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High Risk NeuroblastomaCSHEINNCT07278674

Clinical Study of Eflornithine After Immunotherapy for High-risk Neuroblastoma（CSHEIN）

An observational study of High Risk Neuroblastoma, run by Shanghai Children's Hospital at 1 site in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 1 month to 18 years
Conditions the study is for
High Risk Neuroblastoma
What is being tested?
Oral administration of eflornithine for the prevention of high-risk neuroblastoma
Type of treatment
Pill or oral medicine
How many people
20 participants
Where
1 siteChina
Study dates
8 Apr 2025to 31 Oct 2029
How long will I take part?
5 Years
Phase
Observational
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 month to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Study population: Children with high-risk neuroblastoma

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

1 site in 1 country.

China1 site

  • 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

Other trials in China

About this study

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.

Read the detailed description

The investigators plan to recruit 20 participants and divide them into Group A and Group B based on their clinical status. The inclusion criteria for Group A are: newly diagnosed high-risk neuroblastoma (NB) patients who have completed the standard treatment plan (including immunotherapy) and whose disease has reached complete response (CR) or very good partial response (VGPR). The inclusion criteria for Group B are: recurrent or refractory NB patients who have completed any treatment for recurrent disease, or who have achieved disease stability after any salvage or intensification therapy for primary refractory disease, with at least a partial response (PR) as assessed by CT or MRI and negative bone marrow aspiration. There are no restrictions regarding gender or geographic region. Participants must be under 18 years of age. Eligible subjects will be enrolled into the study after receiving the first oral dose of eflornithine, with a total treatment course of 2 years. Follow-up will continue for up to 3 years after completion of eflornithine therapy.

Being tested
Oral administration of eflornithine for the prevention of high-risk neuroblastoma
Keywords
High risk neuroblastoma, Eflornithine

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Oral administration of eflornithine for the prevention of high-risk neuroblastomaDrug

    Also called No other intervention measures

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

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: 5 years

  2. 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.

    Measured: 5 years

Secondary outcomes (1)

  1. Incidence of Treatment-Emergent Adverse Events

    This study will comprehensively evaluate the safety and tolerability of eflornithine. The primary assessments include the incidence and severity of all treatment-emergent adverse events (graded per NCI CTCAE v5.0)

    Measured: 5 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Dec 2025
  2. Study start8 Apr 2025
  3. Primary completion31 Oct 2029 (estimated)
  4. Study completion31 Oct 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
20 participants (estimated)
Target duration
5 Years
IDs, dates and publications
ClinicalTrials.gov ID
NCT07278674
Sponsor's protocol ID
ShanghaiChildrens
Acronym
CSHEIN
Status last verified
Dec 2025
First submitted
8 Feb 2025
Last updated
12 Dec 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • 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.

    PubMed 38917371

  • 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.

    PubMed 32697811

  • 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.

    PubMed 35605657

  • 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.

    PubMed 31094257

  • 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.

    PubMed 39062971

  • 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.

    PubMed 26304901

  • 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.

    PubMed 30305231

  • 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.

    PubMed 30984908

Questions about this trial

What is this clinical trial studying?

An observational study of High Risk Neuroblastoma, run by Shanghai Children's Hospital at 1 site in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 12 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The event free survival rate after the completion of the first DFMO (time frame: 5 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07278674, which also lists 8 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Dec 2025.

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