Clofazimine

Summary

Clofazimine (CHEMBL1292) is an approved small molecule (ATC J04BA01) targeting ZC3H14; indicated across 10 conditions including leprosy and pulmonary tuberculosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: J04BA01
• Targets: 1 (ZC3H14)
• Indications: 10 conditions
• Clinical trials: 34
• Chemistry: 473.4 Da · C27H22Cl2N4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1292
Name	Clofazimine
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	2794
ATC	J04BA01
Molecular formula	C27H22Cl2N4
Molecular weight	473.4
InChIKey	WDQPAMHFFCXSNU-UHFFFAOYSA-N

SMILES: CC(C)N=C1C=C2C(=NC3=CC=CC=C3N2C4=CC=C(C=C4)Cl)C=C1NC5=CC=C(C=C5)Cl

IUPAC name: N,5-bis(4-chlorophenyl)-3-propan-2-yliminophenazin-2-amine

Also known as: Chlofazimine, Clofazimina, Clofazimine, Clofaziminum, Colfazimine, G 30320, G-30320, G30320, Lamprene, NSC-141046, clofazimine, SID11112506

Patent coverage: 4,213 distinct patent families (15,481 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 15,218 (98%) 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

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
ZC3H14	zinc finger CCCH-type containing 14	Binding		0%	Q6PJT7

Broader ChEMBL bioactivity targets: 31 (assay-derived). Sample: Streptokinase A, Prelamin-A/C, 4’-phosphopantetheinyl transferase ffp, ATP-binding cassette sub-family C member 4, Alpha-2C adrenergic receptor, Glucocorticoid receptor, Bile acid receptor, Estrogen receptor, Menin/Histone-lysine N-methyltransferase MLL, Muscarinic acetylcholine receptor M1, D(2) dopamine receptor, Sodium-dependent serotonin transporter, Histamine H1 receptor, Delta-type opioid receptor, Kappa-type opioid receptor, Voltage-gated inwardly rectifying potassium channel KCNH2, 3’,5’-cyclic-AMP phosphodiesterase 4D, Cytochrome P450 2D6, Glutamate receptor ionotropic, NMDA 1, Gamma-aminobutyric acid receptor subunit alpha-1.

Bioactivity

ChEMBL activities: 24 potent at pChembl ≥ 5 of 46 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
NR3C1	7.47	AC50	34	nM	CHEMBL_ACT_25176330
ESR1	6.89	AC50	130	nM	CHEMBL_ACT_25167767
ABCB1	6.22	IC50	600	nM	CHEMBL_ACT_11000881
GHSR	6.04	AC50	920	nM	CHEMBL_ACT_25173270
KCNH2	6.02	AC50	958.1	nM	CHEMBL_ACT_25118634
ABCB1	6	IC50	1000	nM	CHEMBL_ACT_11001296
ABCB1	5.96	IC50	1100	nM	CHEMBL_ACT_11000899
P10520	5.91	EC50	1243	nM	CHEMBL_ACT_4937482
P10520	5.64	EC50	2281	nM	CHEMBL_ACT_5002664
HIF1A	5.6	Potency	2512	nM	CHEMBL_ACT_4129593
HIF1A	5.6	Potency	2512	nM	CHEMBL_ACT_4518629
CYP2D6	5.6	Potency	2512	nM	CHEMBL_ACT_4961678
CYP2D6	5.6	AC50	2512	nM	CHEMBL_ACT_5987204
ABCG2	5.5	IC50	3200	nM	CHEMBL_ACT_24777419
MAPK1	5.5	Potency	3162	nM	CHEMBL_ACT_4525369
MEN1	5.5	Potency	3162	nM	CHEMBL_ACT_4540856
P0DTC2	5.42	Kd	3820	nM	CHEMBL_ACT_24904592
P22002	5.3	AC50	5000	nM	CHEMBL_ACT_25120010
P25779	5.22	IC50	6000	nM	CHEMBL_ACT_15189974
P25779	5.22	IC50	6000	nM	CHEMBL_ACT_3307702
HIF1A	5.1	Potency	7943	nM	CHEMBL_ACT_4130715
HIF1A	5.1	Potency	7943	nM	CHEMBL_ACT_4518992
HRH1	5.07	AC50	8563	nM	CHEMBL_ACT_25213058
DRD2	5.05	AC50	8961	nM	CHEMBL_ACT_25140878

Target pathways

Aggregated over 1 target gene(s): ZC3H14.

Dominant GO biological processes

GO term	Targets
spermatogenesis	1
cell differentiation	1
regulation of mRNA stability	1
mRNA stabilization	1
spliceosome-depend formation of circular RNA	1

Indications & clinical

Indications

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

Indication	Phase	MONDO	EFO
leprosy	4	MONDO:0005124	EFO:0001054

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
pulmonary tuberculosis	3	MONDO:0006052	EFO:1000049
Mycobacterium avium complex disease	3	MONDO:0005866	EFO:0007386
Crohn disease	3	MONDO:0005011	EFO:0000384
tuberculosis	3	MONDO:0018076	MONDO:0018076
multidrug-resistant tuberculosis	2	MONDO:0005861	EFO:0007381
cryptosporidiosis	2	MONDO:0015474	MONDO:0015474
bacterial infectious disease	2	MONDO:0005113	EFO:0000771
lung disorder	2	MONDO:0005275	EFO:0003818

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

Clinical trials

Total trials: 34.

Phase distribution

Phase	Trials
PHASE2	10
Not specified	9
PHASE3	6
PHASE2/PHASE3	5
PHASE4	3
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00513552	PHASE4	WITHDRAWN	Treatment of Crohn’s Disease With an Antibiotic Regimen Directed Against Mycobacterium Avium Paratuberculosis
NCT01290744	PHASE4	COMPLETED	Effect of Additional Clofazimine on Erythema Nodosum Leprosum (ENL) Reactions in Leprosy
NCT05306223	PHASE4	UNKNOWN	A Study of an Oral Short-course Regimen Including Bedaquiline for the Treatment of Participants With Multidrug-resistant Tuberculosis in China
NCT04310930	PHASE2/PHASE3	RECRUITING	Finding the Optimal Regimen for Mycobacterium Abscessus Treatment
NCT06081361	PHASE3	ACTIVE_NOT_RECRUITING	Innovating Shorter, All- Oral, Precised, Individualized Treatment Regimen for Rifampicin Resistant Tuberculosis：Contezolid, Delamanid and Bedaquiline Cohort
NCT06649721	PHASE3	ACTIVE_NOT_RECRUITING	Innovating Shorter, All- Oral, Precised Treatment Regimen for Rifampicin Resistant Tuberculosis：BDLL Chinese Cohort
NCT00001047	PHASE3	COMPLETED	Study of Four Different Treatment Approaches for Patients Who Have Mycobacterium Avium Complex Disease (MAC) Plus AIDS
NCT02409290	PHASE3	COMPLETED	The Evaluation of a Standard Treatment Regimen of Anti-tuberculosis Drugs for Patients With MDR-TB
NCT02589782	PHASE2/PHASE3	COMPLETED	Pragmatic Clinical Trial for a More Effective Concise and Less Toxic MDR-TB Treatment Regimen(s)
NCT02754765	PHASE3	COMPLETED	Evaluating Newly Approved Drugs for Multidrug-resistant TB
NCT03474198	PHASE2/PHASE3	COMPLETED	Two-month Regimens Using Novel Combinations to Augment Treatment Effectiveness for Drug-sensitive Tuberculosis
NCT04062201	PHASE3	COMPLETED	Building Evidence for Advancing New Treatment for Rifampicin Resistant Tuberculosis (RR-TB) Comparing a Short Course of Treatment (Containing Bedaquiline, Delamanid and Linezolid) With the Current South African Standard of Care
NCT04081077	PHASE2/PHASE3	UNKNOWN	PRACTECAL-PKPD Sub Study
NCT04207112	PHASE2/PHASE3	COMPLETED	Economic Evaluation of New MDR TB Regimens
NCT02968212	PHASE2	ACTIVE_NOT_RECRUITING	Clofazimine in the Treatment of Pulmonary Mycobacterium Avium Complex (MAC)
NCT03828201	PHASE2	ACTIVE_NOT_RECRUITING	Efficacy and Tolerability of Bedaquiline, Delamanid, Levofloxacin, Linezolid, and Clofazimine to Treat MDR-TB
NCT05556746	PHASE2	ACTIVE_NOT_RECRUITING	Ultra-Short Course Bedaquiline, Clofazimine, Pyrazinamide and Delamanid Versus Standard Therapy for Drug-Susceptible TB
NCT07485010	PHASE2	NOT_YET_RECRUITING	Testing a Novel Combination Treatment (Arm D) Versus Standard of Care for Intensive Phase Treatment for Mycobacterium Abscessus Pulmonary Disease in People With or Without Cystic Fibrosis in the Finding the Optimal Regimen for Mycobacterium Abscessus Treatment (FORMaT) Adaptive Platform Trial
NCT00000641	PHASE2	COMPLETED	A Phase II/III Trial of Rifampin, Ciprofloxacin, Clofazimine, Ethambutol, and Amikacin in the Treatment of Disseminated Mycobacterium Avium Infection in HIV-Infected Individuals.
NCT03341767	PHASE2	TERMINATED	A Phase 2A Evaluation of the Safety, Tolerability, Pharmacokinetics, Efficacy of Clofazimine (CFZ) in Cryptosporidiosis
NCT04311502	PHASE2	TERMINATED	Clofazimine- and Rifapentine-Containing Treatment Shortening Regimens in Drug-Susceptible Tuberculosis: The CLO-FAST Study
NCT04465695	PHASE2	UNKNOWN	Dual Therapy With Interferon Beta-1b and Clofazimine for COVID-19
NCT05007821	PHASE2	COMPLETED	Linezolid Dosing Strategies in Drug-Resistant TB
NCT05294146	PHASE2	COMPLETED	Pharmacokinetic Study With a Loading Dose of Clofazimine in Adult Patients With Nontuberculous Mycobacterial Disease
NCT07228702	PHASE1	ENROLLING_BY_INVITATION	Bacteriophage Therapy for Mycobacterium Abscessus Pulmonary Infection
NCT00018044	Not specified	RECRUITING	Study of Mycobacterial Infections
NCT04334070	Not specified	AVAILABLE	Lamprene Multiple Patient Program
NCT07126639	Not specified	NOT_YET_RECRUITING	A Study on the Short-course Treatment Regimen Containing Pretomanid for Drug-resistant Tuberculosis
NCT07198685	Not specified	NOT_YET_RECRUITING	6 Months of Bedaquiline(BDQ), Delamanid(DLM), Linezolid(LZD) and Levofloxacin(LFX) in RR-TB Patients in Hubei Province
NCT00000796	Not specified	COMPLETED	A Prospective Study of Multidrug Resistance and a Pilot Study of the Safety of and Clinical and Microbiologic Response to Levofloxacin in Combination With Other Antimycobacterial Drugs for Treatment of Multidrug-Resistant Pulmonary Tuberculosis (MDRTB) in HIV-Infected Patients.
NCT00002058	Not specified	COMPLETED	A Randomized Controlled Prophylactic Study of Clofazimine To Prevent Mycobacterium Avium Complex Infection in HIV Disease
NCT00002331	Not specified	COMPLETED	The Safety and Effectiveness of Clarithromycin Plus Ethambutol Used With or Without Clofazimine in the Treatment of MAC in Patients With AIDS
NCT03942354	Not specified	COMPLETED	Patient-reported Experiences and Quality of Life Outcomes in the TB-PRACTECAL Clinical Trial
NCT04717908	Not specified	COMPLETED	Refining MDR-TB Treatment (T) Regimens (R) for Ultra(U) Short(S) Therapy(T)-PLUS

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

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).