Table 2.
Ongoing clinical trials recruiting patients for post-registration efficacy assessment.
| NCT Number | Type of Study | Therapeutic Scheme | Estimated Enrollment | Primary Endpoints |
|---|---|---|---|---|
| NCT05450887 | Randomized, double-blind, multicenter, placebo-controlled phase III clinical trial | OCA (5 mg titrated to 10 mg) ± UDCA vs. placebo ± UDCA (13~15 mg/kg/day) | 156 PBC patients (18–75 years) |
Percentage of PBC patients reaching ALP < 1.67× Upper Limit of Normal, and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN after 12-month treatment |
| NCT03703076 | Post-authorization non-interventional observational, multi-site study | OCA (5 or 10 mg) | 150 patients | Response to Ocaliva® after 12-month treatment (monotherapy or combination) assessed by Paris II response criteria |
| NCT05293938 | Retrospective study | OCA (5 or 10 mg) and UDCA | 2544 participants | Time to the first occurrence of the composite endpoint of all-cause death, liver transplant, or hospitalization for hepatic decompensation after 67 months |
|
NCT05292872 (HEROES PBC) |
Retrospective study | OCA (5 or 10 mg) and UDCA | 3156 participants | Time to the first occurrence of all-cause death, liver transplant, or hospitalization for hepatic decompensation after 67 months |
| NCT05239468 | Phase IIa, double-blind, randomized, active-controlled, parallel group study | Bezafibrate 100 or 200 mg, OCA 5 mg, Bezafibrate placebo, OCA placebo |
60 patients | ALP change after 12 weeks vs. baseline |
| NCT04594694 | Phase II, double-blind, randomized, parallel group study | Bezafibrate 200 or 400 mg, OCA mg, Bezafibrate placebos, OCA placebo |
75 patients | ALP change after 12 weeks vs. baseline |
| NCT04076527 | Prospective, multicenter cohort study | OCA vs. UDCA | 1200 patients | Construction of a systematic registry to describe the characteristics and the recent state of usual clinical care of the respective population |
| NCT04956328 | Multicenter, randomized, double-blind trial | OCA (5 to 10 mg) + UDCA, or placebo + UDCA | 120 patients | Percentage of PBC patients reaching ALP < 1.67× ULN, and ALP decrease ≥ 15% from baseline, and total bilirubin ≤ ULN after 48week-treatment |