Actively Recruiting
A Study to Evaluate the Safety, Preliminary Efficacy, and Pharmacokinetics of CNK-UT Cells in Children with Relapsed or Refractory Neuroblastoma
Led by Nanjing Children's Hospital · Updated on 2025-02-07
12
Participants Needed
2
Research Sites
21 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, preliminary effectiveness, and how the body processes universal T-cells engineered with a chimeric natural killer receptor (CNK-UT) in children aged 1 to 12 years with relapsed or refractory Neuroblastoma. This phase I, single-arm, open-label pilot study aims to understand the potential of CNK-UT cell therapy in treating this challenging condition where cancer has returned or not responded to standard treatments. Participants will receive CNK-UT cells through intravenous infusion. The study begins with a dose escalation phase where a single dose of CNK-UT cells ranging from 3 to 34 million CNK+ cells per kilogram of body weight is given to determine the highest safe dose. If no serious side effects occur, participants enter the dose expansion phase, where multiple doses are administered based on prior results. The dosing schedule and frequency in this phase are adjusted according to findings from the initial stage. Throughout the study, children will be monitored for treatment-related side effects for up to one year. Researchers will assess tumor responses using imaging and bone marrow tests, measure disease control, progression-free survival, and overall survival. They will also study how the CNK-UT cells behave in the body over time. Participants will undergo laboratory tests and functional assessments to ensure organ function and safety during the trial, which is expected to last up to a year of follow-up.
CONDITIONS
Brief Title
Chimeric Natural Killer Receptor-Universal T Cells for Relapsed or Refractory Neuroblastoma
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 1 and 12 years with weight at least 10 kg
- Male or female
- Informed consent signed by child and/or guardian with ability to comply with study
- Diagnosed with relapsed or refractory neuroblastoma as per NCCN guidelines
- Disease status is partial remission or stable disease before enrollment
- At least one lesion assessable by functional imaging or bone marrow exam; soft tissue lesions must be ≤ 2 cm in longest diameter
- Tumor tissue confirmed to express B7-H3 by immunohistochemistry
- Lansky score above 60
- Estimated life expectancy greater than 12 weeks
- Adequate organ and bone marrow function within 7 days before enrollment
- Previous treatment toxicities have resolved to grade 1 or less except hair loss
You will not qualify if you...
- Malignant tumors diagnosed within 5 years before enrollment except certain skin, thyroid, and breast cancers
- Symptomatic central nervous system metastasis confirmed by imaging or pathology
- MIBG non-avid disease
- History of organ transplantation excluding stem cell transplantation
- Active autoimmune disease requiring systemic treatment or primary immunodeficiency
- Uncontrolled systemic diseases or metabolic disorders increasing risk
- Active pulmonary tuberculosis under treatment or within past year, HIV infection, or syphilis
- Severe infections within 4 weeks prior to enrollment
- Recent radiotherapy, chemotherapy (excluding lymphodepletion), targeted therapy, or immunotherapy within 4 weeks
- Major surgery within 4 weeks prior to study or severe unhealed wounds
- Participation in other clinical trials within 4 weeks prior to study
- Use of attenuated live vaccines within 4 weeks prior to study
- Prior gene therapy
- Allergy to CNK-UT injection components
- Serious mental or substance abuse disorders impacting compliance
- Other serious complications or investigator-defined unsuitability
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single ascending dose with 21-day observation; multiple doses duration varies based on dose expansion stage
Participants receive intravenous infusions of CNK-UT cells. This includes a dose escalation phase with a single dose followed by a dose expansion phase with multiple doses as determined by the study.
Multiple visits for infusions and monitoring during dose escalation and dose expansion stages
Duration - Up to 1 year
Participants are monitored for safety, efficacy, and pharmacokinetics outcomes for up to 1 year after treatment.
Regular visits for safety and efficacy assessments over 1 year
Trial Site Locations
Total: 2 locations
1
Nanjing Children's Hospital
Nanjing, Jiangsu, China, 210000
Actively Recruiting
2
Nanjing
Nanjing, Jiangsu, China
Actively Recruiting
Research Team
F
Fang Yongjun, Prof.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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