A dose finding study of FFP104 for patients with Primary Biliary Cirrhosis (PBC)

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 24

Inclusion Criteria

- Male or Female Age between 18 and 75 years of age inclusive at the time of signing the informed consent.
- Established diagnosis of PBC according to the EASL criteria (European Association for the Study of the Liver 2009): A diagnosis of PBC can be made with confidence in adult patients with otherwise unexplained elevation of ALP and presence of AMA (=1:40), and/or AMA type M2. A liver biopsy is not essential for the diagnosis of PBC in these patients, but allows activity and stage of the disease to be assessed.
- Having a screening ALP serum level between 1.67 and 5 x ULN inclusive.
- Be on a stable dose of UDCA 12-20 mg/kg/day for at least 3 months prior to Screening or intolerant of UDCA in the opinion of the investigator (no UDCA for at least 8 weeks prior to Screening)
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 4

Exclusion Criteria

- Laboratory Screening results:
a) ALT >5x ULN
b) AST >5x ULN
c) Total bilirubin >2x ULN. Isolated bilirubin >2x ULN is acceptable if bilirubin is fractionated and direct bilirubin <35%
d) Total WBC <3000 cells/mm3
e) Absolute neutrophil count (ANC) <1500 cells/mm3
f) Platelet count <100,000/mm3
g) Prothrombin time (international normalized ratio (INR) >1.2
- BMI =35 or suspected to have relevant nonalcoholic fatty liver disease (NAFLD) as based on the judgment of the Investigator at Screening.
- Subject has a history of, or current viral hepatitis B or C (including hepatitis B surface antigen [HBsAg], hepatitis B core antibody and hepatitis C virus antibody [anti-HCV] positivity), or a positive HIV antibody screen at time of Screening.
- Recipients of liver or other organ transplantation or anticipated need for orthotropic organ transplantation in one year from Screening as determined by the Mayo Risk Score.
- Co-existing liver or biliary diseases, such as primary sclerosing cholangitis, choledocholithiasis, acute or chronic hepatitis, autoimmune hepatitis, alcoholic liver disease, nonalcoholic steatohepatitis (NASH), acute infection of bile duct system or gall bladder, history of gastrointestinal bleeding (secondary to portal hypertension), cholangiocarcinoma diagnosed or suspected liver cancers.
- Recurrent variceal hemorrhage, uncontrolled encephalopathy, Child-Pugh Class B/C, Esophageal - - Varices, or refractory ascites within the previous 6 months of Screening (defined as date informed consent signed).
- Have a family history (more than one first degree relative) of multiple thrombotic events or a personal history of any venous or arterial thrombotic event including deep vein thrombosis, stroke, myocardial infarction, pulmonary embolus, or peripheral arterial thromboembolic events.
- Prohibited medications 6 months prior to Screening: azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates; budesonide and other systemic corticosteroids; potentially hepatotoxic drugs (including a-methyl-dopa, sodium valproic acid, isoniazid, or nitrofurantoin).
- Prohibited medications 12 months prior to Screening: antibodies or immunotherapy directed against interleukins or other cytokines or chemokines.
- Subjects with recurrent bacterial infections (as judged by the Investigator) within 6 months prior to Screening of FFP104), active bacterial, fungal or mycobacterial infections observed during - Screening, or any recent episode of infection requiring hospitalizations or treatment with antibiotics (within the 3 months prior to Screening).
- History of malignancy, with the exception of resected basal cell carcinoma, squamous cell carcinoma of the skin, or resected cervical atypia or carcinoma in situ.
- Immunization with a live vaccine within 4 weeks of Screening, with the exception of influenza vaccine and no planned immunizations within the period of the study
- Known clinically significant cardiac disease (e.g., myocardial infarction or stroke, unstable angina, claudication, etc.), or evidence of a clinically significant electrocardiogram (ECG) abnormality within

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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