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Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (ASSERT)

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ClinicalTrials.gov Identifier: NCT04674761
Recruitment Status : Recruiting
First Posted : December 19, 2020
Last Update Posted : June 3, 2021
Sponsor:
Information provided by (Responsible Party):
Albireo

Study Description
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Brief Summary:
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.

Condition or disease Intervention/treatment Phase
Alagille Syndrome Drug: Odevixibat Drug: Placebo Phase 3

Detailed Description:
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)
Actual Study Start Date : March 19, 2021
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : July 2022

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Odevixibat (A4250)
Capsules for oral administration once daily for 24 weeks.
 Drug: Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
Other Name: A4250
Placebo Comparator: Placebo
Capsules for oral administration (to match active) once daily for 24 weeks.
 Drug: Placebo
Placebo identical in appearance to experimental drug (odevixibat).


Outcome Measures
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Primary Outcome Measures :
  1. Change from Baseline in Scratching Score [ Time Frame: From baseline to Week 24 ]
    Change from baseline in scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome scratching score


Secondary Outcome Measures :
  1. Serum Bile Acid Levels [ Time Frame: From baseline to Week 24 ]
    Change in serum bile acid levels from baseline to Week 24

  2. Safety and Tolerability [ Time Frame: From first dose of study drug up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first ]
    Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Genetically confirmed diagnosis of Alagille syndrome
  • History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument
  • Elevated serum bile acid level

Key Exclusion Criteria:

  • History or ongoing presence of other types of liver disease (eg. biliary atresia, progressive familial intrahepatic cholestasis, hepatocellular carcinoma)
  • History of liver transplant, or a liver transplant is planned within 6 months of randomization
  • ALT >10× upper limit of normal (ULN) at screening
  • Total bilirubin >15 × ULN at screening
  • Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04674761


Contacts
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Contact: Albireo +1 (857) 378-2035 medinfo@albireopharma.com

Locations
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United States, California
University of California - San Francisco Recruiting
San Francisco, California, United States, 94158
Contact: Joanna Curry       joanna.curry@ucsf.edu   
Principal Investigator: Philip Rosenthal, MD         
United States, Maryland
Johns Hopkins Hospital Recruiting
Baltimore, Maryland, United States, 21287
Contact: Naureen Islam    410-614-8583    nislam5@jhmi.edu   
Contact: Ashley Thomas    410-955-9161    ashley_thomas@jhmi.edu   
Principal Investigator: Wikrom Karnsakul, MD         
Sub-Investigator: Ann Scheimann, MD         
United States, New York
The Childrens Hospital at Montefiore Albert Einstein School of Medicine Recruiting
Bronx, New York, United States, 10467
Contact: Megan Leary       mleary@montefiore.org   
Contact: Trang Nguyen, NP    7187412332    trannguy@montefiore.org   
Principal Investigator: Nadia Ovchinsky, MD         
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Contact: Isaac Effiong    212-305-3839    ie2222@cumc.columbia.edu   
Principal Investigator: Jennifer Vittorio, MD         
United States, Oregon
Oregon Health Science University School of Medicine Recruiting
Portland, Oregon, United States, 97239
Contact: Cristina Martinez-Singh    503-418-8108    martincr@ohsu.edu   
Principal Investigator: Henry Lin, MD         
France
Hôpital Femme Mère Enfant de Lyon Recruiting
Bron, France, 69677
Contact: Audrey Trouillot    33,472,681,343    audrey.trouillot@chu-lyon.fr   
Contact: Lioara Restier    33,427,856,020    lioara.restier@chu-lyon.fr   
Hopital Necker Enfants Malades Recruiting
Paris, France, 75015
Contact: Nabila Aknouche    01 44 49 47 58    nabila.oussalem@aphp.fr   
Contact: Déborah Rechard       deborah.rechard@aphp.fr   
Principal Investigator: Florence Lacaille, MD         
Poland
Instytut Pomnik-Centrum Zdrowia Dziecka Recruiting
Warszawa, Poland, 04-730
Contact: Małgorzata Pisera    790023526    mpisera@gmail.com   
Principal Investigator: Piotr Czubkowski, MD         
Sponsors and Collaborators
Albireo
More Information
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Responsible Party: Albireo
ClinicalTrials.gov Identifier: NCT04674761    
Other Study ID Numbers: A4250-012
First Posted: December 19, 2020    Key Record Dates
Last Update Posted: June 3, 2021
Last Verified: June 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Albireo:
ALGS
Alagille syndrome
Additional relevant MeSH terms:
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Alagille Syndrome
Syndrome
Disease
Pathologic Processes
Cholestasis, Intrahepatic
Cholestasis
Bile Duct Diseases
Biliary Tract Diseases
 Digestive System Diseases
Liver Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn