CompletedPhase 3

This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2

United States62 participantsStarted 2018-05-16

Plain-language summary

Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC) types 1 and 2.

Who can participate

Age range6 Months – 18 Years

SexALL

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Exclusion criteria

✕. Biliary atresia of any kind
✕. Benign recurrent intrahepatic cholestasis, indicated by any history of normal s BAs
✕. Suspected or proven liver cancer or metastasis to the liver on imaging studies
✕. Histopathology on liver biopsy that is suggestive of alternate non-PFIC related etiology of cholestasis

What they're measuring

Percentage of Participants With at Least a 70% Reduction in Fasting Serum Bile Acid (s-BA) Concentration From Baseline to the End of Treatment or Reaching a Level <=70 Micromoles Per Liter (Mcmol/L) After 24 Weeks of Treatment

Timeframe: From Baseline (Day 1) up to Week 24

Percentage of Positive Pruritus Assessments at the Participant Level Based on the Albireo Observer-Reported Outcome (ObsRO) Instrument Over the 24-Week Treatment Period

Timeframe: From Baseline (Day 1) up to Week 24

Trial details

NCT IDNCT03566238

SponsorAlbireo

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2020-07-27

Results submitted2021-07-12

View on ClinicalTrials.gov More PFIC1 trials