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

NCT02919046

Description:

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.

Related Conditions:
  • Neuroblastoma
Recruiting Status:

Recruiting

Phase:

N/A

Trial Eligibility

Document

Title

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

Clinical Trial IDs

  • ORG STUDY ID: WM-CART-07
  • NCT ID: NCT02919046

Conditions

  • Relapsed or Refractory Neuroblastoma

Interventions

DrugSynonymsArms
GD2-targeted CAR-T cellssingle arm

Purpose

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.

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.
    

Trial Arms

NameTypeDescriptionInterventions
single armExperimentalName: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
  • GD2-targeted CAR-T cells

Eligibility 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.
      
Maximum Eligible Age:14 Years
Minimum Eligible Age:1 Year
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:The overall efficiency of patients with neuroblastoma after autologous CAR-T cell therapy
Time Frame:28d,56d,90d
Safety Issue:
Description: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

Measure:Progression free survival
Time Frame:3 years
Safety Issue:
Description:From the test of the progression of disease progression or the interval between disease and death.
Measure:Overall survival
Time Frame:3 years
Safety Issue:
Description:For all patients, overall survival refers to the period from being included in the test group to death caused by any reason
Measure:Patients-based Quality of Life Evaluation
Time Frame:3 years
Safety Issue:
Description: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.
Measure:3°or above incidence rate of serious adverse reaction related to treatment
Time Frame:3 years
Safety Issue:
Description:Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

Details

Phase:N/A
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Sinobioway Cell Therapy Co., Ltd.

Trial Keywords

  • neuroblastoma
  • CAR-T

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