Actively Recruiting

Phase 1
Age: 2Years - 16Years
All Genders
ID06674265

Evaluation of Safety and Efficacy of Intravenous Activated NK Cells in Children with Refractory and Recurrent High-risk Neuroblastoma

Led by Marzieh Ebrahimi · Updated on 2025-04-09

10

Participants Needed

1

Research Sites

N/A

Total Duration

On this page

Sponsors

M

Marzieh Ebrahimi

Lead Sponsor

R

Royan Institute

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the safety and effectiveness of systemic injections of allogenic natural killer (NK) cells in children with refractory or recurrent high-risk neuroblastoma. This study compares the group receiving NK cell infusions with a control group undergoing conventional treatment to determine if the new intervention is safe and effective for this challenging condition. The trial is in phase 1 and focuses on patients aged 2 to 16 years who have not responded to standard therapies. The NK cells used in this study are collected from healthy donors through a process called apheresis and purified using a specialized device in a clean room. After quality checks, the cells are stored at very low temperatures until needed. Participants receive three injections of these NK cells, dosed at 5 million cells per kilogram of body weight, administered intravenously 7 to 10 days after each chemotherapy course. Depending on how the patient responds and recovers, two additional injections may be given if improvement is confirmed by MRI MIBG scans. During the study, children will be monitored for safety after each NK cell infusion and compared to those receiving conventional treatments. Researchers will assess response rates at 6 and 12 months after treatment. Evaluations include imaging studies such as MRI and PET scans, laboratory tests, and clinical assessments. Participation involves multiple injections over chemotherapy intervals, with follow-up to track safety and treatment effects. The trial is expected to continue until November 2026.

CONDITIONS

Brief Title

Safety and Efficacy of Systemic Allogenic NK Cells in R/R Neuroblastoma

Who Can Participate

Age: 2Years - 16Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Children aged 2 to 16 years
  • Diagnosed with high-risk neuroblastoma resistant to standard induction therapy (at least 4 cycles of chemotherapy)
  • Evidence of relapse or progression after autologous stem cell transplantation or aggressive therapy
  • Minimum life expectancy of 6 months
  • Pathological diagnosis of neuroblastoma or tumor cells confirmed in bone marrow with increased urinary catecholamines
  • Measurable residual disease by imaging criteria (MRI, CT, MIBG, or PET)
Not Eligible

You will not qualify if you...

  • Platelet count below 50,000/µL or recent transfusion
  • Absolute neutrophil count below 500/µL
  • Hemoglobin less than 10 g/dL
  • Liver function: bilirubin above 1.5 times normal or ALT three times above normal
  • Kidney function: creatinine clearance below 70 ml/min/1.73 m² or elevated creatinine
  • Uncontrolled seizures or insufficient central nervous system function
  • Cardiac function: shortening fraction below 27% or ejection fraction below 50%
  • Pulmonary issues such as dyspnea at rest, oxygen requirement, or pleural/pericardial effusion
  • Unable to tolerate new treatments due to emergency conditions
  • Elevated catecholamines more than twice the upper limit of normal or bone marrow-only disease without pathology confirmation
  • Use of systemic steroids at 0.5 mg/kg/day or higher for at least 7 days before enrollment
  • Use of CYP3A4 inducers or inhibitors within 7 days before enrollment
  • Diagnosis of other cancers alongside neuroblastoma
  • Diarrhea greater than Grade 2
  • Significant illness interfering with study or increasing treatment intensity
  • Participation in another clinical trial
  • Severe organ toxicity above Grade 2
  • Inability to comply with protocol
  • Lack of signed consent by guardians
  • Positive HIV diagnosis or serology

AI-Screening

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Variable based on chemotherapy schedule and patient response

Participants receive 3 to 5 systemic injections of allogeneic NK cells administered intravenously at a dose of 5×10^6 cells per kilogram of body weight. Injections occur 7 to 10 days after each chemotherapy course, with intervals varying based on the standard chemotherapy schedule. Additional injections may be given depending on patient response confirmed by imaging.

3 to 5 injection visits (in-person), scheduled after chemotherapy courses

Follow-up

Duration - Up to 2 weeks after last injection

Participants are monitored for safety after each injection until the next injection and for 2 weeks following the last injection to assess for adverse events and treatment response.

Visits corresponding with safety assessments after injections

Trial Site Locations

Total: 1 location

1

Rasoul Akram Hospital

Tehran, Iran, 1445613131

Actively Recruiting

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Research Team

D

Dr. Marzieh Ebrahimi

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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