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nut midline carcinoma - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

nut midline carcinoma

MONDO:0005563

Also known as:Midline carcinoma of children and Young adults with NUT rearrangementNMCNUT carcinoma

Find Clinical TrialsTrials

Overview

A rare, highly aggressive and lethal carcinoma that affects children and young adults. It arises from midline epithelial structures, most commonly the head, neck, and mediastinum. It is a poorly differentiated carcinoma and is characterized by mutations and rearrangement of the NUT gene. A balanced translocation t(15;19) is present that results in the creation of a fusion gene involving the NUT gene, most commonly BRD4-NUT fusion gene.

TreatmentsNo approved treatments · 4 in pipeline

Clinical Trials8 registered · 3 recruiting

Diagnosis

Biomarker and diagnostic research for nut midline carcinoma 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.

No approved treatments are currently available for nut midline carcinoma. An additional 4 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for nut midline carcinoma, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for nut midline carcinoma. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Estimated prevalence: <1 in 1,000,000 (VERY_RARE).

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.

8 clinical trials registered, 3 recruiting. Interventions under study include other interventions, drug therapy, procedural interventions, and gene therapy. Pipeline includes 1 PHASE2, 3 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT07072143](https://clinicaltrials.gov/study/NCT07072143)

Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 3:32 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:443167

GARD

Genetic and Rare Diseases Info Center

GARD:21852

Questions for Your Care Team

→Are any of the 8 active clinical trials a good fit for my situation?
→With 4 compounds in the orphan drug pipeline, which are furthest along in development and which clinical trials should I be aware of?
→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

Research Type	Count	% of Total
Patient case studies	20	34%
Research summaries	18	31%
Laboratory research	9	16%
Testing and diagnosis research	4	7%
Disease patterns and progression	4	7%
Other research	1	2%
Clinical study results	1	2%
New treatment approaches	1	2%

Kisho

nut midline carcinoma

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Radiotherapy in Thoracic NUT Carcinoma: A Pooled Analysis of Long-term Survivors and Clinical Outcome Heterogeneity.

Head and neck NUT carcinoma-radiotherapy dose, chemotherapy choice, and outcomes: lessons from a 7-year complete remission.

Downhill Esophageal Variceal Rupture Caused by Superior Vena Cava Syndrome in NUT Carcinoma: A Case Report.

nut midline carcinoma

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Radiotherapy in Thoracic NUT Carcinoma: A Pooled Analysis of Long-term Survivors and Clinical Outcome Heterogeneity.

Head and neck NUT carcinoma-radiotherapy dose, chemotherapy choice, and outcomes: lessons from a 7-year complete remission.

Downhill Esophageal Variceal Rupture Caused by Superior Vena Cava Syndrome in NUT Carcinoma: A Case Report.