Table 2.

Principal characteristics of the study of the drugs in clinical trials.

	Study	Phase	Pt Number	Dose	Study Duration	Primary Endpoint	Primary Endpoint Met	Note
24-norursodeoxycholic acid (norUDCA)	[61]	2	161	500 mg, 1 g, 1.5 gr	16 weeks	Change in ALP	yes
	NCT03872921	3	300 *	250 mg 6 cps/d	2 years	Change in ALP and histology	n/a	ongoing
FXR agonist (drugs)
OCA	[75]	2	77	1.5–3 mg 5–10 mg	24 weeks	Change in ALP	yes	5–10 mg
Cilofexor	[76]	2	52	100 mg 30 mg	12 weeks	Safety and liver enzyme improvement	yes
NGM282	[77]	2	62	1 mg 3 mg	12 weeks	Change in ALP	no
ATRA	[78]	Pilot study	15	45 mg/m/d	12 weeks	ALP < 30% from baseline	no	Decrease in ALT and C4
	NCT03359174	2	2	10 mg bd	24 weeks	Change in ALP	n/a	ongoing
PPAR agonists
Bezafibrates	[79]	2	11	200 mg BID	12 weeks	improvements in liver function test	yes
Bezafibrates	[79]	2	11	200 mg BID	12 weeks	improvements in liver function test	yes
Antifibrotic therapy (drugs)
Simtuzumab	[80]	2	234	75 mg, 125 mg	96 weeks	Hepaticcollagencontent	no
Immunomodulator (drugs)
Timolumab	NCT02239211	2	23	8 mg/kg	11 weeks	ALP < 25% from baseline	n/a	Awaiting results
Cenicriviroc	NCT02653625	Open label	24	150 mg	24 weeks	Change in ALP	yes
Vedolizumab	[81]	Retrospective	102		412 days (median)		no	ALP < 20% from baseline
Vidofludimus	NCT03722576	2	14	30 mg	6 months	Change in ALP	n/a	Awaiting results
Modulation of gut microbioma (drugs)
Vancomycin	NCT03710122	2/3	102 *		24 months	Change in ALP	n/a	ongoing
Rifaximin	[82]	Open label	16	550 mg bd	12 weeks	Change in ALP	no
Minocycline	[83]	Pilot study	16	100 mg bd	1 year	Change in biochemistry	yes
FMT	[84]	Open label	10		24 weeks	safety	yes
Other treatments (drugs)
Sulfasalazine	NCT03561584	2	42	500 mg bd	14 weeks	Change in ALP	n/a	ongoing
Curcumin	[85]	Open label	258	750 mg bd	12 weeks	ALP < 1.5 ULN or <40% from baseline	no
HTD1801	NCT03333928	2	59	500 mg 1 gr	18 weeks	Change in ALP	n/a	Awaiting results
DUR-928	NCT03394781	2	5	10 mg 50 mg	28 days	Change in ALP	n/a	ongoing
Docosahexaenoic acid	[86]	Open label	23	800 mg bd	12 months	Change in ALP and safety	yes
Hymecromone	NCT02780752	1	18 *	1.2 gr 2.4 gr 3.6 gr	4 days	Change in spu	n/a	ongoing
Orbcel-C	NCT02997878	2	56 *	0.5, 1.0, 2.5 million cells/kg	56 days	Safety, change in ALP e ALT	n/a	ongoing

* estimated.