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neuroblastoma - Clinical Profile, Treatments & Trials | Kisho | Kisho

Rare Disease

neuroblastoma

MONDO:0005072

Also known as:neural Crest tumor, malignantneuroblastoma (Schwannian Stroma-poor)neuroblastoma, malignant

Find Clinical TrialsTrials

Overview

Neuroblastoma (NB) is the most common solid, extracranial childhood tumor. It is an aggressive pediatric cancer that originates from neural crest tissues of the sympathetic nervous system.

Treatments5 FDA approved

Clinical Trials189 registered · 96 recruiting

Diagnosis

Biomarker and diagnostic research for neuroblastoma has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

5 FDA-approved treatments are available for neuroblastoma, including NAXITAMAB (DANYELZA, approved 2020), CYCLOPHOSPHAMIDE (CYCLOPHOSPHAMIDE, approved 2020), and DINUTUXIMAB (UNITUXIN, approved 2015). An additional 37 compounds hold orphan drug designation.

Approved Therapies

Brand Name	Generic Name	Mechanism	Approved

Prognosis

Estimated prevalence: 1-5 in 10,000 (Uncommon).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

189 clinical trials registered, 96 recruiting. Interventions under study include drug therapy, biologic therapy, other interventions, and procedural interventions. Pipeline includes 2 PHASE4, 9 PHASE3, 51 PHASE2. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT06540963](https://clinicaltrials.gov/study/NCT06540963)

Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 7:13 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:635

GARD

Genetic and Rare Diseases Info Center

GARD:7185

Questions for Your Care Team

→What are the benefits and potential side effects of DANYELZA for managing neuroblastoma?
→Are any of the 189 active clinical trials a good fit for my situation?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Brand Name	Generic Name	Sponsor	Designated	Exclusivity End	Designation Status
dicaprate	dicaprate	Aminex Therapeutics, Inc.	2025	—	Designated
a genetically modified salmonella comprising of Diphtheria toxin A subunit	a genetically modified salmonella comprising of Diphtheria toxin A subunit	Shanghai Salvectors Biotechnology LTD.	2025	—	Designated
PHOX2B PC-CAR T Cells	PHOX2B PC-CAR T Cells	Hula Therapeutics Inc.	2024	—	Designated
silmitasertib	silmitasertib	Senhwa Biosciences, Inc.	2024	—	Designated
Opaganib	Opaganib	RedHill Biopharma Ltd.	2024	—	Designated
humanized anti-GD2 monoclonal antibody	humanized anti-GD2 monoclonal antibody	Renaissance Pharma, Ltd.	2024	—	Designated
bispecific anti-GD2/anti-B7-H3 monoclonal antibody	bispecific anti-GD2/anti-B7-H3 monoclonal antibody	Invenra Inc.	2024	—	Designated
ribociclib	ribociclib	Novartis Pharmaceuticals Corporation	2022	—	Designated
64-fluoro-36-methox-27-methyl-5-thia-4,8-diaza-2(4,6)-tieno[3,2-d]pyrimidine-3(3,5)-pyridina-1(2,5)-thiophena-6(1,3)-benzeneacyclononaphan-7-one 5,5-dioxide	64-fluoro-36-methox-27-methyl-5-thia-4,8-diaza-2(4,6)-tieno[3,2-d]pyrimidine-3(3,5)-pyridina-1(2,5)-thiophena-6(1,3)-benzeneacyclononaphan-7-one 5,5-dioxide	AUM Biosciences	2022	—	Designated
alrizomadlin	alrizomadlin	Ascentage Pharma Group Inc.	2022	—	Designated
Rilpivirine oral suspension	Rilpivirine oral suspension	Aptorum International Limited	2022	—	Withdrawn
Nivatrotamab	Nivatrotamab	Y-mAbs Therapeutics, Inc.	2020	—	Designated
2-(4-(4-((1-(107-(4-(guanidinomethyl)phenoxy)-(polyethyleneglycol36))-1H-1,2,3-triazol-4-yl)methoxy)-3,5-diiodophenoxy)-3,5-diiodophenyl)acetic acid	2-(4-(4-((1-(107-(4-(guanidinomethyl)phenoxy)-(polyethyleneglycol36))-1H-1,2,3-triazol-4-yl)methoxy)-3,5-diiodophenoxy)-3,5-diiodophenyl)acetic acid	NanoPharmaceuticals, LLC	2020	—	Designated
[64Cu]Cu-[MeCOSar0-Tyr3]-octreotate	[64Cu]Cu-[MeCOSar0-Tyr3]-octreotate	Clarity Pharmaceuticals Ltd.	2020	—	Designated
[67Cu]Cu-[MeCOSar0-Tyr3]-octreotate	[67Cu]Cu-[MeCOSar0-Tyr3]-octreotate	Clarity Pharmaceuticals Ltd.	2020	—	Designated
Gallium-68 DOTANOC	Gallium-68 DOTANOC	Advanced Imaging Projects, LLC	2020	—	Designated
(2R,4R)-1-(3-chloro-2-fluorobenzyl)-4-((3-fluoro-6-((5-methyl-1H-pyrazol-3-yl)amino)pyridin-2-yl)methyl)-2-methylpiperidine-4-carboxylic acid; 2- methylpropan-2-amine	(2R,4R)-1-(3-chloro-2-fluorobenzyl)-4-((3-fluoro-6-((5-methyl-1H-pyrazol-3-yl)amino)pyridin-2-yl)methyl)-2-methylpiperidine-4-carboxylic acid; 2- methylpropan-2-amine	Eli Lilly and Company	2020	—	Designated
18F-meta-fluorobenzylguanidine	18F-meta-fluorobenzylguanidine	Innervate Radiopharmaceuticals, LLC	2019	—	Designated
Temozolomide	Temozolomide	ORPHELIA Pharma	2019	—	Designated
Rose bengal disodium	Rose bengal disodium	Provectus BioPharmaceuticals, Inc.	2018	—	Designated
[4-(4-{1-[2-(2-{2-[2-(2-{2-[2-(4-guanidinomethyl-phenoxy)-ethoxy]-ethoxy}-ethoxy)-ethoxy]-ethoxy}-ethoxy)-ethyl]-1H-[1,2,3]triazol-4-ylmethoxy}-3,5-diiodo-phenoxy)-3,5-diiodo-phenyl]-acetic acid	[4-(4-{1-[2-(2-{2-[2-(2-{2-[2-(4-guanidinomethyl-phenoxy)-ethoxy]-ethoxy}-ethoxy)-ethoxy]-ethoxy}-ethoxy)-ethyl]-1H-[1,2,3]triazol-4-ylmethoxy}-3,5-diiodo-phenoxy)-3,5-diiodo-phenyl]-acetic acid	NanoPharmaceuticals, LLC	2018	—	Designated
18-(p-[131]-iodophenyl)octadecyl phosphocholine	18-(p-[131]-iodophenyl)octadecyl phosphocholine	Cellectar Biosciences, Inc.	2018	—	Designated
taurolidine	taurolidine	CorMedix, Inc.	2018	—	Designated
16 base single stranded PNA oligonucleotide¿7 aminoacids peptide	16 base single stranded PNA oligonucleotide¿7 aminoacids peptide	BIOGENERA SpA	2017	—	Designated
3-(5-Fluoro-benzofuran-3-yl)-4-(5-methyl-5H-[1,3]dioxolo[4,5 f]indol-7-yl)-pyrrole-2,5-dione	3-(5-Fluoro-benzofuran-3-yl)-4-(5-methyl-5H-[1,3]dioxolo[4,5 f]indol-7-yl)-pyrrole-2,5-dione	Actuate Therapeutics	2017	—	Designated
131-I-8H9 monoclonal antibody	131-I-8H9 monoclonal antibody	Y-mAbs Therapeutics, Inc.	2016	—	Designated
sodium 2-hydroxylinoleate	sodium 2-hydroxylinoleate	Ability Pharmaceuticals, SL	2015	—	Designated
anisina	anisina	Novogen Ltd	2015	—	Designated
entrectinib	entrectinib	Genentech, Inc.	2014	—	Withdrawn
MV-NB-02, its bivalent ganglioside vaccine consisting of GD2-lactone and GD3-lactone each covalently conjugated to keyhole hemocyanin	MV-NB-02, its bivalent ganglioside vaccine consisting of GD2-lactone and GD3-lactone each covalently conjugated to keyhole hemocyanin	Y-mAbs Therapeutics, Inc.	2014	—	Designated
crizotinib	crizotinib	Pfizer, Inc.	2012	—	Designated
dendritic hybrid cell vaccine	dendritic hybrid cell vaccine	Orbis Health Solutions, LLC	2011	—	Designated
firtecan pegol	firtecan pegol	Enzon Pharmaceuticals, Inc.	2011	—	Designated
perifosine	perifosine	Aeterna Zentaris GmbH	2010	—	Withdrawn
monoclonal antibody 3F8	monoclonal antibody 3F8	United Therapeutics Corporation	2008	—	Designated
Fenretinide	Fenretinide	USC-CHLA Institute for Pediatric Clinical Research	2005	—	Designated
N-[2-[(4-hydroxyphenyl)amino]-3-pyridinyl]-4-methoxybenzenesulfonamide	N-[2-[(4-hydroxyphenyl)amino]-3-pyridinyl]-4-methoxybenzenesulfonamide	AbbVie, Inc.	2004	—	Withdrawn

Research Type	Count	% of Total
Laboratory research	192	43%
Research summaries	68	15%
New treatment approaches	60	13%
Testing and diagnosis research	42	9%
Disease patterns and progression	40	9%
Clinical study results	30	7%
Patient case studies	16	4%
Other research	3	1%

Kisho

neuroblastoma

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Adverse Event Profiles

Clinical Trials

Recent Publications

Latest News & Developments

In Vivo Cascade Cyclic Amplification Strategy for Detecting the Relative Expression of MYCN mRNA in Neuroblastoma.

CoCoRV-nf: a powerful and cost-effective tool for rare variant analysis leveraging external biobank sequence data identified new candidate predisposition genes in amyotrophic lateral sclerosis and neuroblastoma.

Association Between Dinutuximab Beta Exposure and Post-End-of-Treatment Survival in Neuroblastoma: A Weighted Patient-Level Analysis of Three Clinical Studies.

neuroblastoma

Overview

Diagnosis

Treatment

Approved Therapies

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Subtypes

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Orphan Drug Designations

Adverse Event Profiles

Clinical Trials

Recent Publications

Latest News & Developments

In Vivo Cascade Cyclic Amplification Strategy for Detecting the Relative Expression of MYCN mRNA in Neuroblastoma.

CoCoRV-nf: a powerful and cost-effective tool for rare variant analysis leveraging external biobank sequence data identified new candidate predisposition genes in amyotrophic lateral sclerosis and neuroblastoma.

Association Between Dinutuximab Beta Exposure and Post-End-of-Treatment Survival in Neuroblastoma: A Weighted Patient-Level Analysis of Three Clinical Studies.