Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC

Phase 3

Active, not recruiting

• Conditions: Progressive Familial Intrahepatic Cholestasis
• Interventions: Drug: A4250 (odevixibat)

• Registration Number: NCT03659916
• Lead Sponsor: Albireo, an Ipsen Company

Brief Summary

Open Label Extension Study to evaluate long term safety and persistence of effect of A4250 in children with PFIC.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-08-24
• Study First Submitted QC: 2018-09-04
• Study First Posted: 2018-09-06
• Study Start: 2018-09-28
• Primary Completion: 2024-02-15
• Status Verified: 2024-10
• Last Update Submitted: 2024-10-30
• Last Update Posted: 2024-10-31
• Study Completion: 2025-03-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 116

Inclusion Criteria

Not provided

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
A4250	A4250 (odevixibat)	Capsules for oral administration (40 or 120 µg/kg) once daily for 72 weeks, or 40 µg/kg/day for the first 12 weeks followed by 120 µg/kg/day for the remaining 60 weeks"

Primary Outcome Measures

Name	Time	Method
US: Change in Pruritus	From baseline over 72 weeks	Change in pruritus as indexed by caregiver-reported (Albireo ObsRO instrument) observed scratching
EU and rest of world: Change in serum bile acids	From baseline up to week 72

Secondary Outcome Measures

Name	Time	Method
All regions. All-cause mortality	From baseline to weeks 24, 48, and 72
EU and rest of world: Proportion of positive pruritus assessments at the patient level over the 72-week treatment period using the Albireo ObsRO instrument.	72 weeks
US: Change from baseline in serum bile acids after 72 weeks of treatment.	72 weeks
All regions: Proportion of individual assessments meeting the definition of a positive pruritus assessment at the patient level using the Albireo ObsRO instrument.	weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, 0-70, or the proportion of positive pruritus assessments at each 4-week interval between V1/Screening and V5/Week 24, then by each visit between V5/Week 24 to V12/Week 76
All regions: Proportion of individual PM assessments meeting the definition of a positive pruritus assessment at the patient level using the Albireo ObsRO instrument	weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, 0-70, or the proportion of positive pruritus assessments at each 4-week interval between V1/Screening and V5/Week 24, then by each visit between V5/Week 24 to V12/Week 76
All regions: Number of patients undergoing biliary diversion surgery	From baseline to weeks 24, 48, and 72
All regions: Number of patients undergoing liver transplantation	From baseline to weeks 24, 48, and 72
All regions: Change in growth	From baseline to weeks 24, 48, and 72	The linear growth deficit compared to the standard growth curve
All regions: Change in AST to platelet ratio idex (APRI) score	From baseline to week 72
All regions: 3. Change from baseline in serum bile acids at Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, 72, and 76	Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, 72, and 76
All regions: Proportion of individual AM assessments meeting the definition of a positive pruritus assessment at the patient level using the Albireo ObsRO instrument	weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, 0-70, or the proportion of positive pruritus assessments at each 4-week interval between V1/Screening and V5/Week 24, then by each visit between V5/Week 24 to V12/Week 76
All regions: Change in Fib-4 score	From baseline to week 72
All regions: Change in pediatric end-stage liver disease (PELD)/model for end-stage liver disease (MELD) score	From baseline to week 72
All regions: Change in use of antipruritic medication	From baseline to weeks 24, 48, and 72

Trial Locations

Locations (41)

Children's Hospital Los Angeles; 🇺🇸 Los Angeles, California, United States

Emory University School of Medicine; 🇺🇸 Atlanta, Georgia, United States

Johns Hopkins School of Medicine; 🇺🇸 Baltimore, Maryland, United States

Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Akdeniz University; 🇹🇷 Antalya, Turkey

Riley Hospital for Children - Riley Children's Specialists; 🇺🇸 Indianapolis, Indiana, United States

Icahn School of Medicine at Mount Sinai; 🇺🇸 New York, New York, United States

Columbia University Medical Center - Presbyterian Hospital Building; 🇺🇸 New York, New York, United States

Cliniques Universitaires Saint-Luc; 🇧🇪 Woluwe-Saint-Lambert, Belgium

Baylor College of Medicine - Texas Children's Liver Center; 🇺🇸 Houston, Texas, United States

The Royal Children's Hospital; 🇦🇺 Melbourne, Australia

University and Pediatric Hospital of Lyon; 🇫🇷 Bron, France

Universite Paris SUD - Hpitaux Universitaires Paris-Sud - Hopital Bicetre; 🇫🇷 Le Kremlin-Bicêtre, France

Hospital De La Timone; 🇫🇷 Marseille, France

Hospital Necker-Enfants Maladies; 🇫🇷 Paris, France

Medizinische Hochschule Hannover; 🇩🇪 Hannover, Germany

Kinderklinik Tubingen, Universitatsklinikum Tubingen; 🇩🇪 Tübingen, Germany

Univesitatsklinikum Tubingen Klinik fur Kinder und Jugendmedizin; 🇩🇪 Tübingen, Germany

University Medical Center Groningen; 🇳🇱 Groningen, Netherlands

King Faisal Specialist Hospital & Research Centre; 🇸🇦 Riyadh, Saudi Arabia

Hospital Universitari Vall d'Hebron; 🇪🇸 Barcelona, Spain

Instytut Pomnik - Centrum Zarowia Dziecka; 🇵🇱 Warsaw, Poland

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain

Astrid Lindgren Children's Hospital, Karolinska University Hospital; 🇸🇪 Solna, Sweden

Gazi University; 🇹🇷 Ankara, Turkey

Hacettepe University Faculty of Medicine; 🇹🇷 Ankara, Turkey

Istanbul University Medical Faculty; 🇹🇷 Istanbul, Turkey

Inonu University Medical Faculty; 🇹🇷 Malatya, Turkey

Leeds General Infirmary; 🇬🇧 Leeds, United Kingdom

Birmingham Women's and Children's NHS Foundation Trust; 🇬🇧 Birmingham, United Kingdom

Institute of Liver Studies - Kings College Hospital; 🇬🇧 London, United Kingdom

Universitair Medisch Centrum (UMC) Utrecht; 🇳🇱 Utrecht, Netherlands

Schneider Children's Medical Center Of Israel; 🇮🇱 Petach-Tikva, Israel

Shaare-Zedek Mc; 🇮🇱 Jerusalem, Israel

Azienda Ospedaliera Papa Giovanni XXIII; 🇮🇹 Bergamo, Italy

University Hospital Of Padova; 🇮🇹 Padova, Italy

Ospedale Regina Margherita; 🇮🇹 Torino, Italy

Children's Hospital Colorado; 🇺🇸 Denver, Colorado, United States

The Hospital for Sick Children; 🇨🇦 Toronto, Canada

Washington University School of Medicine; 🇺🇸 Saint Louis, Missouri, United States