Protocol summary

Study aim
evaluation of safety and efficacy evaluation post intravenous infusion of activated NK cells in recurrent and refractory of high-risk neuroblastoma patients:
Design
Phase I/IIa clinical trial, controlled, randomized, non-blinded
Settings and conduct
Among patients with recurrent neuroblastoma who have not responded to conventional treatments, 10 patients with confirmed tumor type based on pathology examination will be consecutively enrolled in the study and assigned to either the intervention or control group through blocked randomization.
Participants/Inclusion and exclusion criteria
High-risk neuroblastoma that is resistant to standard induction therapy based on COG criteria, Evidence of relapse or progression of neuroblastoma after autologous, A minimum life expectancy of 6 months. Patients must have a pathological diagnosis of neuroblastoma and/or confirmation of tumor cells in the bone marrow with increased urinary catecholamines. Measurable residual disease based on imaging findings using Curie scoring or MIBG or PET imaging criteria Patients with impaired function of the bone marrow, liver, kidney, respiratory system, and nervous system will be excluded.
Intervention groups
A specific number of cells for each patient, based on the patient's weight (5×10^6 cells/kg), are thawed and prepared in a volume of 1×10^6 cells/ml for each injection. The cells are injected intravenously at intervals between chemotherapy courses, specifically 10 days after each chemotherapy course. A total of three injections will be administered. Patients in the control group will not receive any cells and will only undergo standard treatment methods.
Main outcome variables
safety, Efficacy

General information

Reason for update
Acronym
IRCT registration information
IRCT registration number: IRCT20170122032121N8
Registration date: 2024-08-05, 1403/05/15
Registration timing: registered_while_recruiting

Last update: 2024-08-05, 1403/05/15
Update count: 0
Registration date
2024-08-05, 1403/05/15
Registrant information
Name
Marzieh Ebrahimi
Name of organization / entity
Royan Institute
Country
Iran (Islamic Republic of)
Phone
+98 23562516
Email address
mebrahimi@royaninstitute.org
Recruitment status
recruiting
Funding source
Expected recruitment start date
2024-08-05, 1403/05/15
Expected recruitment end date
2026-08-06, 1405/05/15
Actual recruitment start date
empty
Actual recruitment end date
empty
Trial completion date
empty
Scientific title
Safety and efficacy evaluation post intravenous infusion of activated NK cells in recurrent and refractory of high-risk neuroblastoma patients: a phase I/IIa clinical trial
Public title
Safety and efficacy evaluation of using natural killer cells to treat patients with recurrent and refractory high-risk neuroblastoma
Purpose
Treatment
Inclusion/Exclusion criteria
Inclusion criteria:
High-risk neuroblastoma that is resistant to standard induction therapy based on COG (Children’s Oncology Group) criteria (according to INRG criteria and having received at least 4 cycles of multi-drug induction chemotherapy, and not responding to conventional treatments). Evidence of relapse or progression of neuroblastoma after autologous peripheral blood stem cell transplantation or aggressive therapy. A minimum life expectancy of 6 months. Patients must have a pathological diagnosis of neuroblastoma and/or confirmation of tumor cells in the bone marrow with increased urinary catecholamines. Measurable residual disease based on imaging findings using Curie scoring or MIBG or PET imaging criteria (1: measurable tumor of at least 10 mm in one dimension on MRI or CT scan with positive uptake on I-123 MIBG scan (“MIBG avid”) or 2) increased FDG uptake on 18F-FDG PET-CT or PET-MRI (“PET avid”)).
Exclusion criteria:
Insufficient bone marrow function: Platelet count > 50,000/µL, independent of transfusion (no platelet transfusion within one week).Absolute neutrophil count (ANC) maximum of 500 per microliter.Hemoglobin > 10 grams per deciliter. Insufficient liver function: Plasma bilirubin level more than 1.5 times the upper limit of normal (ULN).SGPT (ALT) at least three times the upper limit of normal (a level of 45 units per liter is considered the upper limit of normal). Insufficient kidney function: Creatinine clearance or estimated radioisotope GFR < 70 ml/min/1.73m².Plasma creatinine level more than 1.5 times the upper limit of normal based on age/gender. Insufficient central nervous system functionIf seizures are present, entry into the study is not possible if seizures are not well controlled with anticonvulsant drugs.CNS toxicity > Grade 2. Insufficient cardiovascular functionShortening fraction < 27% by ECHO OREjection fraction < 50% by ECHO or gated radionuclide study. Insufficient pulmonary functionEvidence of dyspnea at rest.Exercise intolerance.Chronic need for oxygen and room air pulse oximetry < 94% if pulse oximetry evaluation is clinically indicated.Presence of current pleural or pericardial effusion. Inability to tolerate new treatment due to emergency conditions. Elevated catecholamines (more than twice the ULN) or sole involvement of bone marrow (bone marrow positive for NB as the only evaluable disease without confirmatory pathology report). Receiving 0.5 mg/kg/day of systemic steroids (equivalent to prednisone) for at least 7 days before enrollment. Receiving CYP3A4 inducers or inhibitors at least 7 days before study enrollment. Diagnosis of any other malignancy alongside the diagnosis of neuroblastoma. Diarrhea > Grade 2 (4 to 6 stools per day). Significant illness not covered by exclusion criteria but interfering with the study process or increasing the intensity of treatment with NK cells. Participation in another clinical trial. Severe impairment of major organ functions, such as renal, cardiac, hepatic, neurological, pulmonary, or gastrointestinal toxicity above Grade 2 according to the National Cancer Institute’s Common Terminology Criteria for Adverse Events version 5.0 (CTC v5.0). Inability to comply with protocol requirements. Lack of confirmed and signed consent by the patient's guardians. Evidence of HIV disease (Human Immunodeficiency Virus) or positive serology for HIV.
Age
From 2 years old to 16 years old
Gender
Both
Phase
1-2
Groups that have been masked
No information
Sample size
Target sample size: 10
Randomization (investigator's opinion)
Randomized
Randomization description
Among patients with recurrent neuroblastoma who have not responded to conventional treatments and are referred to the Children's Growth and Development Center or Hazrat Rasoul Akram Hospital, 10 patients with confirmed tumor type based on pathology examination will be consecutively enrolled in the study and assigned to either the intervention or control group through blocked randomization.
Blinding (investigator's opinion)
Not blinded
Blinding description
Placebo
Not used
Assignment
Parallel
Other design features

Secondary Ids

empty

Ethics committees

1

Ethics committee
Name of ethics committee
Royan institute ethics committee
Street address
Royan ethics committee, Royan alley, Hafez street, Banihashem square, Banihashem street, Resalat highway, Tehran, Iran
City
Tehran
Province
Tehran
Postal code
1665659911
Approval date
2024-06-11, 1403/03/22
Ethics committee reference number
IR.ACECR.ROYAN.REC.1400.032

Health conditions studied

1

Description of health condition studied
recurrent and refractory of high-risk neuroblastoma
ICD-10 code
C74.90
ICD-10 code description
adrenal gland, Cancer, neuroblastoma

Primary outcomes

1

Description
Safety
Timepoint
After each injection to next injection and 2 weeks after last injection
Method of measurement
Using CTCAEs (Common Terminology Criteria for Adverse Events) checklist

Secondary outcomes

1

Description
Response rate comparison between control and intervention groups
Timepoint
6 and 12 months after the last injection
Method of measurement
MRI and MIBG or PET scan

2

Description
Overall survival and progression-free survival
Timepoint
up to 12 months after the last injection
Method of measurement
using Kaplan-Meier method

3

Description
alteration in bone marrow sample
Timepoint
12 months after the last injection
Method of measurement
Bone marrow aspiration and H&E staining

Intervention groups

1

Description
A specific number of cells for each patient, based on the patient's weight (5×10^6 cells/kg), are thawed and prepared in a volume of 1×10^6 cells/ml for each injection. For patients whose treatment starts with chemotherapy, the cells are injected intravenously at intervals between chemotherapy courses, specifically 10 days after each chemotherapy course. A total of three injections will be administered.
Category
Treatment - Other

2

Description
Control Group: Patients in the control group will not receive any cells and will only undergo standard treatment methods (radiotherapy, chemotherapy, or surgery) as determined by their treating physician. In this study, no placebo injections will be administered to the control group. However, if results six months after the last patient's injection indicate a significant efficacy of NK cells, control group patients may opt to receive the NK cell therapy protocol free of charge. Follow-ups for the control group will continue for one year after entering the study to compare with the intervention group.
Category
Other

Recruitment centers

1

Recruitment center
Name of recruitment center
Rasoul Akram hospital
Full name of responsible person
Dr. Mohammad Faranoush
Street address
Pediatric oncology ward, floor 4, Rasoul Akram hospital, Niayesh street, Shahr Ara street, Sattar Khan vicinity, Tehran, Iran
City
tehran
Province
Tehran
Postal code
1445613131
Phone
+98 21 6652 5328
Email
faranoush47@gmail.com

Sponsors / Funding sources

1

Sponsor
Name of organization / entity
Kian immune cell company
Full name of responsible person
Hassan Salehi Moghadam
Street address
Kian immune cell company, N0. 8, , west shaghayegh alley, Banihashem square, Banihashem street, Resalat highway, Tehran, Iran
City
tehran
Province
Tehran
Postal code
1665613179
Phone
+98 21 2614 1850
Email
kiacell.immune@gmail.com
Web page address
https://kiacell-immune.com
Grant name
Grant code / Reference number
Is the source of funding the same sponsor organization/entity?
Yes
Title of funding source
Kian immune cell company
Proportion provided by this source
100
Public or private sector
Private
Domestic or foreign origin
Domestic
Category of foreign source of funding
empty
Country of origin
Type of organization providing the funding
Industry

Person responsible for general inquiries

Contact
Name of organization / entity
Kian immune cell
Full name of responsible person
Mobina Ghasemi
Position
Research Assistant
Latest degree
Medical doctor
Other areas of specialty/work
Immunology
Street address
No 8. West Shaghayegh Alley, Bani Hashem square, Tehran
City
Tehran
Province
Tehran
Postal code
1665613179
Phone
+98 21 2614 1850
Email
ghasemi.mobina@gmail.com

Person responsible for scientific inquiries

Contact
Name of organization / entity
Kian immune cell
Full name of responsible person
Mobina Ghasemi
Position
Research Assistant
Latest degree
Medical doctor
Other areas of specialty/work
Immunology
Street address
No 8. West Shaghayegh Alley, Bani Hashem square, Tehran
City
Tehran
Province
Tehran
Postal code
1665613179
Phone
+98 21 2614 1850
Email
ghasemi.mobina@gmail.com

Person responsible for updating data

Contact
Name of organization / entity
Kian immune cell
Full name of responsible person
Mobina Ghasemi
Position
Research Assistant
Latest degree
Medical doctor
Other areas of specialty/work
Immunology
Street address
No 8. West Shaghayegh Alley, Bani Hashem square, Tehran
City
Tehran
Province
Tehran
Postal code
1665613179
Phone
+98 21 2614 1850
Email
ghasemi.mobina@gmail.com

Sharing plan

Deidentified Individual Participant Data Set (IPD)
Yes - There is a plan to make this available
Study Protocol
Yes - There is a plan to make this available
Statistical Analysis Plan
Yes - There is a plan to make this available
Informed Consent Form
Yes - There is a plan to make this available
Clinical Study Report
Yes - There is a plan to make this available
Analytic Code
Undecided - It is not yet known if there will be a plan to make this available
Data Dictionary
Not applicable
Title and more details about the data/document
After concealment of personal information of participants, data of eligibility, CRFs and measured outcomes can be shared in case of request
When the data will become available and for how long
After publishing results in a scientific journal with no deadline
To whom data/document is available
Just to academic PIs
Under which criteria data/document could be used
Any usage of data should be consulted and approved by the PIs
From where data/document is obtainable
Applicants should send an e-mail to Dr. Ghasemi and propose their detailed requirements for the study data.
What processes are involved for a request to access data/document
After sending an e-mail to Dr.Ghasemi, she will assess the authenticity of the applicant in almost 2 weeks. Then he will plan a meeting (visual or attendance) for the applicant with Dr.Ebrahimi in next month. The applicant can obtain approval of data sharing in this meeting and the data will be sent in next 14 days
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