Table 1 Drug targets and NCT number of clinical trials

From: Targeting fibrosis: mechanisms and clinical trials

Target		Drug Name	Conditions	Highest Status (phase)	NCT	Status	Sample size
TGF-β/TGFβR	TGF-βs	Pirfendione	IPF	Marketed	NCT00662038	Completed	1058
	p38 MAPK, TGFβ1, FGFR1	Hydronidone	Liver fibrosis	III	NCT05115942	Recruiting	248
	TGFβR1	HEC-585	IPF	II	NCT05060822	Recruiting	270
	αV/β1, αV/β6	PLN-74809	IPF	II	NCT04396756	Recruiting	112
	αV/β6, TGF-β	BG00011	IPF	II	NCT03573505	Terminated	109
	αV/β1, αV/β3, αV/β6	IDL-2965	IPF	I	NCT03949530	Terminated	6
	TGF-β1	TRK-250	IPF	I	NCT03727802	Completed	34
	TGF-βs	Luspatercept	Myelofibrosis	III	NCT04717414	Recruiting	309
	TGF-β, BMPRII	Sotatercept	Myelofibrosis	II	NCT01712308	Completed	63
	TGF-β1 and TGF-β3	AVID200	Myelofibrosis	I	NCT03895112	Active, not recruiting	22
FGF	FGF21	BIO89-100	NASH	II	NCT04048135	Active, not recruiting	101
	FGF21	Efruxifermin	NASH	II	NCT03976401	Completed	110
	FGF21	Pegbelfermin	NASH	II	NCT02413372	Completed	184
	FGF19	Aldafermin	NASH	II	NCT03912532	Completed	171
RTKs	PDGFRs, FGFRs, VEGFRs	Nintedanib	IPF	Marketed	NCT02598193	Completed	89
	PDGFRα, β, FGFR1-4, and VEGFR1-3	ZSP1603	IPF	II	NCT05119972	Recruiting	36
	β-Klotho/FGFR1c receptor complex	MK-3655	NASH	II	NCT04583423	Recruiting	328
CTGF	CTGF	Pamrevlumab	IPF	III	NCT03955146	Recruiting	340
PI3K	PI3Kδ	Parsaclisib	Myelofibrosis	III	NCT04551053	Recruiting	212
	PI3K/mTOR	Omipalisib	IPF	I	NCT01725139	Completed	17
	PI3K/mTOR	HEC-68498	IPF	I	NCT03502902	Completed	55
	PI3K p110α/β/δ/γ	Buparlisib	Myelofibrosis	I	NCT01730248	Terminated	63
	PI3Kδ, CK1-epsilon	Umbralisib	Myelofibrosis	I	NCT02493530	Active, not recruiting	60
JAK	JAK1/2	Ruxolitinib	Myelofibrosis	Marketed	NCT02386800	Recruiting	356
	JAK2, FLT3	Fedratinib	Myelofibrosis	III	NCT03755518	Active, not recruiting	110
	JAK1/2, TBK1, ACVR1/ALK2	Momelotinib	Myelofibrosis	III	NCT04173494	Active, not recruiting	195
	JAK2, FLT3, IRAK1	Pacritinib	Myelofibrosis	III	NCT03165734	Recruiting	348
	JAK1/2/3	Jaktinib	Myelofibrosis	III	NCT04617028	Recruiting	105
	JAK1	Itacitinib	Myelofibrosis	II	NCT04640025	Recruiting	100
	JAK2	Ilginatinib	Myelofibrosis	II	NCT01423851	Completed	77
WNT/β-catenin	WNT	SM04646	IPF	II	NCT03591926	Withdrawn	0
	β-catenin	PRI-724	liver cirrhosis	II	NCT03620474	Completed	27
ASK, MAPK	ASK1, MAPKKK5	Selonsertib	NASH	III	NCT03053050	Terminated	808
	JNK1, MAPK8	CC-90001	NASH	II	NCT04048876	Terminated	56
	MAP3K19	MG-S-2525	IPF	I	NCT03650075	Completed	81
LOXL	LOXL2, LTD4 receptor, PDE3 /4	Epeleuton	NAFLD	II	NCT02941549	Completed	96
	LOXL2, LTD4 receptor, PDE3 /4	Tipelukast	IPF	II	NCT02503657	Completed	15
	LOXL2	PAT-1251	Myelofibrosis	II	NCT04054245	Withdrawn	0
	LOXL2	PXS-5382A	IPF, NASH	I	NCT04183517	Completed	18
PPAR	PPAR α/δ	Elafibranor	NASH	III	NCT02704403	Terminated	2157
	PPAR α/γ	Saroglitazar	NASH	III	NCT04193982	Recruiting	250
	PPAR α/δ/γ	Lanifibranor	NASH	III	NCT04849728	Recruiting	2000
	PPAR α	Pemafibrate	NASH	II	NCT03350165	Completed	118
	PPARα/δ	ZSP0678	NASH	I	NCT04137055	Completed	104
FXR	FXR	Obeticholic Acid	NASH	III	NCT02548351	Active, not recruiting	2480
	FXR	Cilofexor	Liver fibrosis, NASH	II	NCT02854605	Completed	140
	FXR	Nidufexor	NASH	II	NCT02913105	Terminated	122
	FXR	TERN-101	NASH	II	NCT04328077	Completed	101
	FXR	Vonafexor	NASH	II	NCT03812029	Completed	120
	FXR	EDP-305	NASH	II	NCT04378010	Recruiting	336
	FXR	Tropifexor	NASH	II	NCT04147195	Terminated	41
TLR	TLR4	JKB-121	NASH	II	NCT02442687	Completed	65
	TLR4	JKB-122	NASH	II	NCT04255069	Active, not recruiting	300
GLP/GIP	GLP-1 receptor	Semaglutide	NASH	III	NCT04822181	Recruiting	1200
	GLP-1/GIP receptor	Tirzepatide	NASH	II	NCT04166773	Recruiting	196
	GLP-1/Glucagon receptor	Cotadutide	NASH	II	NCT05364931	Active, not recruiting	1860
	GLP-1/GIP/Glucagon	HM-15211	NASH	II	NCT04505436	Recruiting	217
CFTR	CFTR	Elexacaftor	Cystic fibrosis	III	NCT03525444	Completed	405
	CFTR	Ivacaftor	Cystic fibrosis	III	NCT01707290	Completed	125
	CFTR	GLPG1837	Cystic fibrosis	II	NCT02707562	Completed	26
	CFTR	FDL169	Cystic fibrosis	II	NCT02767297	Completed	46
	CFTR	Olacaftor	Cystic fibrosis	II	NCT02951182	Completed	74
	CFTR	VX-152	Cystic fibrosis	II	NCT02951195	Completed	80
	CFTR	MRT5005	Cystic fibrosis	II	NCT03375047	Recruiting	40
	CFTR	GLPG2737	Cystic fibrosis	II	NCT03474042	Completed	22
	CFTR	Nesolicaftor	Cystic fibrosis	II	NCT03591094	Completed	40
	CFTR	VX-121	Cystic fibrosis	II	NCT03912233	Completed	87
	CFTR	ABBV-3067	Cystic fibrosis	II	NCT03969888	Active, not recruiting	189
	CFTR	ELX-02	Cystic fibrosis	II	NCT04135495	Recruiting	16
	CFTR	Eluforsen	Cystic fibrosis	II	NCT02532764	Completed	70
	CFTR	Dirocaftor	Cystic fibrosis	II	NCT03251092	Completed	179
	CFTR	FDL176	Cystic fibrosis	I	NCT03173573	Completed	109
	CFTR	Posenacaftor	Cystic fibrosis	I	NCT03140527	Completed	171
	CFTR	GLPG2451	Cystic fibrosis	I	NCT02788721	Completed	31
HDAC	HDAC	Panobinostat	Myelofibrosis	Marketed	NCT02386800	Recruiting	356
	HDAC	Pracinostat	Myelofibrosis	II	NCT01200498	Completed	23
THRβ	THRβ	Resmetirom	NASH	III	NCT03900429	Recruiting	2000
	THRβ	VK2809	NASH	II	NCT04173065	Recruiting	337
CCR	CCR2/CCR5	Cenicriviroc	NASH	III	NCT03028740	Terminated	1778
Galectin	Galectin-3	Belapectin	NASH	III	NCT04365868	Recruiting	1010
	Galectin-3	GB1211	NASH	II	NCT04607655	withdrawn	0
	Galectin-3	GB0139	IPF	II	NCT03832946	Active, not recruiting	426
MPC	MPC	Azemiglitazone potassium	NASH	III	NCT03970031	Active, not recruiting	1800
	MPC	Deuterium-Stabilized (R)-Pioglitazone	NASH	II	NCT04321343	Active, not recruiting	123
SCD	SCD-1	Aramchol	NASH	III	NCT04104321	Recruiting	2000
ATX	ATX	Ziritaxestat	IPF	III	NCT03711162	Terminated	526
FATP5	FATP5	Ursodiol	Cystic Fibrosis	II	NCT00004315	Unkonwn	20
ACC	ACC1/2	PF-05221304	NASH	II	NCT03248882	Completed	305
	ACC	Firsocostat	NASH	II	NCT03449446	Completed	395
PDE	PDEs (mainly PDE2)	ZSP1601	NASH	II	NCT04140123	Completed	37
	LOXL2, LTD4 receptor, PDE3 /4	Epeleuton	NAFLD	II	NCT02941549	Completed	96
	LOXL2, LTD4 receptor, PDE3 /4	Tipelukast	IPF	II	NCT02503657	Completed	15
	PDE 3/4	Ensifentrine	Cystic fibrosis	II	NCT02919995	Completed	10
AMPK	AMPK	PXL-770	NAFLD	II	NCT03763877	Completed	121
MMP	MMP2, MMP9, VEGF-A	ALS-L1023	NASH	II	NCT04342793	Unknown	60
A3AR	A3AR	Namodenoson	NASH	II	NCT02927314	Completed	60
FASN	FASN	TVB-2640	NASH	II	NCT03938246	Completed	142
Bioidentical testosterone	Bioidentical testosterone	LPCN 1144	NASH	II	NCT04134091	Completed	56
Stem cell	Stem cell	HepaStem	NASH	II	NCT03963921	Completed	23
HSP	HSP 47	BMS-986263	NASH	II	NCT04267393	Recruiting	270
	HSP 90	PU-H71	Myelofibrosis	I	NCT03935555	Recruiting	24
CD	CD3	Foralumab	NASH	II	NCT03291249	Withdrawn	0
	CD123	Tagraxofusp	Myelofibrosis	II	NCT02268253	Recruiting	130
ileal bile acid transport	ileal bile acid transport	Elobixibat	NASH	II	NCT04006145	Completed	47
aldosterone receptor	aldosterone receptor	Apararenone	NASH	II	NCT02923154	Completed	48
GPR	GPR-35	RVT1601	IPF	II	NCT03864328	Terminated	108
	GPR-84	GLPG-1205	IPF	II	NCT03725852	Completed	68
	GPR-40, GPR-84	PBI-4050	IPF	II	NCT02538536	Completed	41
ROCK2	ROCK2	Belumosudil	IPF	II	NCT02688647	Completed	76
BAFFR	BAFFR	Ianalumab	IPF	II	NCT03287414	Terminated	30
LPA1	LPA1	BMS-986278	IPF	II	NCT04308681	Recruiting	360
Telomerase	Telomerase	Imetelstat	Myelofibrosis	III	NCT04576156	Recruiting	320
KHK	KHK	PF-06835919	NASH	II	NCT03969719	Completed	164
calpain	calpain 1, 2, and 9	BLD-2660	IPF	II	NCT04244825	Withdrawn	0
P selectin	P selectin	Crizanlizumab	Myelofibrosis	II	NCT04097821	Recruiting	243
SMO	SMO	Sonidegib	Myelofibrosis	II	NCT01787552	Completed	50
Bcl-2	Bcl-2	Navitoclax	Myelofibrosis	II	NCT03222609	Active, not recruiting	191
BET family	BET family	Pelabresib	Myelofibrosis	II	NCT02158858	Recruiting	341
ENaC	ENaC	BI-1265162	Cystic fibrosis	II	NCT04059094	Terminated	52
	ENaC	P-1037	Cystic fibrosis	II	NCT02343445	Completed	142
	ENaC	QBW276	Cystic fibrosis	II	NCT02566044	Completed	16
	ENaC	IONIS-ENaCRx	Cystic fibrosis	I	NCT03647228	Completed	98
	ENaC	AZD5634	Cystic fibrosis	I	NCT02950805	Completed	9
	ENaC	BI 443651	Cystic fibrosis	I	NCT02976519	Completed	64
	ENaC	Idelalisib	Myelofibrosis	I	NCT02436135	Terminated	10
DNase I	DNase I	AIR DNase	Cystic fibrosis	II	NCT02722122	Unkonwn	15
AA/DHA imbalance	AA/DHA imbalance	Fenretinide	Cystic fibrosis	II	NCT03265288	Completed	166
Neutrophil elastase	Neutrophil Elastase	Lonodelestat	Cystic fibrosis	II	NCT03748199	Completed	32
	Neutrophil Elastase	CHF 6333	Cystic fibrosis	I	NCT04010799	Completed	68
leukotriene B4	leukotriene B4	Acebilustat	Cystic fibrosis	II	NCT02443688	Completed	200
CDK	CDK1, CDK2/E, CDK2/A, CDK5, 7, 9	Seliciclib	Cystic fibrosis	II	NCT02649751	Terminated	49
	CDK4/6	Ribociclib	Myelofibrosis	I	NCT02370706	Completed	15
LSD	LSD1	Bomedemstat bis-tosylate	Myelofibrosis	II	NCT03136185	Completed	89
MDM2	MDM2	KRT-232	Myelofibrosis	III	NCT03662126	Recruiting	385
PLK1	PLK1	Rigosertib	Myelofibrosis	II	NCT02730884	Terminated	3
IL-1α	IL-1α	Bermekimab(MABp1)	Systemic Sclerosis	II	NCT04045743	Active, not recruiting	20
HSD17B13	HSD17B13	ARO-HSD	NASH	I	NCT04202354	Completed	50
MOTS-c	MOTS-c	CB4211	NAFLD	I	NCT03998514	Completed	88
IFN-γ	IFN-γ	Interferon gamma	IPF	I	NCT00563212	Completed	12
Autotaxin	Autotaxin	BBT-877	IPF	I	NCT03830125	Completed	88
Glutathione dependent PGD synthase	Glutathione dependent PGD synthase	ZL-2102	IPF	I	NCT02397005	Unknown	120
Arginase	Arginase	CB-280	Cystic fibrosis	I	NCT04279769	Completed	32
GSNOR	GSNOR	N-6022	Cystic fibrosis	I	NCT01746784	Completed	66
Pim kinase inhibitor	Pim-1, -2, -3 kinase	TP-3654	Myelofibrosis	II	NCT04176198	Recruiting	60
PRMT	PRMT5	PRT-543	Myelofibrosis	I	NCT03886831	Active, not recruiting	227

AA/DHA ascorbic acid/ docosahexaenoic acid, ACC acetyl-coenzyme A carboxylase, ACVR1 activin A receptor type 1, ALK2 activin receptor-like kinase 2, ATX autotoxin, A3AR A3 adenosine receptor, BET family bromodomain and extra-terminal domain family, BMPRII bone morphogenic protein receptor type II, CCR2 chemokine receptor 2, CCR5 chemokine receptor 5, CDK cyclin-dependent kinase, EnaC epithelial sodium channel, FASN fatty acid synthase, FATP5 fatty acid transport protein 5, FLT3 FMS-like tyrosine kinase 3, GSNOR S-nitrosoglutathione reductase, HDAC histone deacetylase, HSD17B13 17-beta hydroxysteroid dehydrogenase 13, HSP47 heat shock protein 47, IRAK1 Interleukin-1 receptor-associated kinases, KHK ketohexokinase, LSD1 lysine-specific demethylase 1, LTD4 leukotriene D4, MAPKKK5 MEK Kinase5, MDM2 mouse double minute 2, MMP matrix metallopeptidase, MOTS-c mitochondrial open reading frame of the 12S rRNA-c, MPC mitochondrial pyruvate carrier, NASH non-alcoholic steatosis, PDE phosphodiesterase, PLK1 polo-like kinase 1, PRMT5 protein arginine methyltransferase 5, TBK1 TANK-binding kinase 1, THRβ thyroid hormone receptor beta, SCD-1 stearoyl CoA desaturase-1, SMO Smoothened, SP-B surfactant proteins B

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Table 1 Drug targets and NCT number of clinical trials

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