Childhood acute myeloid leukemia

Summary

Childhood acute myeloid leukemia (MONDO:0004996) is a cancer and 318 clinical trials. Top therapeutic interventions include daunorubicin, venetoclax, and gilteritinib. A subtype of childhood leukemia — broader associated-gene and molecular evidence is on the parent page (see Disease family below).

At a glance

• Classification: Cancer
• Clinical trials: 318

Clinical features

No curated clinical features (Orphanet) for this disease.

Identifiers

Disease identifiers

Field	Value
Canonical name	childhood acute myeloid leukemia
Mondo ID	MONDO:0004996
EFO	EFO:0000330
DOID	DOID:0070323
NCIT	C9160
UMLS	C0220621
MedGen	65075
GARD	0008199
Is cancer (heuristic)	yes

Also known as: acute myeloid leukaemia (AML) · acute myeloid leukaemia of childhood · acute myeloid leukemia (AML) · acute myeloid leukemia of childhood · childhood acute granulocytic leukaemia · childhood acute granulocytic leukemia · childhood acute myeloblastic leukaemia · childhood acute myeloblastic leukemia · childhood acute myelocytic leukaemia · childhood acute myelocytic leukemia · childhood acute myelogenous leukaemia · childhood acute myelogenous leukemia · childhood acute myeloid leukemia · childhood AML · paediatric acute myeloblastic leukaemia · paediatric acute myelocytic leukaemia · paediatric acute myelogenous leukaemia · paediatric AML · pediatric acute myeloblastic leukemia · pediatric acute myelocytic leukemia (+3 more)

Data availability: 28 cell lines.

Disease family

This is a subtype of childhood leukemia. Genetic, therapeutic, and trial evidence is largely curated at the broader-term level — see the parent page for the associated-gene cohort and molecular evidence.

Classification path: disease › human disease › disease by etiologic mechanism › cancer or benign tumor › neoplastic disease or syndrome › neoplasm › hematopoietic and lymphoid system neoplasm › hematopoietic and lymphoid cell neoplasm › leukemia › childhood leukemia › childhood acute myeloid leukemia

Related subtypes (2): childhood acute lymphoblastic leukemia, neonatal leukemia

Genetics & variants

GWAS landscape

No GWAS associations recorded — common-variant (GWAS) studies don’t cover this disease (typical for Mendelian / rare diseases). See the curated gene cohort and Mendelian overlap below.

Variant details and genetic-evidence tiers

No tiered GWAS variants or ClinVar records for this disease.

Genes & proteins

No associated-gene cohort resolved for this disease. Atlas builds the molecular and therapeutic sections — associated genes, protein families, druggability, pathways, interactions, and drug associations — by aggregating over a disease’s associated genes (resolved via GWAS / GenCC / ClinVar / CIViC), and none resolved here. This is expected for antibody-mediated, autoimmune, or otherwise non-gene-defined conditions; the curated evidence for this disease is its clinical features, GWAS susceptibility, and clinical trials (above).

Function

No pathway enrichment — requires an associated-gene cohort.

Therapeutics

No druggable-target or therapeutic data for this disease’s cohort.

Clinical trials & evidence

Clinical trials

Clinical trials: 318.

Phase distribution (across all retrieved trials)

Phase	Trials
PHASE2	87
PHASE1	81
Not specified	65
PHASE1/PHASE2	41
PHASE3	31
EARLY_PHASE1	6
PHASE2/PHASE3	5
PHASE4	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05144243	PHASE4	ACTIVE_NOT_RECRUITING	Study to Assess Adverse Events and Change in Disease State of Oral Venetoclax in Combination With Subcutaneous (SC) Azacitidine in Newly Diagnosed Adult Participants With Acute Myeloid Leukemia (AML) Who Are Ineligible for Intensive Chemotherapy in China
NCT03176849	PHASE4	COMPLETED	A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT
NCT02416388	PHASE2/PHASE3	RECRUITING	Study to Improve OS in 18 to 60 Year-old Patients, Comparing Daunorubicin Versus High Dose Idarubicin Induction Regimens, High Dose Versus Intermediate Dose Cytarabine Consolidation Regimens, and Standard Versus MMF Prophylaxis of GvHD in Allografted Patients in First CR
NCT05907057	PHASE3	RECRUITING	An Open-label Phase 3b Study of Ivosidenib in Combination With Azacitidine in Adult Patients Newly Diagnosed With IDH1m Acute Myeloid Leukemia (AML) Ineligible for Intensive Induction Chemotherapy.
NCT06788756	PHASE2/PHASE3	RECRUITING	L-Annamycin for Injection in Combination With Cytarabine Injection as Second Line Therapy for Remission Induction in Adult Subjects With Refractory/Relapsed AML
NCT06990321	PHASE3	RECRUITING	Effectiveness of Medium-Dose Cytarabine Combined With Venetoclax for Consolidation Therapy in Elderly Patients With Intermediate to High-Risk Acute Myeloid Leukemia
NCT07007312	PHASE3	RECRUITING	Studies to Assess Ziftomenib in Combination With Ven+Aza or 7+3 in Patients With Untreated NPM1-m or KMT2A-r AML
NCT07264010	PHASE2/PHASE3	NOT_YET_RECRUITING	Sorafenib Combined With Venetoclax as Pre-emptive Therapy Strategy for MRD+ AML: a Prospective, Single-arm, Multicenter Clinical Study
NCT07463651	PHASE3	RECRUITING	MRD-guided Maintenance Post-HCT: Gilteritini vs Sorafenib
NCT07469046	PHASE3	NOT_YET_RECRUITING	VAH vs VA in Newly Diagnosed Elderly AML
NCT07486479	PHASE3	NOT_YET_RECRUITING	Venetoclax, Azacitidine, and Mitoxantrone Hydrochloride Liposome Versus Idarubicin and Cytarabine in Newly Diagnosed AML
NCT07583888	PHASE2/PHASE3	RECRUITING	VABu Conditioning in Elderly AML HSCT
NCT00322101	PHASE3	COMPLETED	Low-Dose or High-Dose Conditioning Followed by Peripheral Blood Stem Cell Transplant in Treating Patients With Myelodysplastic Syndrome or Acute Myelogenous Leukemia
NCT00568633	PHASE3	TERMINATED	Allogeneic HCT Using Nonmyeloablative Host Conditioning With TLI & ATG vs SOC in AML
NCT00606723	PHASE3	COMPLETED	Allogenic Stem Cell Transplantation for Children, Adolescents and Young Adults With Relapsed or Refractory AML
NCT01147939	PHASE3	COMPLETED	Study of Elacytarabine Versus Investigator’s Choice in Patients With Late Stage Acute Myeloid Leukaemia (AML)
NCT01191957	PHASE3	COMPLETED	Busulfan (BU) Plus Fludarabine Vs Intravenous BU Plus Cyclophosphamide as Conditioning Regimens Prior Allogeneic Hematopoetic Stem Cells Transplant (HSCT) in AML
NCT01237808	PHASE3	COMPLETED	Study of Low-Dose Cytarabine and Etoposide With or Without All-Trans Retinoic Acid in Older Patients Not Eligible for Intensive Chemotherapy With Acute Myeloid Leukemia and NPM1 Mutation
NCT01305200	PHASE3	COMPLETED	Supersaturated Calcium Phosphate Rinse in Preventing Oral Mucositis in Young Patients Undergoing Autologous or Donor Stem Cell Transplant
NCT01307579	PHASE3	COMPLETED	Caspofungin Versus Fluconazole in Preventing Invasive Fungal Infections (IFI) in Patients Undergoing Chemotherapy for Acute Myeloid Leukemia
NCT01371656	PHASE3	COMPLETED	Levofloxacin in Preventing Infection in Young Patients With Acute Leukemia Receiving Chemotherapy or Undergoing Stem Cell Transplantation
NCT01817075	PHASE3	COMPLETED	Chlorhexidine Gluconate Cleansing in Preventing Central Line Associated Bloodstream Infection and Acquisition of Multi-drug Resistant Organisms in Younger Patients With Cancer or Undergoing Donor Stem Cell Transplant
NCT01828489	PHASE3	UNKNOWN	Research Study for Treatment of Children and Adolescents With Acute Myeloid Leukaemia 0-18 Years
NCT02013648	PHASE3	COMPLETED	Randomized Phase III Study of Intensive Chemotherapy With or Without Dasatinib (Sprycel™)
NCT02172872	PHASE3	UNKNOWN	InDACtion vs 3+7 Induction in AML
NCT02752035	PHASE3	COMPLETED	A Study of ASP2215 (Gilteritinib) by Itself, ASP2215 Combined With Azacitidine or Azacitidine by Itself to Treat Adult Patients Who Have Recently Been Diagnosed With Acute Myeloid Leukemia With a FLT3 Gene Mutation and Who Cannot Receive Standard Chemotherapy
NCT02927938	PHASE3	TERMINATED	Leukemia Stem Cell Detection in Acute Myeloid Leukemia
NCT02993523	PHASE3	COMPLETED	A Study of Venetoclax in Combination With Azacitidine Versus Azacitidine in Treatment Naïve Participants With Acute Myeloid Leukemia Who Are Ineligible for Standard Induction Therapy
NCT03069352	PHASE3	COMPLETED	A Study of Venetoclax in Combination With Low Dose Cytarabine Versus Low Dose Cytarabine Alone in Treatment Naive Patients With Acute Myeloid Leukemia Who Are Ineligible for Intensive Chemotherapy
NCT03512197	PHASE3	COMPLETED	A Global Study of the Efficacy and Safety of Midostaurin + Chemotherapy in Newly Diagnosed Patients With FLT3 Mutation Negative (FLT3-MN) Acute Myeloid Leukemia (AML)
NCT03941964	PHASE3	COMPLETED	A Study of the Effectiveness of Venetoclax in Combination With Azacitidine or Decitabine in an Outpatient Setting in Patients With Acute Myeloid Leukemia (AML) Ineligible for Intensive Chemotherapy
NCT04102020	PHASE3	COMPLETED	A Study of Oral Venetoclax Tablets and Oral Azacitidine as Maintenance Therapy in Adult Participants With Acute Myeloid Leukemia in First Remission After Conventional Chemotherapy
NCT04161885	PHASE3	TERMINATED	A Study Evaluating Safety and Efficacy of Venetoclax in Combination With Azacitidine Versus Standard of Care After Allogeneic Stem Cell Transplantation (SCT) in Participants With Acute Myeloid Leukemia (AML)
NCT04326764	PHASE3	TERMINATED	Panobinostat Maintenance After HSCT fo High-risk AML and MDS
NCT04509622	PHASE3	COMPLETED	A Study of Oral Venetoclax Tablet in Combination With Subcutaneous Low-Dose Cytarabine (LDAC) Injection to Assess Adverse Events in Adult Japanese Participants With Acute Myeloid Leukemia (AML)
NCT04716114	PHASE3	UNKNOWN	A Study of SKLB1028 Versus Salvage Chemotherapy in Patients With Relapsed or Refractory (R/R) AML With FLT3-Mutated
NCT05313958	PHASE2/PHASE3	UNKNOWN	Treateament of Newly Diagnosed Acute Monocytic Leukemia in Children
NCT06211452	PHASE3	COMPLETED	Comparison of Consolidation Strategies for Pediatric Patients With Acute Myeloid Leukemia
NCT02094794	PHASE2	ACTIVE_NOT_RECRUITING	Total Marrow and Lymphoid Irradiation and Chemotherapy Before DSCT in Treating Patients With High-Risk ALL or AML
NCT02781883	PHASE2	RECRUITING	Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML

Drugs tested across these trials (top 30)

Molecule	Max phase	Trials referencing
DAUNORUBICIN	4	14
VENETOCLAX	4	13
GILTERITINIB	4	11
AZACITIDINE	4	10
IDARUBICIN	4	6
ALDESLEUKIN	4	5
SORAFENIB	4	5
DASATINIB ANHYDROUS	4	4
IVOSIDENIB	4	4
MITOXANTRONE	4	4
TREOSULFAN	4	4
DECITABINE	4	3
MIDOSTAURIN	4	3
ALEMTUZUMAB	4	2
AXITINIB	4	2
BLINATUMOMAB	4	2
BUSULFAN	4	2
CLADRIBINE	4	2
OMACETAXINE MEPESUCCINATE	4	2
QUIZARTINIB	4	2
REVUMENIB	4	2
SELINEXOR	4	2
TAGRAXOFUSP	4	2
2-MERCAPTOETHANESULFONIC ACID	4	1
ASPARAGINASE ERWINIA CHRYSANTHEMI	4	1
ATORVASTATIN CALCIUM	4	1
CASPOFUNGIN ACETATE	4	1
CEDAZURIDINE	4	1
CHOLECALCIFEROL	4	1
CRISANTASPASE	4	1