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childhood acute myeloid leukemia - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

childhood acute myeloid leukemia

MONDO:0004996

Also known as:acute myeloid leukaemia (AML)acute myeloid leukemia (AML)acute myeloid leukaemia of childhood

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Overview

Acute myeloid leukemia occurring in childhood.

TreatmentsNo approved treatments · 1 in pipeline

Clinical Trials21 registered · 6 recruiting

Diagnosis

Biomarker and diagnostic research for childhood acute myeloid leukemia 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 childhood acute myeloid leukemia. An additional 1 compound holds orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for childhood acute myeloid leukemia, 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 childhood acute myeloid leukemia. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

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.

21 clinical trials registered, 6 recruiting. Interventions under study include drug therapy, procedural interventions, other interventions, and biologic therapy. Pipeline includes 6 PHASE2, 10 PHASE1, 1 EARLY_PHASE1. Research is primarily sponsored by academic and government institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT05857969](https://clinicaltrials.gov/study/NCT05857969)

Data assembled from 4 of 12 sources · Last updated Sep 19, 2026, 11:57 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Patient Support

Financial & Access Support (External Programs)

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Acute Myeloid Leukemia Fund

Premium/CopayCurrently AcceptingOperated by HealthWell Foundation

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Program availability and eligibility requirements are set by each foundation. Contact them directly to learn more about your options.

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Medical References

External Resources

GARD

Genetic and Rare Diseases Info Center

GARD:8199

Questions for Your Care Team

→Are any of the 21 active clinical trials a good fit for my situation?
→What is the current development status of luveltamab tazevibulin, and when might it become available?
→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
Research summaries	86	29%
Laboratory research	69	24%
Disease patterns and progression	30	10%
New treatment approaches	30	10%
Clinical study results	29	10%
Testing and diagnosis research	24	8%
Patient case studies	13	4%
Other research	12	4%

Kisho

childhood acute myeloid leukemia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Patient Support

Financial & Access Support (External Programs)

Are you a patient organization for this condition?

Medical References

External Resources

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Comprehensive genomic analysis of five kindreds with multiple childhood leukemias: importance of individual functional analysis for rare ETV6 germline variants.

childhood acute myeloid leukemia

Overview

Diagnosis

Treatment

Orphan Drug Designations

Research Landscape

Patient Support

Financial & Access Support (External Programs)

Are you a patient organization for this condition?

Medical References

External Resources

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Comprehensive genomic analysis of five kindreds with multiple childhood leukemias: importance of individual functional analysis for rare ETV6 germline variants.