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Relapsed or Refractory NeuroblastomaNCT02919046

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

A study testing GD2-targeted CAR-T cells in people with Relapsed or Refractory Neuroblastoma, run by Sinobioway Cell Therapy Co., Ltd. at 2 sites in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 1 year to 14 years
Conditions the study is for
Relapsed or Refractory Neuroblastoma
What is being tested?
GD2-targeted CAR-T cells
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
2 sitesChina
Study dates
Sep 2016to Sep 2020
Phase
Interventional
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 14 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • 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.
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  • 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.

Trial locations and contacts

2 sites in 1 country.

Yongjun Fang, Ph.D contact

18951769586

fyj322@189.cn

China2 sites, 2 recruiting

  • Nanjing Children's Hospital

    Nanjing, Jiangsu, 210008

    Yongjun Fang, Ph.D, 18951769586, fyj322@189.cn

    Yongjun Fang, Ph.D

    Recruiting

  • Children's Hospital of Fudan University

    Shanghai, Shanghai Municipality, 201102

    Kuiran Dong, Ph.D, 18017591156, kuirand@hotmail.com

    Recruiting

Other trials in China

About this study

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.

Read the 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.

Being tested
GD2-targeted CAR-T cells
Keywords
neuroblastoma, CAR-T
Medical subject headings
Recurrence, Neuroblastoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • GD2-targeted CAR-T cellsBiological

    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.

Study groups in NCT02919046
Study groupRoleWhat they receive
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
Receives: Biological: GD2-targeted CAR-T cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: 28d,56d,90d

Secondary outcomes (4)

  1. Progression free survival

    From the test of the progression of disease progression or the interval between disease and death.

    Measured: 3 years

  2. Overall survival

    For all patients, overall survival refers to the period from being included in the test group to death caused by any reason

    Measured: 3 years

  3. 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.

    Measured: 3 years

  4. 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

    Measured: 3 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Sep 2016 (estimated)
  2. Study startSep 2016
  3. Primary completionSep 2020 (estimated)
  4. Study completionSep 2020 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase N/A
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
22 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02919046
Sponsor's protocol ID
WM-CART-07
Status last verified
Mar 2017
First submitted
26 Sep 2016
Last updated
14 Mar 2017
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Publications

  • 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.

    PubMed 27030986

Questions about this trial

What is this clinical trial studying?

A study testing GD2-targeted CAR-T cells in people with Relapsed or Refractory Neuroblastoma, run by Sinobioway Cell Therapy Co., Ltd. at 2 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 14 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Yongjun Fang, Ph.D (18951769586, fyj322@189.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The overall efficiency of patients with neuroblastoma after autologous CAR-T cell therapy (time frame: 28d,56d,90d). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02919046, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Mar 2017.

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