NCT07278674

Brief Summary

Evaluate the impact of maintenance therapy with eflornithine on event-free survival and overall survival in high-risk neuroblastoma (NB) children after immunotherapy, and assess its safety.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for all trials

Timeline
43mo left

Started Apr 2025

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress24%
Apr 2025Oct 2029

First Submitted

Initial submission to the registry

February 8, 2025

Completed
2 months until next milestone

Study Start

First participant enrolled

April 8, 2025

Completed
8 months until next milestone

First Posted

Study publicly available on registry

December 12, 2025

Completed
3.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 31, 2029

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 31, 2029

Last Updated

December 12, 2025

Status Verified

December 1, 2025

Enrollment Period

4.6 years

First QC Date

February 8, 2025

Last Update Submit

December 11, 2025

Conditions

High Risk Neuroblastoma

Keywords

High risk neuroblastomaEflornithine

Outcome Measures

Primary Outcomes (2)

  • The event free survival rate after the completion of the first DFMO

    The event free survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy.

    5 years

  • overall survival rate after the completion of the first DFMO

    overall survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy.

    5 years

Secondary Outcomes (1)

  • Incidence of Treatment-Emergent Adverse Events

    5 years

Interventions

Oral administration of eflornithine for the prevention of high-risk neuroblastomaDRUG

This study is a single arm clinical observational study and does not involve control interventions

Also known as: No other intervention measures

Eligibility Criteria

Age1 Month - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-Probability Sample
Study Population

Children with high-risk neuroblastoma

You may qualify if:

  • According to the International Neuroblastoma Risk Group Classification, it has been histologically diagnosed as high-risk NB.
  • Under the age of 18.
  • The disease assessment status is PR (via CT or MRI) and the bone marrow smear is negative.
  • If the residual mass is MIBG negative or MIBG positive and lacks FDG-PET affinity, it is considered evidence that the mass does not represent active disease. Subjects with stable residual tumor mass visible on CT/MRI will be included in the study.
  • Qualified hematological parameters and organ function; Refer to the CI CTC 4.0 adverse reaction grading standard of grade 2 and below.
  • Eflornithine needs to be activated within 120 days after the completion of previous treatment.

You may not qualify if:

  • According to the International Neuroblastoma Risk Group Classification, it has been histologically diagnosed as non high risk NB.
  • Prior to enrollment, the disease assessment status was either progressive disease (PD) or relapse (via CT or MRI).
  • Positive bone marrow smear.
  • Hematological parameters and organ function are not qualified, according to the CI CTC 4.0 adverse reaction grading standard of grade 3 or above.
  • The guardian does not agree to participate.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ruijin Hospital Hainan Branch, affiliated with Shanghai Jiao Tong University School of Medicine and Children's Hospital of Shanghai, affiliated with Shanghai Jiao Tong University School of Medicine

Hainan, China

Location

Related Publications (8)

  • Duke ES, Bradford D, Sinha AK, Mishra-Kalyani PS, Lerro CC, Rivera D, Wearne E, Miller CP, Leighton J, Sabit H, Zhao H, Lane A, Scepura B, Pazdur R, Singh H, Kluetz PG, Donoghue M, Drezner N. US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy. J Clin Oncol. 2024 Sep 1;42(25):3047-3057. doi: 10.1200/JCO.24.00546. Epub 2024 Jun 25.

    PMID: 38917371RESULT

  • Diccianni MB, Kempinska K, Gangoti JA, Yu AL, Sorkin LS. Anti-GD2 induced allodynia in rats can be reduced by pretreatment with DFMO. PLoS One. 2020 Jul 22;15(7):e0236115. doi: 10.1371/journal.pone.0236115. eCollection 2020.

    PMID: 32697811RESULT

  • Salemi F, Alam W, Hassani MS, Hashemi SZ, Jafari AA, Mirmoeeni SMS, Arbab M, Mortazavizadeh SMR, Khan H. Neuroblastoma: Essential genetic pathways and current therapeutic options. Eur J Pharmacol. 2022 Jul 5;926:175030. doi: 10.1016/j.ejphar.2022.175030. Epub 2022 May 20.

    PMID: 35605657RESULT

  • Jabbari P, Hanaei S, Rezaei N. State of the art in immunotherapy of neuroblastoma. Immunotherapy. 2019 Jun;11(9):831-850. doi: 10.2217/imt-2019-0018. Epub 2019 May 16.

    PMID: 31094257RESULT

  • Pieniazek B, Cencelewicz K, Bzdziuch P, Mlynarczyk L, Lejman M, Zawitkowska J, Derwich K. Neuroblastoma-A Review of Combination Immunotherapy. Int J Mol Sci. 2024 Jul 15;25(14):7730. doi: 10.3390/ijms25147730.

    PMID: 39062971RESULT

  • Pinto NR, Applebaum MA, Volchenboum SL, Matthay KK, London WB, Ambros PF, Nakagawara A, Berthold F, Schleiermacher G, Park JR, Valteau-Couanet D, Pearson AD, Cohn SL. Advances in Risk Classification and Treatment Strategies for Neuroblastoma. J Clin Oncol. 2015 Sep 20;33(27):3008-17. doi: 10.1200/JCO.2014.59.4648. Epub 2015 Aug 24.

    PMID: 26304901RESULT

  • Newman EA, Abdessalam S, Aldrink JH, Austin M, Heaton TE, Bruny J, Ehrlich P, Dasgupta R, Baertschiger RM, Lautz TB, Rhee DS, Langham MR Jr, Malek MM, Meyers RL, Nathan JD, Weil BR, Polites S, Madonna MB; APSA Cancer committee. Update on neuroblastoma. J Pediatr Surg. 2019 Mar;54(3):383-389. doi: 10.1016/j.jpedsurg.2018.09.004. Epub 2018 Sep 19.

    PMID: 30305231RESULT

  • Hubbard AK, Spector LG, Fortuna G, Marcotte EL, Poynter JN. Trends in International Incidence of Pediatric Cancers in Children Under 5 Years of Age: 1988-2012. JNCI Cancer Spectr. 2019 Mar;3(1):pkz007. doi: 10.1093/jncics/pkz007. Epub 2019 Apr 9.

    PMID: 30984908RESULT

Study Officials

  • Jingbo Shao, PhD，chief physician

    Shanghai Children's Hospital

    PRINCIPAL INVESTIGATOR

  • Wen Su, PhD，associate chief physician

    Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 8, 2025

First Posted

December 12, 2025

Study Start

April 8, 2025

Primary Completion (Estimated)

October 31, 2029

Study Completion (Estimated)

October 31, 2029

Last Updated

December 12, 2025

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)