Full results & analysis
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Odevixibat in PFIC Types 1 and 2
NCT03566238Phase 3
Key facts
- Status
- Completed
- Start
- May 2018
- Primary completion
- Jul 2020
- Countries
- 15
- Sponsor
- Albireo Pharma
- Modality
- Small Molecules
- Drugs
- odevixibat
- Conditions
- progressive familial intrahepatic cholestasis type 1 (PFIC1)progressive familial intrahepatic cholestasis type 2 (PFIC2)
01
Overview
Phase 3, randomised, double-blind trial of odevixibat in progressive familial intrahepatic cholestasis type 1 (PFIC1) and progressive familial intrahepatic cholestasis type 2 (PFIC2) (3 arms), sponsored by Albireo Pharma. Primary endpoints: Percentage of Participants With at Least a 70% Reduction in Fasting Serum Bile Acid Concentration From Baseline to the End of Treatment or Reaching a Level ≤70 Micromoles Per Liter After 24 Weeks of… and Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome Instrument Over the 24-Week Treatment Period. Started May 2018; status: completed.
02
Design
3 arms- Study design
- RandomizedDouble-blind
A4250 low dose
Odevixibat 40 µg/kg once daily for 24 weeks
A4250 high dose
Odevixibat 120 µg/kg once daily for 24 weeks
Placebo
Matching placebo once daily for 24 weeks
03
Who it enrols
Children with genetically confirmed PFIC type 1 or 2, body weight >5 kg, elevated serum bile acids, history of pruritus, without complete BSEP protein absence, other liver diseases, chronic diarrhea, recent biliary surgery, planned transplant, or decompensated liver disease
04
Endpoints
Primary
Percentage of Participants With at Least a 70% Reduction in Fasting Serum Bile Acid Concentration From Baseline to the End of Treatment or Reaching a Level ≤70 Micromoles Per Liter After 24 Weeks of…
Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome Instrument Over the 24-Week Treatment Period
Secondary
Change From Baseline in Fasting Serum Bile Acid
Change From Baseline in Serum Alanine Aminotransferase Concentration
Change From Baseline in Growth Parameters
Percentage of Responders for Pruritus Assessments Based on Bi-Weekly and Monthly Scores Using the Albireo Observer-Reported Outcome Instrument
Change From Baseline in Sleep Parameters Based on the Albireo Observer-reported Outcome Instrument
Change From Baseline in Sleep Parameters Based on the Albireo Patient-Reported Outcome Instrument
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo ObsRO Instrument
Percentage of Individual Assessments Meeting the Definition of a Positive Pruritus Assessment at the Participant Level Using the Albireo PRO Instrument
05
Results
Primary endpoints met
Beyond the preview
Go deeper on NCT03566238.
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 NCT03566238.