Immunotherapy of Neuroblastoma Patients Using a Combination of Anti-GD2 and NK Cells

NCT ID: NCT03242603

Last Updated: 2018-03-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1/PHASE2

Total Enrollment

5 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-10-03

Study Completion Date

2020-08-15

Brief Summary

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Neuroblastoma is a neoplasm of the sympathetic nervous system which affects mostly children younger than 5 years of age. It is a heterogeneous disease, with nearly 50% of patients presenting with a high-risk phenotype. After standard treatment, the 2-year event-free survival (EFS) for high risk neuroblastoma (EFS) is only about 50%. Immunotherapy with anti-GD2 antibodies has been shown to improve EFS in Children's Oncology Group and SIOPEN trials.

The anti-GD2 antibody mediates neuroblastoma cell killing primarily through antibody-dependent cell-mediated cytotoxicity (ADCC). Natural killer (NK) cells are the main effectors of ADCC. We postulate that infusion of expanded activated NK cells from healthy haploidentical donors along with anti-GD2 antibody will enhance neuroblastoma killing.

Detailed Description

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Adoptive transfer of haploidentical NK cells has been shown to be safe in clinical trials at NUH. There is experience combining antibody infusion with autologous NK cells in the clinical trial with good safety data.

The proposed trial is a phase I/II study to determine the safety and efficacy of expanded activated haploidentical NK cells in combination with anti-GD2 (ch14.18/CHO). We plan to enrol patients with high risk or relapsed neuroblastoma with evidence of residual disease who are at high risk of recurrence or progression on current treatment.

In the proposed protocol , we plan to infuse NK cells at escalating dose levels to find the optimum dose tolerated by the patients in combination with anti-GD2 (ch14.18/CHO) . There are 3 NK cell dose levels :

Dose level 1 (1 x 10\^6/kg) , Dose level 2 (1 x 10\^7/kg) , Dose level 3 (1 x 10\^8/kg)

If a partial response or stable disease is observed, further infusions of NK cells can be administered. There will be intra- and inter - patient dose escalation.

The donor will be either parent, based on the best NK cell donor as determined by the study team. The donor will be harvested and NK cells expanded prior to infusion into the patient along with anti-GD2 (ch14.18/CHO).

The study aims to study safety and efficacy of a combination of NK cells and anti-GD2 (ch14.18/CHO).

Conditions

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Neuroblastoma Recurrent

Keywords

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Relapsed, Refractory, Neuroblastoma Anti-GD2 Natural Killer (NK) Cells Immunotherapy

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

To determine the feasibility, safety and effectiveness of anti-GD2 in combination with expanded, activated NK cells in research participants with Neuroblastoma
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Anti-GD2 in combination with NK cells

This is a single arm study. Patients with high risk neuroblastoma who have residual measurable disease will receive a combination of 5 days of anti-GD2 with expanded activated NK cells.

Group Type EXPERIMENTAL

Anti-GD2 in combination with NK cells

Intervention Type BIOLOGICAL

Haploidentical donor NK cells will be expanded over 10 days and infused in combination with anti-GD2. Anti-GD2 will be given as daily infusion for 5 days ; D-1, 0,+1, +2 and +3. NK cells will be infused at single dose on day 0. The patient will receive cyclophosphamide 60mg/kg on day -3 and day -2 prior to the NK cell infusion. IL- 2 will be given subcutaneously for 6 doses every alternate day starting on day -1, for NK cell survival.

Interventions

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Anti-GD2 in combination with NK cells

Haploidentical donor NK cells will be expanded over 10 days and infused in combination with anti-GD2. Anti-GD2 will be given as daily infusion for 5 days ; D-1, 0,+1, +2 and +3. NK cells will be infused at single dose on day 0. The patient will receive cyclophosphamide 60mg/kg on day -3 and day -2 prior to the NK cell infusion. IL- 2 will be given subcutaneously for 6 doses every alternate day starting on day -1, for NK cell survival.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

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1. Age 6 months to 25 years old.
2. Patients with high risk or relapsed neuroblastoma who have measurable residual disease (based on imaging findings with Curie scoring or MIBG or PET imaging criteria) after receiving or has refused to receive standard therapy.

1. High risk will be defined as stage IV disease with poor response to chemotherapy. Residual disease after surgery or prior to autologous stem cell rescue which is part of Standard of Care. Infants with nMYC amplification will not automatically qualify for the protocol unless they have residual disease after surgery.
2. Recurrence after completion of standard treatment.
3. Shortening fraction greater than or equal to 25% or Left ventricular ejection fraction (LVEF) greater than or equal to 40%.
4. Glomerular filtration rate greater than or equal to 60 ml/min/1.73 m2.
5. Pulse oximetry greater than or equal to 92% on room air.
6. Direct bilirubin less than or equal to 3.0 mg/dL (50 mmol/L).
7. Alanine aminotransferase (ALT) is no more than 2 times the upper limit of normal.
8. Aspartate transaminases (AST) is no more than 2 times the upper limit of normal.
9. Karnofsky or Lansky performance score of greater than or equal to 50.
10. Does not have a current pleural or pericardial effusion.
11. Has a suitable adult family member donor available for NK cell donation.
12. Has recovered from all acute NCI Common Terminology Criteria for Adverse Events (CTCAE) grade II-IV non-hematologic acute toxicities resulting from prior therapy per the judgment of the PI.
13. At least two weeks since receipt of any biological therapy, systemic chemotherapy, and/or radiation therapy.
14. Is not receiving more than the equivalent of prednisone 10 mg daily.
15. Not pregnant (negative serum or urine pregnancy test to be conducted within 7 days prior to enrollment).
16. Not lactating.


1. First and second degree relative acceptable.
2. 18 years of age or above.
3. Not lactating.
4. Greater than or equal to 3 of 6 HLA match to recipient.
5. .Meets eligibility and suitability criteria for hematopoietic cells donation as per institutional guidelines.
6. Not pregnant (negative serum or urine pregnancy test to be conducted within 7 days prior to enrollment).

Exclusion Criteria

* Failure to meet any of the above criteria
Minimum Eligible Age

6 Months

Maximum Eligible Age

25 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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National University Hospital, Singapore

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Miriam Kimpo, MD

Role: PRINCIPAL_INVESTIGATOR

National University Hospital, Singapore

Dario Campana, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

National University Singapore, Singapore

Locations

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National University Hospital

Singapore, , Singapore

Site Status RECRUITING

Countries

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Singapore

Central Contacts

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Miriam Kimpo, MD

Role: CONTACT

Phone: +65 8494 3914

Email: [email protected]

Chetan Dhamne, MBBS, MS, MD

Role: CONTACT

Phone: +65 6772 3361

Email: [email protected]

Facility Contacts

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Miriam Kimpo, MD

Role: primary

Chetan Dhamne, MBBS, MS, MD

Role: backup

References

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Yu AL, Gilman AL, Ozkaynak MF, London WB, Kreissman SG, Chen HX, Smith M, Anderson B, Villablanca JG, Matthay KK, Shimada H, Grupp SA, Seeger R, Reynolds CP, Buxton A, Reisfeld RA, Gillies SD, Cohn SL, Maris JM, Sondel PM; Children's Oncology Group. Anti-GD2 antibody with GM-CSF, interleukin-2, and isotretinoin for neuroblastoma. N Engl J Med. 2010 Sep 30;363(14):1324-34. doi: 10.1056/NEJMoa0911123.

Reference Type BACKGROUND
PMID: 20879881 (View on PubMed)

Cho D, Shook DR, Shimasaki N, Chang YH, Fujisaki H, Campana D. Cytotoxicity of activated natural killer cells against pediatric solid tumors. Clin Cancer Res. 2010 Aug 1;16(15):3901-9. doi: 10.1158/1078-0432.CCR-10-0735. Epub 2010 Jun 11.

Reference Type BACKGROUND
PMID: 20542985 (View on PubMed)

Tarek N, Le Luduec JB, Gallagher MM, Zheng J, Venstrom JM, Chamberlain E, Modak S, Heller G, Dupont B, Cheung NK, Hsu KC. Unlicensed NK cells target neuroblastoma following anti-GD2 antibody treatment. J Clin Invest. 2012 Sep;122(9):3260-70. doi: 10.1172/JCI62749. Epub 2012 Aug 6.

Reference Type BACKGROUND
PMID: 22863621 (View on PubMed)

Other Identifiers

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NKEXPGD2

Identifier Type: -

Identifier Source: org_study_id