Table 1.

Summary of the main pre-registration studies described in the text.

NCT Number [Ref]	Type of Study	Therapeutic Scheme	Population	Outcome	Adverse Events
NCT00570765 [31]	Phase II study, 3-month randomized, double-blind, placebo-controlled, parallel group phase, followed by a long-term safety extension (LTSE)	OCA monotherapy (10 or 50 mg)	60 PBC patients (18–70 years)	ALP reduction at both dosages after a 3-month treatment. Improvement of GGT, ALT, conjugated bilirubin, IgG	Pruritus (placebo 35%, OCA 10 70%, 94% OCA 50
NCT01473524 [32,33]	Phase III study, international 12-month randomized, double-blind (DB), placebo-controlled, parallel group phase, followed by a long-term safety extension (LTSE) phase of up to 5 years	OCA 5 mg (6 months) up to 10 mg or 10 mg vs. placebo	217 patients (≥18 years)	ALP reduction only after 12-month treatment with combination Reduction in total and direct bilirubin	Pruritus (56% in the 5–10% group and 68% in the 10 mg group vs. 38% placebo
NCT03253276 [38]	Early phase I, double-blind placebo-controlled crossover study	OCA vs. placebo	8 PBC patients	OCA reduced the time hepatocytes are exposed to potentially cytotoxic bile acids.	1 patient dropped for pruritus
NCT00550862 [39]	Phase II, randomized, double-blind study	OCA (10, 25, 50 mg) plus UDCA combination	165 patients (18–75 years)	Significant reduction in ALP, γ-GT, and ALT compared with placebo, in patients with PBC experiencing an inadequate response to UDCA	13% discontinuation for pruritus