Clinical trial • Phase III • Rare Disease
odevixibat for Alagille syndrome
Phase III trial of odevixibat for Alagille syndrome. open-label, none/not specified-controlled. 25 participants.
Overview
- Trial Therapeutic Area
- Rare Disease
- Trial Disease
- Alagille syndrome
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 20-12-2023
- First CTIS Authorization Date
- 07-02-2024
Trial design
open-label, none/not specified-controlled Phase III trial in Belgium, Netherlands, France and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 25
- Trial Duration For Participant
- 504
Eligibility
Recruits 25 paediatric patients.
- Vulnerable Population
- Vulnerable populations included children; informed consent and assent procedures are specified. The protocol requires "Signed informed consent and assent as appropriate." Age-specific participant information and consent/assent forms are provided (adult, parent/guardian, adolescent assent 12-17, assent 6-11, pregnant partner forms) in multiple languages (English, Dutch, French, Polish, Italian as available in site documents). Patients who reach legal adulthood during the study must re-consent to remain on study. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary).
Inclusion criteria
- {"criterion_text":"- 1. Completion of the 24-week Treatment Period of Study A4250-012\n- 2. Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study\n- 3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study\n- 4. Sexually active males and females must agree to use a reliable contraceptive method with ≤ 1% failure rate (such as intra-uterine device or complete abstinence) from signed informed consent through 90 days after last dose of study drug."}
Exclusion criteria
- {"criterion_text":"- 1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy\n- 2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012\n- 3. Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study\n- 4. Known hypersensitivity to any components of odevixibat"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.","definition_or_measurement_approach":"Change from baseline in scratching measured through Week 72 using the Albireo ObsRO (observer-reported outcome) caregiver instrument."}
Secondary endpoints
- {"endpoint_text":"- • Change in serum bile acid levels from baseline to Week 72;","definition_or_measurement_approach":"Change in serum bile acid levels from baseline to Week 72 (laboratory measurement of serum bile acids)."}
- {"endpoint_text":"- • Change from baseline through Week 72 in patient reported and observer reported itching and scratching severity scores, respectively, for the morning and evening assessment;","definition_or_measurement_approach":"Change from baseline through Week 72 in PRO and ObsRO itching and scratching severity scores for morning and evening assessments (patient- and observer-reported instruments)."}
- {"endpoint_text":"- • Percentage of patients achieving a clinically meaningful decrease in pruritus (pruritus responders) at each visit as measured by the Albireo ObsRO/patient reported outcomes (PRO) instruments;","definition_or_measurement_approach":"Proportion of patients meeting predefined clinically meaningful decrease in pruritus at each visit as assessed by Albireo ObsRO and PRO instruments."}
- {"endpoint_text":"- • Change from baseline to Week 72 in sleep parameters as measured with the Albireo ObsRO/PRO instruments (e.g. tiredness and number of awakenings);","definition_or_measurement_approach":"Change from baseline to Week 72 in sleep-related parameters (e.g. tiredness, number of awakenings) measured using Albireo ObsRO/PRO instruments."}
- {"endpoint_text":"- • Change from baseline to Week 72 in Pediatric Quality of Life Inventory (PedsQL) scores","definition_or_measurement_approach":"Change from baseline to Week 72 in PedsQL (Pediatric Quality of Life Inventory) total and domain scores."}
- {"endpoint_text":"- • Assessment of Global Symptom Relief from baseline to Weeks 4, 12, 24, 48, and 72 as measured by patient, caregiver, and clinician Global impression of Symptoms (PGIS, CaGIS, CGIS) items","definition_or_measurement_approach":"Global symptom relief assessed at Weeks 4, 12, 24, 48, and 72 using PGIS, CaGIS, and CGIS (patient, caregiver, clinician global impression of symptoms)."}
- {"endpoint_text":"- • Assessment of Global Symptom Relief as measured by patient, caregiver, and clinician Global Impression of Change (PGIC, CaGIC, CGIC) items at Weeks 4, 12, 24, 48, and 72","definition_or_measurement_approach":"Global impression of change assessed at Weeks 4, 12, 24, 48, and 72 using PGIC, CaGIC, and CGIC instruments."}
- {"endpoint_text":"- • Change in serum bile acid levels from baseline through Week 72","definition_or_measurement_approach":"Change in serum bile acid levels from baseline through Week 72 (laboratory measurement)."}
Recruitment
- Planned Sample Size
- 25
- Recruitment Window Months
- 63
- Consent Approach
- Signed informed consent required; assent obtained where appropriate. Specific mention: "Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study." Age-specific information and consent/assent forms are provided (adult, parent/guardian, 12-17y assent, 6-11y assent), and documents are available in multiple languages (English, Dutch, French, Polish, Italian as provided in site-specific document list). Caregivers provide assistance and some instruments (eDiary) require caregiver or age-appropriate patient use.
Geography
- Total Number Of Sites
- 12
- Total Number Of Participants
- 35
Belgium
- Earliest CTIS Part Ii Submission Date
- 19-01-2024
- Latest Decision Or Authorization Date
- 18-08-2025
- Processing Time Days
- 577
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Cliniques Universitaires Saint-Luc
- Department Name
- Paediatric Department
- Principal Investigator Name
- Xavier Stephenne
- Principal Investigator Email
- xavier.stephenne@saintluc.uclouvain.be
- Contact Person Name
- Xavier Stephenne
- Contact Person Email
- xavier.stephenne@saintluc.uclouvain.be
Netherlands
- Earliest CTIS Part Ii Submission Date
- 19-01-2024
- Latest Decision Or Authorization Date
- 20-08-2025
- Processing Time Days
- 579
- Number Of Sites
- 2
- Number Of Participants
- 6
Sites
- Site Name
- Universitair Medisch Centrum Groningen
- Department Name
- Pediatric Gastro/Hepatology, Department Pediatrics
- Principal Investigator Name
- Hendrik Jan Verkade
- Principal Investigator Email
- h.j.verkade@umcg.nl
- Contact Person Name
- Hendrik Jan Verkade
- Contact Person Email
- h.j.verkade@umcg.nl
- Site Name
- Wilhelmina Childrens Hospital
- Department Name
- Paediatric Gastroenterology
- Principal Investigator Name
- Wendy Van der Woerd
- Principal Investigator Email
- wwoerd@umcutrecht.nl
- Contact Person Name
- Wendy Van der Woerd
- Contact Person Email
- wwoerd@umcutrecht.nl
France
- Earliest CTIS Part Ii Submission Date
- 19-01-2024
- Latest Decision Or Authorization Date
- 13-08-2025
- Processing Time Days
- 572
- Number Of Sites
- 2
- Number Of Participants
- 3
Sites
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Centre d'lnvestigation Clinique pediatrique
- Principal Investigator Name
- Madeleine AUMAR
- Principal Investigator Email
- madeleine.aumar@chu-lille.fr
- Contact Person Name
- Madeleine AUMAR
- Contact Person Email
- madeleine.aumar@chu-lille.fr
- Site Name
- Hopital Necker Enfants Malades
- Department Name
- Centre d'lnvestigation Clinique
- Principal Investigator Name
- Florence LACAILLE
- Principal Investigator Email
- florence.lacaille@aphp.fr
- Contact Person Name
- Florence LACAILLE
- Contact Person Email
- florence.lacaille@aphp.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 19-01-2024
- Latest Decision Or Authorization Date
- 13-02-2026
- Processing Time Days
- 756
- Number Of Sites
- 3
- Number Of Participants
- 8
Sites
- Site Name
- Universitaetsklinikum Tuebingen AöR
- Department Name
- Klinik für Kinder- und Jugendmedizin, Gastroenterologie und Hepatologie
- Principal Investigator Name
- Ekkehard Sturm
- Principal Investigator Email
- Ekkehard.sturm@med.uni-tuebingen.de
- Contact Person Name
- Ekkehard Sturm
- Contact Person Email
- Ekkehard.sturm@med.uni-tuebingen.de
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Klinik für Paediatrie mit Schwerpunkt Gastroenterologie, Nephrologie und Stoffwechselmedizin
- Principal Investigator Name
- Philip Bufler
- Principal Investigator Email
- Philip.bufler@charite.de
- Contact Person Name
- Philip Bufler
- Contact Person Email
- Philip.bufler@charite.de
- Site Name
- Medizinische Hochschule Hannover
- Department Name
- Paediatrische Gastroenterologie, Hepatologie und Lebertransplantation
- Principal Investigator Name
- Ulrich Baumann
- Principal Investigator Email
- Baumann.U@mh-hannover.de
- Contact Person Name
- Ulrich Baumann
- Contact Person Email
- Baumann.U@mh-hannover.de
Poland
- Earliest CTIS Part Ii Submission Date
- 19-01-2024
- Latest Decision Or Authorization Date
- 13-02-2026
- Processing Time Days
- 756
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Instytut Pomnik Centrum Zdrowia Dziecka
- Department Name
- Poradnia Chorob i Transplantacji Watroby
- Principal Investigator Name
- Piotr Czubkowski
- Principal Investigator Email
- p.czubkowski@ipczd.pl
- Contact Person Name
- Piotr Czubkowski
- Contact Person Email
- p.czubkowski@ipczd.pl
Italy
- Earliest CTIS Part Ii Submission Date
- 19-01-2024
- Latest Decision Or Authorization Date
- 13-02-2026
- Processing Time Days
- 756
- Number Of Sites
- 3
- Number Of Participants
- 6
Sites
- Site Name
- Bambino Gesu Childrens Hospital
- Department Name
- Division of Hepatology, Gastroenterology and Nutrition
- Principal Investigator Name
- Andrea Pietrobattista
- Principal Investigator Email
- andrea.pietrobattista@opbg.net
- Contact Person Name
- Andrea Pietrobattista
- Contact Person Email
- andrea.pietrobattista@opbg.net
- Site Name
- Azienda Ospedaliera Universitaria Meyer IRCCS
- Department Name
- Epatologia
- Principal Investigator Name
- Giuseppe Indolfi
- Principal Investigator Email
- giuseppe.indolfi@meyer.it
- Contact Person Name
- Giuseppe Indolfi
- Contact Person Email
- giuseppe.indolfi@meyer.it
- Site Name
- Azienda Ospedale-Universita Padova
- Department Name
- Dipartimento Di Salute Della Donna E Del Bambino
- Principal Investigator Name
- Mara Cananzi
- Principal Investigator Email
- mara.cananzi@aopd.veneto.it
- Contact Person Name
- Mara Cananzi
- Contact Person Email
- mara.cananzi@aopd.veneto.it
Sponsor
Primary sponsor
- Full Name
- Ipsen Pharma
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- France
Contract research organisations
- Name
- Syneos Health Netherlands B.V.
- Responsibilities
- Vendor Management, Clinical Site Monitoring and Site Management, Pharmacovigilance (Safety Management), Project and Site Start-up Services, DSMB Management, IAC Management, Investigator Payments, Project and Site Close-out Services
- Name
- PPD Global Limited
- Responsibilities
- sponsorDuties codes: [8] (as listed); contact yavor.angelski@ppd.com
- Name
- Almac Clinical Services Limited
- Responsibilities
- sponsorDuties codes: [14]; contact info@almacgroup.com
- Name
- Almac Clinical Technologies LLC
- Responsibilities
- sponsorDuties codes: [3]; contact info@almacgroup.com
Third parties
- {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties codes: [7]; contact helpdesk@mdsol.com","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"sponsorDuties codes: [4]; contact siteservices.eu@ppd.com","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"sponsorDuties codes: [14]; contact info@almacgroup.com","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"PPD Global Limited","duties_or_roles":"sponsorDuties codes: [8]; contact yavor.angelski@ppd.com","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"sponsorDuties codes: [15]; value: \"Patient Travel Reimbursement\"; contact support@greenphire.com","organisation_type":"Non-Pharmaceutical company"}
- {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"sponsorDuties codes: [3]; contact info@almacgroup.com","organisation_type":"Pharmaceutical company"}
- {"country":"Netherlands","full_name":"Syneos Health Netherlands B.V.","duties_or_roles":"sponsorDuties codes: [1,12,13,15,5,6,8,9]; value: \"Vendor Management, Clinical Site Monitoring and Site Management, Pharmacovigilance (Safety Management), Project and Site Start-up Services, DSMB Management, IAC Management, Investigator Payments, Project and Site Close-out Services\"; contact sm_clinopsams@syneoshealth.com","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"sponsorDuties codes: [15]; value: \"electronic clinical outcome assessment (\\\"eCOA\\\") Services\"; contact customercare@clario.com","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- KAYFANDA 200 microgram hard capsules
- Active Substance
- odevixibat
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Marketing authorisation (EU) EU/1/24/1854/001
- Dose Levels
- 200 microgram hard capsule
- Frequency
- Daily
- Maximum Dose
- 120 µg/Kg per day
- Investigational Product Name
- KAYFANDA 400 microgram hard capsules
- Active Substance
- odevixibat
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Marketing authorisation (EU) EU/1/24/1854/002
- Dose Levels
- 400 microgram hard capsule
- Frequency
- Daily
- Maximum Dose
- 120 µg/Kg per day
- Investigational Product Name
- KAYFANDA 600 microgram hard capsules
- Active Substance
- odevixibat
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Marketing authorisation (EU) EU/1/24/1854/003
- Dose Levels
- 600 microgram hard capsule
- Frequency
- Daily
- Maximum Dose
- 120 µg/Kg per day
- Investigational Product Name
- KAYFANDA 1 200 microgram hard capsules
- Active Substance
- odevixibat
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Marketing authorisation (EU) EU/1/24/1854/004
- Dose Levels
- 1200 microgram hard capsule
- Frequency
- Daily
- Maximum Dose
- 120 µg/Kg per day
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