Nelarabine

Summary

Nelarabine (CHEMBL1201112) is an approved small-molecule antineoplastic agent (ATC L01BB07); indicated across 15 conditions including neoplasm and t-cell acute lymphoblastic leukemia; with CIViC clinical evidence for 3 variant-indication associations (e.g. NT5C2 D407A in t-cell acute lymphoblastic leukemia).

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: L01BB07
• Indications: 15 conditions
• Clinical trials: 31
• Precision-oncology evidence (CIViC): 3 variant–indication associations
• Chemistry: 297.27 Da · C11H15N5O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201112
Name	Nelarabine
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	3011155
ChEBI	CHEBI:63612
ATC	L01BB07
Molecular formula	C11H15N5O5
Molecular weight	297.27
InChIKey	IXOXBSCIXZEQEQ-UHTZMRCNSA-N

SMILES: COC1=NC(=NC2=C1N=CN2[C@H]3[C@H]([C@@H]([C@H](O3)CO)O)O)N

IUPAC name: (2R,3S,4S,5R)-2-(2-amino-6-methoxypurin-9-yl)-5-(hydroxymethyl)oxolane-3,4-diol

ChEBI definition: A purine nucleoside in which O-methylguanine is attached to arabinofuranose via a β-N9-glycosidic bond. Inhibits DNA synthesis and causes cell death; a prodrug of 9-β-D-arabinofuranosylguanine (ara-G).

Pharmacological roles (ChEBI): antineoplastic agent, DNA synthesis inhibitor, prodrug.

Also known as: 506U, 506U78, Arranon, Atriance, Attriance, GW-506U78, MAY, Nelarabina, Nelarabine, Nelzarabine, NSC-686673, NSC-755985

Patent coverage: 6,914 distinct patent families (28,176 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 27,871 (99%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

5 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
neoplasm	4	MONDO:0005070	EFO:0000616
T-cell acute lymphoblastic leukemia	4	MONDO:0004963	EFO:0000209
precursor T-cell acute lymphoblastic leukemia	4	MONDO:0020512	EFO:1001830
leukemia	3	MONDO:0005059	EFO:0000565
acute lymphoblastic leukemia	3	MONDO:0004967	EFO:0000220

8 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
lymphoma	2	MONDO:0005062	EFO:0000574
Sezary syndrome	2	MONDO:0017844	EFO:1000785
mycosis fungoides	2	MONDO:0009691	EFO:1001051
lymphoid leukemia	1	MONDO:0005402	EFO:0004289
myelodysplastic syndrome	1	MONDO:0018881	EFO:0000198
plasma cell myeloma	1	MONDO:0009693	EFO:0001378
myeloproliferative neoplasm	1	MONDO:0020076	EFO:0002428
plasma cell neoplasm	1	MONDO:0004959	EFO:0000200

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 31.

Phase distribution

Phase	Trials
PHASE2	14
Not specified	5
PHASE3	4
PHASE1/PHASE2	3
PHASE1	3
PHASE2/PHASE3	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03020030	PHASE3	ACTIVE_NOT_RECRUITING	Treatment of Newly Diagnosed Acute Lymphoblastic Leukemia in Children and Adolescents
NCT03117751	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Total Therapy XVII for Newly Diagnosed Patients With Acute Lymphoblastic Leukemia and Lymphoma
NCT06434467	PHASE3	NOT_YET_RECRUITING	The Efficacy and Safety of Nelarabine Injection in Patients With T-lymphoblastic Leukemia and T-lymphoblastic Lymphoma
NCT07072585	PHASE2/PHASE3	NOT_YET_RECRUITING	Testing the Addition of Daratumumab to Chemotherapy for Treating Patients With Newly-Diagnosed T-Cell Lymphoblastic Leukemia (T-ALL) and T-Cell Lymphoblastic Lymphoma (T-LL)
NCT00408005	PHASE3	COMPLETED	Combination Chemotherapy in Treating Young Patients With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia or T-cell Lymphoblastic Lymphoma
NCT02881086	PHASE3	COMPLETED	Optimization of Therapy in Adult Patients With Newly Diagnosed Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma by Individualised, Targeted and Intensified Treatment
NCT00501826	PHASE2	RECRUITING	Combination Chemotherapy and Nelarabine in Treating Patients With T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma
NCT06390319	PHASE2	RECRUITING	Adding Dasatinib Or Venetoclax To Improve Responses In Children With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (ALL) Or Lymphoma (T-LLY) Or Mixed Phenotype Acute Leukemia (MPAL)
NCT07294677	PHASE1/PHASE2	RECRUITING	CApivasertib, Venetoclax And Low-intensity chemotheRapY for Adults With ALL/LBL
NCT00002970	PHASE2	COMPLETED	506U78 in Treating Patients With Refractory Hematologic Cancer
NCT00003545	PHASE2	COMPLETED	506U78 in Treating Patients With Refractory or Relapsed Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma
NCT00003635	PHASE2	COMPLETED	506U78 in Treating Patients With Chronic Lymphocytic Leukemia That Has Not Responded to Fludarabine or Alkylating Agents
NCT00005080	PHASE2	COMPLETED	506U78 in Treating Patients With Lymphoma
NCT00005950	PHASE2	TERMINATED	506U78 in Treating Patients With Recurrent or Refractory Non-Hodgkin’s Lymphoma or T-cell Lymphoma
NCT00005982	PHASE2	TERMINATED	506U78 in Treating Patients With Recurrent or Refractory Cutaneous T-cell Lymphoma
NCT00006020	PHASE2	COMPLETED	S0010 506U78 in Treating Patients With Recurrent or Refractory Acute Lymphocytic Leukemia
NCT00684619	PHASE2	COMPLETED	506U78 In Relapsed Or Refractory Acute Lymphoblastic Leukemia (T-ALL)
NCT00981799	PHASE1/PHASE2	TERMINATED	Trial of Nelarabine, Etoposide and Cyclophosphamide in Relapsed T-cell ALL and T-cell LL
NCT02518750	PHASE2	TERMINATED	Re-Induction Therapy for Relapsed Pediatric T-Cell Acute Lymphoblastic Leukemia or Lymphoma
NCT02619630	PHASE2	UNKNOWN	Multicenter Study of Risk-adapted Treatment for T-lineage ALL of Young Adults (18-59 Years Old)
NCT02763384	PHASE2	TERMINATED	BL-8040 and Nelarabine for Relapsed or Refractory T-Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma
NCT03808610	PHASE1/PHASE2	TERMINATED	Low-Intensity Chemotherapy and Venetoclax in Treating Patients With Relapsed or Refractory B- or T-Cell Acute Lymphoblastic Leukemia
NCT06210750	PHASE2	WITHDRAWN	Adding Targeted Drugs to Usual Chemotherapy for Adults With Newly Diagnosed T-Cell Acute Lymphoblastic Leukemia (T-ALL) and T-Cell Lymphoblastic Lymphoma (T-LBL)
NCT00004239	PHASE1	TERMINATED	506U78 in Treating Patients With Hematologic Cancer and Kidney or Liver Impairment
NCT01094860	PHASE1	COMPLETED	Pharmacokinetic (PK) and Pharmacodynamic (PD) Study of Nelarabine in Patients With Relapsed/Refractory Lymphoid Malignancies
NCT03328104	PHASE1	COMPLETED	Everolimus in Combination With Nelarabine, Cyclophosphamide and Etoposide in Lymphoblastic Leukemia/Lymphoma
NCT00003837	Not specified	COMPLETED	506U78 in Treating Patients With Relapsed or Refractory T-cell Acute Lymphoblastic Leukemia or T-cell Lymphoblastic Lymphoma
NCT00016302	Not specified	COMPLETED	Combination Chemotherapy in Treating Patients With Newly Diagnosed Acute Lymphoblastic Leukemia
NCT00866671	Not specified	COMPLETED	Observational Study of Nelarabine in Children and Young Adults
NCT01085617	Not specified	COMPLETED	Standard Chemotherapy with or Without Nelarabine or Rituximab in Treating Patients with Newly Diagnosed Acute Lymphoblastic Leukemia
NCT01376115	Not specified	COMPLETED	Drug Use Investigation for ARRANON G (Nelarabine) Injection 250mg

Clinical evidence (CIViC)

Variant × indication × effect (3 predictive associations from 3 curated evidence items):

Variant	Indication	Effect	Therapy	Level	CIViC
NT5C2 D407A	T-cell Acute Lymphoblastic Leukemia	Resistance	Nelarabine + Arabinosylguanine	CIViC D	EID635
NT5C2 K359Q	T-cell Acute Lymphoblastic Leukemia	Resistance	Arabinosylguanine + Nelarabine	CIViC D	EID636
NT5C2 R367Q	T-cell Acute Lymphoblastic Leukemia	Resistance	Nelarabine + Arabinosylguanine	CIViC D	EID633

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).