NeuroblastomaEarly Phase 1NCT06684639
A Study on the Efficacy of GD2-CAR T Cells in the Treatment of Neuroblastoma
A Early Phase 1 study testing GD2-CAR-T cell in people with Neuroblastoma, run by The General Hospital of Western Theater Command at 1 site in China.
Trial summary
Neuroblastoma (NB) is a malignant tumor of the sympathetic nervous system.Chemotherapy and autologous hematopoietic stem cell transplantation are the main treatments for neuroblastoma, and the prognosis of patients with high-risk recurrence and refractory treatment is very poor. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Neuroblastoma
- What is being tested?
- GD2-CAR-T cell
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Dec 2024to 1 Dec 2030
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- All cases were diagnosed as neuroblastoma with positive expression of GD2 antigen in tumor cells. Informed consent of patient or guardian.
- Diagnosis of recurrent/refractory neuroblastoma.
- At least 2 weeks or 5 half-lives (whichever is shorter) from the beginning of preconditioning chemotherapy after prior systemic treatment.
- Toxic reactions caused by previous antitumor therapy must be stabilized and restored to ≤ grade 1.
- Over 1 years old, under 18 years old.
- Physical strength score 0-3 (ECOG standard).
Show all 10Show fewer
- No obvious active infection.
- Expected survival ≥3 months
- Adequate kidney, liver, lung and heart function, defined as creatinine clearance (estimated by the Cockcroft Gault formula) > 60 mL/min; Serum ALT/AST ≤ 2.5 ULN; Total bilirubin ≤1.5 ULN, excluding subjects with Gilbert's syndrome; Cardiac ejection fraction ≥ 50%, echocardiography confirmed centropericardial effusion, and ECG showed no clinically significant abnormal findings. There was no clinically significant pleural effusion. Baseline blood oxygen saturation under indoor ventilation was > 92%.
- The serum pregnancy test results of fertile women must be negative (women who have undergone surgical sterilization or at least 2 years after menopause are considered to be infertile).
Who cannot join
- The subject has had other malignancies, non-melanoma skin tumors, carcinoma in situ (e.g. Cervix, bladder, breast), unless disease-free survival of at least 3 years.
- There is an uncontrollable infection, including fungal, bacterial, viral or other.
- Known human immunodeficiency virus (HIV) infection.
- Known history of hepatitis B (HBsAg positive) or hepatitis C (HCV antibody positive). Subjects with latent or prehepatitis B infection (defined as HBcAb positive and HBsAg negative) can be enrolled only if PCR tests for HBV DNA are negative. In addition, these subjects were required to undergo a monthly PCR test for HBV DNA. Participants who are serologically positive for HCV antibodies can also be enrolled if their PCR test results for HCV RNA are negative.
- Existing or past CNS disease, such as seizures, cerebrovascular ischemia/bleeding, dementia, cerebellar disease, or any CNS-related autoimmune disease.
- Serious heart disease, such as uncontrolled or symptomatic arrhythmia, congestive heart failure, or myocardial infarction within 6 months prior to screening, or any grade 3 (moderate) or 4 (severe) heart disease (according to the New York Heart Society Functional Grading Method NYHA).
Show all 13Show fewer
- A history of myocardial infarction, angioplasty or stent placement, unstable angina pectoris, or other clinically significant heart disease in the 12 months prior to enrollment.
- Any medical condition that may affect the evaluation of safety or efficacy.
- Have had severe rapid hypersensitivity reactions to any of the drugs to be used in this study.
- Live vaccine should be administered within ≤6 weeks before starting the pretreatment regimen.
- Pregnant or lactating female subjects.
- Male or female subjects who do not consent to effective contraception from the time they sign informed consent until 6 months after completing immune cell therapy.
- Subjects judged by the investigator had difficulty in completing all visits or procedures required by the study protocol (including follow-up visits), or were not compliant enough to participate in the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The General Hospital of Western Theater Command
Chengdu, Sichuan
hai Yi, 0086-28-86571279, yihaimail@163.com
About this study
Official title: An Open Clinical Study on the Safety and Efficacy of Autologous GD2-CAR T Cells in the Treatment of Relapsed Refractory Neuroblastoma
Neuroblastoma (NB) is a malignant tumor of the sympathetic nervous system.Chemotherapy and autologous hematopoietic stem cell transplantation are the main treatments for neuroblastoma, and the prognosis of patients with high-risk recurrence and refractory treatment is very poor. There is a large unmet medical need in patients with relapsed refractory neuroblastoma, and further research into new therapeutic approaches is needed for these patients.GD2 is a dissialic ganglioside expressed by neuroectodermal tumors. The proportion of GD2 expression in neuroblastoma is up to 100%, so GD2 is a specific target for neuroblastoma immunotherapy and an ideal target for CAR-T treatment of neuroblastoma.
Read the detailed description
The subjects received the target dose of GD2-CAR-T from 1×106 to 1×108 /kg. Each subject will start with a low dose of 1×106/kg and if there are no significant side effects will be increased to the next dose until the maximum tolerated dose is reached. A variety of adverse events (including neurological events, hematological events, infections, and secondary tumors) will be collected from the time of infusion of CAR T cells to 24 months after infusion. To understand the complete response rate (CR) and partial response rate (PR) at 3 months; Recurrence rate, progression-free survival (PFS) and overall survival (OS) after 1 to 5 years of GD2-CAR-T reinfusion; The amount and duration of GD2-CAR-T in vivo.
- Conditions
- Neuroblastoma
- Being tested
- GD2-CAR-T cell
- Keywords
- Neuroblastoma, Relapsed, Refractory
- Medical subject headings
- Neuroblastoma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GD2-CAR-T cellBiological
Split intravenous infusion of GD2-CAR-T cells [dose escalating infusion of (1-100)x10^6 GD2-CAR-T cells/kg]
| Study group | Role | What they receive |
|---|---|---|
| Experimental group | Experimental | GD2 positive relapsed or refractory neuroblastoma Receives: Biological: GD2-CAR-T cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse Events
Adverse events are evaluated with CTCAE V4.03
Measured: 24 months
Overall response rate (ORR)
ORR includes CR, PR,MR.SD,PD.Complete response (CR)#All components CR.Partial response (PR)#PR in at least one component and all other components CR,minimal disease (bone marrow), PR or not involved at baseline.Minor response (MR)#PR or CR in at least one component but at least one other component with SD; no component with PD.Stable disease (SD)#SD in one component with no better than SD or not involved at baseline in any other component, no component with PD.Progressive disease (PD)#Any component with PD.
Measured: 24 months
Secondary outcomes (3)
Duration of overall response (DOR)
Duration of overall response will be assessed from the CAR-T cell infusion to progression, death or last follow-up.
Measured: 24 months
Progression-free survival(PFS)
PFS will be assessed from the CAR-T cell infusion to progression, death or last follow-up.
Measured: 24 months
Overall survival(OS)
OS will be assessed from the CAR-T cell infusion to death or last follow-up.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Nov 2024
- Study start1 Dec 2024
- Primary completion1 Dec 2029 (estimated)
- Study completion1 Dec 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- The General Hospital of Western Theater Command
- Sponsor type
- University, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Responsible party
- Sponsor
- Investigators
- Hai Yi, Ph.D, Principal investigator, The General Hospital of Western Theater Command
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06684639
- Sponsor's protocol ID
- Yihai2024
- Status last verified
- Oct 2024
- First submitted
- 28 Aug 2024
- Last updated
- 20 Dec 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Dec 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Clinical Study on the Safety and Efficacy of Autologous GD2-CAR T Cells in the Treatment of Relapsed Refractory Neuroblastoma
- ClinicalTrials.gov ID
- NCT06684639
- Acronym / sponsor ID
- Yihai2024
- Conditions
- Neuroblastoma
- Keywords
- Neuroblastoma, Relapsed, Refractory
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- The General Hospital of Western Theater CommandUniversity, hospital or other
- Collaborators
- Yake Biotechnology Ltd.
- Study start
- 1 Dec 2024 (actual)
- Primary completion
- 1 Dec 2029 (estimated)
- Study completion
- 1 Dec 2030 (estimated)
- First posted
- 12 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Neuroblastoma (NB) is a malignant tumor of the sympathetic nervous system.Chemotherapy and autologous hematopoietic stem cell transplantation are the main treatments for neuroblastoma, and the prognosis of patients with high-risk recurrence and refractory treatment is very poor. There is a large unmet medical need in patients with relapsed refractory neuroblastoma, and further research into new therapeutic approaches is needed for these patients.GD2 is a dissialic ganglioside expressed by neuroectodermal tumors. The proportion of GD2 expression in neuroblastoma is up to 100%, so GD2 is a specific target for neuroblastoma immunotherapy and an ideal target for CAR-T treatment of neuroblastoma.
Detailed description
The subjects received the target dose of GD2-CAR-T from 1×106 to 1×108 /kg. Each subject will start with a low dose of 1×106/kg and if there are no significant side effects will be increased to the next dose until the maximum tolerated dose is reached. A variety of adverse events (including neurological events, hematological events, infections, and secondary tumors) will be collected from the time of infusion of CAR T cells to 24 months after infusion. To understand the complete response rate (CR) and partial response rate (PR) at 3 months; Recurrence rate, progression-free survival (PFS) and overall survival (OS) after 1 to 5 years of GD2-CAR-T reinfusion; The amount and duration of GD2-CAR-T in vivo.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental group | Experimental | Biological: GD2-CAR-T cellGD2 positive relapsed or refractory neuroblastoma |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- All cases were diagnosed as neuroblastoma with positive expression of GD2 antigen in tumor cells. Informed consent of patient or guardian.
- Diagnosis of recurrent/refractory neuroblastoma.
- At least 2 weeks or 5 half-lives (whichever is shorter) from the beginning of preconditioning chemotherapy after prior systemic treatment.
- Toxic reactions caused by previous antitumor therapy must be stabilized and restored to ≤ grade 1.
- Over 1 years old, under 18 years old.
Key exclusion criteria (13 listed)
- The subject has had other malignancies, non-melanoma skin tumors, carcinoma in situ (e.g. Cervix, bladder, breast), unless disease-free survival of at least 3 years.
- There is an uncontrollable infection, including fungal, bacterial, viral or other.
- Known human immunodeficiency virus (HIV) infection.
- Known history of hepatitis B (HBsAg positive) or hepatitis C (HCV antibody positive). Subjects with latent or prehepatitis B infection (defined as HBcAb positive and HBsAg negative) can be enrolled only if PCR tests for HBV DNA are negative. In addition, these subjects were required to undergo a monthly PCR test for HBV DNA. Participants who are serologically positive for HCV antibodies can also be enrolled if their PCR test results for HCV RNA are negative.
- Existing or past CNS disease, such as seizures, cerebrovascular ischemia/bleeding, dementia, cerebellar disease, or any CNS-related autoimmune disease.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| GD2-CAR-T cellSplit intravenous infusion of GD2-CAR-T cells [dose escalating infusion of (1-100)x10^6 GD2-CAR-T cells/kg] | Biological | Experimental group |
Primary outcome
Number of Adverse Events
Time frame: 24 months
Adverse events are evaluated with CTCAE V4.03
Overall response rate (ORR)
Time frame: 24 months
ORR includes CR, PR,MR.SD,PD.Complete response (CR)#All components CR.Partial response (PR)#PR in at least one component and all other components CR,minimal disease (bone marrow), PR or not involved at baseline.Minor response (MR)#PR or CR in at least one component but at least one other component with SD; no component with PD.Stable disease (SD)#SD in one component with no better than SD or not involved at baseline in any other component, no component with PD.Progressive disease (PD)#Any component with PD.
3 secondary outcomes
Duration of overall response (DOR)
Time frame: 24 months
Progression-free survival(PFS)
Time frame: 24 months
Overall survival(OS)
Time frame: 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Dec 2024.
- Individual participant dataSharing planned: No
Related trials for Neuroblastoma
RecruitingPhase 1/2
Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse
Recurrent Ganglioneuroblastoma, Recurrent Neuroblastoma
Active, not recruitingObservational study
Genomic Structural Variation in Cancer Susceptibility
Breast Cancer, Colon Cancer
RecruitingPhase 2
Enasidenib in IDH2-Mutated Malignant Sinonasal and Skull Base Tumors
Metastatic Chondrosarcoma, Locally Advanced Chondrosarcoma
RecruitingObservational study
Prospective Comprehensive Molecular Analysis of Endocrine Neoplasms
Endocrine Tumors, Thyroid Neoplasms
RecruitingPhase 1
PEACH TRIAL- Precision Medicine and Adoptive Cellular Therapy
Neuroblastoma, Diffuse Intrinsic Pontine Glioma
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Hai Yi, Ph.D principal investigator
The General Hospital of Western Theater Command
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing GD2-CAR-T cell in people with Neuroblastoma, run by The General Hospital of Western Theater Command at 1 site 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 13 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: looking for participants now, as of the sponsor's last update on 20 Dec 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact JunXia Wang, Ph.D (0086-28-86571605, lzwjxyy@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Adverse Events (time frame: 24 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 20 Dec 2024.