CompletedPhase 3

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

United States116 participantsStarted 2018-09-28

Plain-language summary

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

Age range0 Months – 100 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of intolerable symptoms after completing at least 12 weeks of treatment
✓. Signed informed consent and assent as appropriate
✓. Patients expected to have a consistent caregiver for the duration of the study
✓. Caregivers (and age appropriate patients) must be willing and able to use an eDiary device as required by the study
✓. A male or female patient of any age, with a clinical diagnosis of PFIC, including episodic forms (i.e., BRIC), and with a body weight ≥5 kg at Visit S-1.
✓. Patient must have clinical genetic confirmation of PFIC
✓. Patients with PFIC, excluding BRIC, must have elevated serum bile acid concentration,specifically measured to be ≥100 μmol/L, taken as the average of 2 samples at least 7 days apart (Visits S-1 and S-2) prior to the Screening/Inclusion Visit (Visit 1).
✓. Patients with PFIC, excluding BRIC, must have history of significant pruritus and a caregiver-reported observed scratching or patient-reported itching (for patients \>18 with no caregiver-reported observed scratching) in the eDiary average of ≥2 (on 0 to 4 scale) in the 2 weeks prior to the Screening/Inclusion Visit (Visit 1).

✕. Decompensated liver disease: coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
✕. Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter
✕. Patients not compliant with treatment in study A4250-005
✕. Any other conditions or abnormalities which, in the opinion of the investigator or Medical Monitor, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
✕. Known pathologic variations of the ABCB11 gene that have been demonstrated to result in 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:
✕. Biliary atresia of any kind
✕. Suspected or proven liver cancer or metastasis to the liver on imaging studies

Change From Baseline in Serum Bile Acids

Timeframe: Baseline and Week 72

Proportion of Positive Pruritus Assessments at the Participant Level Over 72-Week Using the Albireo Observer-Reported Outcome (ObsRo) Instrument

Timeframe: Baseline and Week 72

NCT IDNCT03659916

SponsorAlbireo, an Ipsen Company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-02-15

Results submitted2025-01-23

Who can participate

Age range0 Months – 100 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of intolerable symptoms after completing at least 12 weeks of treatment
✓. Signed informed consent and assent as appropriate
✓. Patients expected to have a consistent caregiver for the duration of the study
✓. Caregivers (and age appropriate patients) must be willing and able to use an eDiary device as required by the study
✓. A male or female patient of any age, with a clinical diagnosis of PFIC, including episodic forms (i.e., BRIC), and with a body weight ≥5 kg at Visit S-1.
✓. Patient must have clinical genetic confirmation of PFIC
✓. Patients with PFIC, excluding BRIC, must have elevated serum bile acid concentration,specifically measured to be ≥100 μmol/L, taken as the average of 2 samples at least 7 days apart (Visits S-1 and S-2) prior to the Screening/Inclusion Visit (Visit 1).
✓. Patients with PFIC, excluding BRIC, must have history of significant pruritus and a caregiver-reported observed scratching or patient-reported itching (for patients \>18 with no caregiver-reported observed scratching) in the eDiary average of ≥2 (on 0 to 4 scale) in the 2 weeks prior to the Screening/Inclusion Visit (Visit 1).

✕. Decompensated liver disease: coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
✕. Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter
✕. Patients not compliant with treatment in study A4250-005
✕. Any other conditions or abnormalities which, in the opinion of the investigator or Medical Monitor, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
✕. Known pathologic variations of the ABCB11 gene that have been demonstrated to result in 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:
✕. Biliary atresia of any kind
✕. Suspected or proven liver cancer or metastasis to the liver on imaging studies