Relapsed or Refractory Neuroblastoma Clinical Trial
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
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.
| Status | Recruiting |
| Enrollment | 22 |
| Est. completion date | September 2020 |
| Est. primary completion date | September 2020 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 1 Year to 14 Years |
| Eligibility |
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 : ? 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. ? 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 ? or above. 2. Liver:class ? or above( Refer to Classification of Wuhan Conference (1983)). 3. Kidney: The second stage of renal insufficiency or above. 4. Lung: class ?decreased 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. |
| Country | Name | City | State |
|---|---|---|---|
| China | Nanjing Children's Hospital | Nanjing | Jiangsu |
| China | Children's Hospital of Fudan University | Shanghai | Shanghai |
| Lead Sponsor | Collaborator |
|---|---|
| Sinobioway Cell Therapy Co., Ltd. | Children's Hospital of Fudan University, Nanjing Children's Hospital |
China,
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. — View Citation
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | Progression free survival | From the test of the progression of disease progression or the interval between disease and death. | 3 years | |
| Secondary | Overall survival | For all patients, overall survival refers to the period from being included in the test group to death caused by any reason | 3 years | |
| Secondary | Patients-based Quality of Life Evaluation | 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. | 3 years | |
| Secondary | 3°or above incidence rate of serious adverse reaction related to treatment | Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 | 3 years |
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT02761915 -
A Phase I Trial of Anti-GD2 T-cells (1RG-CART)
|
Phase 1 |