Allogeneic Hematopoietic Stem Cell Transplantation for 4/M Neuroblastoma

NCT05303727 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: 2021-KY-126
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 1

Brief Summary

Neuroblastoma (NB) is the most common extracranial solid tumor of embryonal origin in children. According to the International Neuroblastoma Risk Group (INRG) staging criteria and the International Neuroblastoma Staging System (INSS) ，NB preoperative staging is divided into L1, L2, M and Ms stages, the postoperative staging is divided into 1 to 4 stages and 4s stage. Among them, 4/M stage is of the highest degree of malignancy and the worst prognosis. Despite the aggressive combination therapy, the 5-year survival rate (OS) is still less than 15%, and the 2-year relapse rate is 80%. Currently, no effective treatment is accessible for refractory/relapsed stage 4/M NB after completing conventional therapy. In hematopoietic stem cell transplantation (HSCT) , conditioning regimen with high-dose radiotherapy and chemotherapy is implemented to eradicate tumor cells and abnormal clonal cells in the patient, block the pathogenesis, and restore the patient's hematopoietic and immune systems by transplanting normal hematopoietic stem cells. According to the source of hematopoietic stem cells, HSCT can be divided into two types: autologous hematopoietic stem cell transplantation (auto-HSCT) and allogeneic hematopoietic stem cell transplantation (allo-HSCT). It has been confirmed that benefiting from the graft versus tumor(GVT) effect, allo-HSCT can clear residual lesions in refractory/relapsed NB patients post-auto-HSCT，and prolong the survival time of patients. Our center has explored the conditioning regimen, treatment of residual tumor lesions before transplantation, and strategies to reduce transplantation-related death (TRM) and enhance the GVT effect. However, the sample size is small, and multicenter and larger sample size research are needed. This study will further observe the clinical efficacy and safety of allo-HSCT in the treatment of 4/M stage NB, and provide a new treatment method and option for 4/M stage NB.

Conditions

Neuroblastoma

Keywords

allogeneic stem cell transplantation

Outcome Measures

Primary Outcomes (2)

• overall survival(OS) at 3 year

  From the date of day 0 of transplantation until the date of death from any cause

  3 years post-HSCT

• event free survival(EFS) at 3 year

  Survival time from day 0 of transplantation to the occurrence of the first adverse event. Disease or treatment-related adverse events, such as tumor recurrence, implant failure, and death, are counted in this study; accidental deaths that assessed unrelated to the above factors are not included

  3 years post-HSCT

Secondary Outcomes (8)

• incidence of conditioning toxicity

  100 days post-HSCT

• incidence of donor engraftment

  100 days post-HSCT

• early transplant-related mortality

  100 days post-HSCT

• incidence of sinusoidal obstruction syndrome

  3 years post-HSCT

• incidence of transplant associated thrombotic microangiopathy(TA-TMA)

  3 years post-HSCT

Study Arms (1)

Conditioning regimen for different sources of donors

EXPERIMENTAL

There are 3 groups according to different sources of donor: (1) Cord blood HSCT: Flu+Bu+CTX+Topotecan (without ATG); (2) Peripheral blood HSCT or haploid bone marrow combined with peripheral stem cell transplantation: Flu+Bu+Melphalan+Antithymocyte globulin (ATG)+ Thiotepa (TT) or (3) Flu+Bu+Melphalan+ATG (applicable to peripheral stem cells or haploid bone marrow combined with peripheral stem cell transplantation for which TT cannot be used).

Drug: Anti Thymocyte Globulin; Drug: Fludarabine; Drug: Cyclophosphamide injection; Drug: Topotecan; Drug: Melphalan; Drug: Thiotepa; Drug: Busulfan; Drug: Cyclosporine; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Drug: Methotrexate

Interventions

Anti Thymocyte Globulin DRUG

2.5 mg/kg/day；2 doses on day -3 and day -2 for matched sibling donor transplantation；3 doses on day -4，-3 and day -2 for unrelated donor transplantation；4 doses on day -5，-4，-3 and day -2 for haploidentical donor transplantation

Also known as: ATG

Conditioning regimen for different sources of donors

Fludarabine DRUG

30 mg/m2/day for 5 days

Also known as: Fludara

Conditioning regimen for different sources of donors

Cyclophosphamide injection DRUG

60 mg/kg/day for 2 days in cord blood stem cell transplantation

Also known as: CTX

Conditioning regimen for different sources of donors

Topotecan DRUG

2mg/m2/day for 3 days in cord blood stem cell transplantation

Also known as: Topotecan Hydrochloride

Conditioning regimen for different sources of donors

Melphalan DRUG

70mg/m2/day，for peripheral stem cell transplantation or haploidentical bone marrow combined with peripheral stem cell transplantation；2 doses on day -3 and day -2 when conditioning regimen containing thiotepa；3 doses on day -4，-3 and day -2 when conditioning regimen not containing thiotepa；

Also known as: Alkeran

Conditioning regimen for different sources of donors

Thiotepa DRUG

5 mg/kg/day for 2 days in peripheral stem cell transplantation or haploidentical bone marrow combined with peripheral stem cell transplantation

Also known as: thiophosphoramide

Conditioning regimen for different sources of donors

Busulfan DRUG

0.8mg/kg/dose；8 doses in cord blood stem cell transplantation；12 doses in peripheral stem cell transplantation or haploidentical bone marrow combined with peripheral stem cell transplantation when conditioning regimen containing thiotepa；16 doses in peripheral stem cell transplantation or haploidentical bone marrow combined with peripheral stem cell transplantation when conditioning regimen not containing thiotepa；

Also known as: Busulfex

Conditioning regimen for different sources of donors

Cyclosporine DRUG

2.5\~4 mg/kg/dose every 12 hours orally；1.5\~2 mg /kg/dose every 12 hours intravenously; trough concentration maintained at 150\~250ng/ml

Also known as: Sandimmune

Conditioning regimen for different sources of donors

Tacrolimus DRUG

0.02\~0.03 mg/kg/day as continuous infusion or 12 hour divided doses

Also known as: Prograf

Conditioning regimen for different sources of donors

Mycophenolate Mofetil DRUG

15 mg/kg/dose every 12 hours

Also known as: Cellcept

Conditioning regimen for different sources of donors

Methotrexate DRUG

15 mg/m2/dose on d+1 and 10 mg/m2/dose on d+3，d+6 in peripheral stem cell transplantation

Also known as: amethopterin

Conditioning regimen for different sources of donors

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age≤18 years old;
• After at least 7 courses of induction chemotherapy (surgical resection of the primary tumor or metastatic disease has been completed during the period), evaluation of disease is CR, tumor markers (blood NSE and urine VMA) and minimal residual disease by flow cytometry of bone marrow and peripheral blood are negative; the primary tumor has completed radiotherapy before HSCT;
• For patients with PR or VGPR, tumor markers (blood NSE and urine VMA) and minimal residual disease by flow cytometry of bone marrow and peripheral blood are negative; the primary tumor and metastatic lesions have completed radiotherapy before HSCT;
• Relapsed patients achieve CR/VGPR/PR after re-induction or salvage chemotherapy, tumor markers (blood NSE and urine VMA) and minimal residual disease by flow cytometry of bone marrow and peripheral blood are negative; the primary tumor and metastatic lesions have completed radiotherapy before HSCT;
• Whole brain and whole spinal cord radiotherapy have completed before HSCT in patients with central invasion at onset;
• The blood routine has generally returned to normal and there is no dysfunction of major organs such as the heart, liver, lung, and kidney;
• The guardian/patient accept the treatment of this research, sign the informed consent, and complete the follow-up.

You may not qualify if:

• With severe cardiac insufficiency, cardiac ejection fraction (EF) is less than 50%; or severe cardiac disease, the patient can not tolerate the conditioning regimen according to the investigators' evaluation;
• With severe pulmonary insufficiency (severe obstructive and/or restrictive ventilation disorders), the patient can not tolerate the conditioning regimen according to the investigators' evaluation;
• With severe liver function impairment, ALT\>5 times upper limit of normal, or total bilirubin\>3 times upper limit of normal; the patient can not tolerate the conditioning regimen according to the investigators' evaluation;
• With severe renal insufficiency, creatinine\>2 times upper limit of normal; or corrected creatinine clearance rate Ccr\<50ml/min; the patient can not tolerate the conditioning regimen according to the investigators' evaluation;
• With severe active bleeding or severe active infection; the patient can not tolerate the conditioning regimen according to the investigators' evaluation;
• Allergic reactions or serious adverse reactions occurred in the previous use of conditioning regimen-related drugs, the patient can not tolerate the conditioning regimen according to the investigators' evaluation;
• The guardian/patient cannot understand or comply with the treatment plan;
• Other reasons for not being selected due to the investigator's evaluation.

Sponsors & Collaborators

• Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University: lead

Study Sites (1)

Sun Yat-sen Memorial Hospital, Sun Yat-sen University

Guangzhou, Guangdong, 510120, China

Location

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MeSH Terms

Conditions

Neuroblastoma

Interventions

Antilymphocyte Serum; fludarabine; fludarabine phosphate; Cyclophosphamide; Topotecan; Melphalan; Thiotepa; Busulfan; Cyclosporine; Tacrolimus; Mycophenolic Acid; Methotrexate

Condition Hierarchy (Ancestors)

Neuroectodermal Tumors, Primitive, Peripheral; Neuroectodermal Tumors, Primitive; Neoplasms, Neuroepithelial; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Glandular and Epithelial; Neoplasms, Nerve Tissue

Intervention Hierarchy (Ancestors)

Immune Sera; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Biological Products; Complex Mixtures; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Camptothecin; Alkaloids; Heterocyclic Compounds; Phenylalanine; Amino Acids, Aromatic; Amino Acids, Cyclic; Amino Acids; Triethylenephosphoramide; Aziridines; Azirines; Heterocyclic Compounds, 1-Ring; Butylene Glycols; Glycols; Alcohols; Mesylates; Alkanesulfonates; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Sulfonic Acids; Sulfur Acids; Sulfur Compounds; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Macrolides; Lactones; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Aminopterin; Pterins; Pteridines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Officials

• Yang Li

  Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

  STUDY DIRECTOR

• Jianpei Fang

  Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

  STUDY DIRECTOR

Central Study Contacts

Ke Huang, MD

CONTACT

+8602034070821; hke@mail.sysu.edu.cn

Su Liu, MD

CONTACT

+8613512742517; liusu2009@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: There are 3 groups according to different sources of donor: (1) Cord blood HSCT: Flu+Bu+CTX+Topotecan (without ATG); (2) Peripheral blood HSCT or haploid bone marrow combined with peripheral stem cell transplantation: Flu+Bu+Melphalan+Antithymocyte globulin (ATG)+ Thiotepa (TT) or (3) Flu+Bu+Melphalan+ATG (applicable to peripheral stem cells or haploid bone marrow combined with peripheral stem cell transplantation for which TT cannot be used).

Study Record Dates

• First Submitted: March 16, 2022
• First Posted: March 31, 2022
• Study Start: August 1, 2022
• Primary Completion (Estimated): August 1, 2027
• Study Completion (Estimated): August 1, 2027
• Last Updated: July 1, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)