Full results & analysis
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
INDIGO: Maralixibat in PFIC
NCT02057718Phase 2
Key facts
- Status
- Completed
- Start
- Mar 2014
- Primary completion
- May 2020
- Countries
- 4
- Sponsor
- Mirum Pharmaceuticals, Inc.
- Modality
- Small Molecules
- Drugs
- maralixibat
- Conditions
- progressive familial intrahepatic cholestasis (PFIC)
01
Overview
Phase 2, open-label, single-arm trial of maralixibat in progressive familial intrahepatic cholestasis (PFIC), sponsored by Mirum Pharmaceuticals, Inc. Primary endpoint: Change From Baseline to Endpoint in Fasting Serum Bile Acid Level at 13 weeks. Started Mar 2014; status: completed.
02
Design
1 arm- Study design
- Non-randomizedOpen-label
LUM001 (Maralixibat)
Participants receive LUM001 (Maralixibat) twice a day (BID).
03
Who it enrols
Pediatric patients (1-18 years) with a diagnosis of PFIC, elevated serum bile acids (>3x ULN), and low GGTP (<100 IU/L). Excludes patients with decompensated cirrhosis, prior liver transplant, or surgical disruption of enterohepatic circulation.
04
Endpoints
Primary
Change From Baseline to Endpoint in Fasting Serum Bile Acid Level
Secondary
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)
Change From Baseline to Week 13/ET in ALT
Change From Baseline to Week 13/ET in Total Bilirubin
Change From Baseline to Week 13/ET in Direct Bilirubin
Beyond the preview
Go deeper on NCT02057718.
Full results, competitive context and analysis are in the Gosset platform.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NCT02057718.