Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
- 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
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 116
Not provided
Not provided
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