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



Status:Recruiting
Healthy:No
Age Range:Any - 18
Updated:3/30/2019
Start Date:May 16, 2018
End Date:December 2019
Contact:Albireo
Email:medinfo@albireopharma.com
Phone:(857) 254-5555

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A Double-Blind, Randomized, Placebo-Controlled, Phase 3 Study to Demonstrate Efficacy and Safety of A4250 in Children With Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 1)

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 PFIC
(deficiencies of familial intrahepatic cholestasis-1 or bile salt export pump).

Up to 50 sites in the following countries will take part in this study:

Australia, Belgium, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain,
Sweden, Turkey, United Kingdom, United States, and Saudi Arabia

Key Inclusion Criteria:

- A male or female patient with a clinical diagnosis of PFIC Type 1 or 2 and with a body
weight above 5 kg

- Patient must have clinical genetic confirmation of PFIC-1 or PFIC-2

- Patient must have elevated s-BA concentration

- Patient must have history of significant pruritus and a caregiver reported observed
scratching in the eDiary

- Patient and/or legal guardian must sign informed consent (and assent) as appropriate.

- Patients will be expected to have a consistent caregiver(s) for the duration of the
study

- Caregivers and age-appropriate patients (≥8 years of age) must be willing and able to
use an eDiary device as required by the study

Key Exclusion Criteria:

- Patient with pathologic variations of the ABCB11 gene that predict complete absence of
the BSEP protein

- Patient with past medical history or ongoing presence of other types of liver disease
including, but not limited to, the following:

1. Biliary atresia of any kind

2. Benign recurrent intrahepatic cholestasis, indicated by any history of normal s
BAs

3. Suspected or proven liver cancer or metastasis to the liver on imaging studies

4. Histopathology on liver biopsy that is suggestive of alternate non-PFIC related
etiology of cholestasis

- Patient with past medical history or ongoing chronic diarrhea

- Any patient with suspected or confirmed cancers except for basal cell carcinoma

- Patient with a past medical history of chronic kidney disease with an impaired renal
function and a glomerular filtration rate <70 mL/min/1.73 m^2

- Patient with surgical history of disruption of the enterohepatic circulation excluding
those who have undergone a successful reversal procedure that has permanently restored
flow of bile acids from the liver to the duodenum

- Patient has had a liver transplant or listed for liver transplant, excluding those who
have been listed for transplant for pruritus who have since been removed from the
transplant list

- Decompensated liver disease

- Patient suffers from uncontrolled, recalcitrant pruritic condition other than PFIC

- Patient who has been previously treated with an IBAT inhibitor whose pruritus has not
responded to treatment
We found this trial at
11
sites
660 S Euclid Ave
Saint Louis, Missouri 63110
(314) 362-5000
Washington University School of Medicine Washington University Physicians is the clinical practice of the School...
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1648 Pierce Dr NE
Atlanta, Georgia 30322
(404) 727-5640
Emory University School of Medicine Emory University School of Medicine has 2,359 full- and part-time...
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Atlanta, GA
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9500 Euclid Avenue
Cleveland, Ohio 44106
216.444.2200
Cleveland Clinic Cleveland Clinic is committed to principles as presented in the United Nations Global...
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Cleveland, OH
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4650 Sunset Blvd
Los Angeles, California 90027
 (323) 660-2450
Childrens Hospital Los Angeles Children's Hospital Los Angeles is a 501(c)(3) nonprofit hospital for pediatric...
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Los Angeles, CA
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Philadelphia, PA
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Baltimore, Maryland 21287
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Baltimore, MD
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Houston, TX
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Melbourne, 3051
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Melbourne,
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1428 Madison Ave
New York, New York 10029
(212) 241-6500
Icahn School of Medicine at Mount Sinai Icahn School of Medicine at Mount Sinai is...
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New York, NY
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New York, NY
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3414 Fifth Avenue
Pittsburgh, Pennsylvania 15213
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Pittsburgh, PA
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