter clinical study to evaluate efficacy and safety of chimeric antigen receptor T cell immunotherapy (CAR-T) in the treatment of relapsed or refractory neuroblastoma in children. The study will be conducted using a phaseⅠ/Ⅱdesign the study will have the following sequential phases: part A (screening, leukapheresis,cell product preparation and cytoreductive chemotherapy) and part B (treatment and follow-up). the follow-up period for each participant is approximately 35 months after the final CAR-T infusion. The total duration of the study are expected to be approximately 3 years. A total of 22 patients may be enrolled over a period of 3 years.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 22 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Single Arm and Multicenter Clinical Trial to Evaluating the Efficacy and Safety of the Chimeric Antigen Receptor T Cell Immunotherapy (CAR-T) for Relapsed or Refractory Neuroblastoma in Children
Study Start Date : September 2016
Estimated Primary Completion Date : September 2020
Estimated Study Completion Date : September 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Neuroblastoma

Arm Intervention/treatment
Experimental: single arm
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
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.




Primary Outcome Measures :
  1. The overall efficiency of patients with neuroblastoma after autologous CAR-T cell therapy [ Time Frame: 28d,56d,90d ]
    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.


Secondary Outcome Measures :
  1. Progression free survival [ Time Frame: 3 years ]
    From the test of the progression of disease progression or the interval between disease and death.

  2. Overall survival [ Time Frame: 3 years ]
    For all patients, overall survival refers to the period from being included in the test group to death caused by any reason

  3. Patients-based Quality of Life Evaluation [ Time Frame: 3 years ]
    According to EORTC quality of life measurement scale PedsQL4.0_ children's quality of life of the core scale of the evaluation and comparison of physical condition before and after treatment.

  4. 3°or above incidence rate of serious adverse reaction related to treatment [ Time Frame: 3 years ]
    Number of participants with treatment-related adverse events as assessed by CTCAE v4.0



Information from the National Library of Medicine

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Ages Eligible for Study:   1 Year to 14 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. 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.
  2. 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 ).
  3. Age: 1~14 years old of age at the time of enrollment, male or female.
  4. Physical condition is good: ECOG score reaches 0 to 2 points.
  5. Body weights greater than or equal to 10 kg.
  6. White blood cell counts acuity≥ 1.0 x10^9 / L.
  7. Estimated survival times > 90 days.
  8. Voluntary participation, good compliance, can cooperate with the experimental observation and signed an informed consent form.

Exclusion Criteria:

  1. Positive pregnancy tests.
  2. Uncontrolled infection.
  3. HIV infection, hepatitis B or C activity period.
  4. Patients who need long-term immunosuppressive therapy (Such as allergies, autoimmune diseases, GVHD, etc.)
  5. Combined activity of the central nervous system malignant tumor invasion.
  6. Abnormal coagulation function, patients with severe thrombosis.
  7. Organ failure

    1. Heart:class Ⅱ or above.
    2. Liver:class Ⅱ or above( Refer to Classification of Wuhan Conference (1983)).
    3. Kidney: The second stage of renal insufficiency or above.
    4. Lung: class Ⅱdecreased slightly or above.
    5. Brain: The central nervous system transfer or have active lesions.
  8. Patients who have participated in other clinical trials or other clinical trials in the past 30 days.
  9. The researchers believe that the patient is not suitable to participate in the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02919046


Contacts
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Contact: Yongjun Fang, Ph.D 18951769586 fyj322@189.cn

Locations
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China, Jiangsu
Nanjing Children's Hospital Recruiting
Nanjing, Jiangsu, China, 210008
Contact: Yongjun Fang, Ph.D    18951769586    fyj322@189.cn   
Principal Investigator: Yongjun Fang, Ph.D         
China, Shanghai
Children's Hospital of Fudan University Recruiting
Shanghai, Shanghai, China, 201102
Contact: Kuiran Dong, Ph.D    18017591156    kuirand@hotmail.com   
Sponsors and Collaborators
Sinobioway Cell Therapy Co., Ltd.
Nanjing Children's Hospital
Children's Hospital of Fudan University
Investigators
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Principal Investigator: Yongjun Fang, Ph.D Nanjing Children's Hospital
Principal Investigator: Kuiran Dong, Ph.D Children's Hospital of Fudan University

Additional Information:
Publications:
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Responsible Party: Sinobioway Cell Therapy Co., Ltd.
ClinicalTrials.gov Identifier: NCT02919046     History of Changes
Other Study ID Numbers: WM-CART-07
First Posted: September 29, 2016    Key Record Dates
Last Update Posted: March 14, 2017
Last Verified: March 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Sinobioway Cell Therapy Co., Ltd.:
neuroblastoma
CAR-T
Additional relevant MeSH terms:
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Neuroblastoma
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