Phase II AutoImmune Hepatitis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03593460
• Date of registration: 07/06/2018
• Prospective Registration: Yes
• Primary sponsor: Christopher O'Brien, MD
• Public title: Phase II AutoImmune Hepatitis
• Scientific title: Phase II, Open Label, Adaptive Design, Multiple Dose Finding Study to Investigate Synthetic PreImplantation Factor (sPIF) in Patients With Autoimmune Hepatitis (AIH)
• Date of first enrolment: January 1, 2019
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT03593460
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Contacts

• Name: eytan barnea, MD
• Affiliation: BioIncept LLC

Key inclusion & exclusion criteria

Written informed consent must be obtained before any assessment is performed. Patients with
a confirmed diagnosis of AIH either Type II or Type II, based on the International Criteria
for the Diagnosis of AIH, will be enrolled at screening.

- Males and females aged from 18 to 75 years old, of non-child bearing potential

- Females must be either:

Postmenopausal for greater than two years Postmenopausal for less than two years with an
FSH level greater > 40mIU/mL Documented as surgically sterile (bilateral tubal ligation,
bilateral oophorectomy or post-hysterectomy) at least three months prior to the screening
evaluation Or be greater than age 40, does not want more children, is currently using at
least one effective method of birth control at the time of screening and agrees to using
two effective methods of birth control starting with Study day 0 and through the 42 days
duration of the study

• Must have in the judgment of the Investigator, the diagnosis of AIH, or have a score on
the International Criteria for the Diagnosis of AIH (Appendix 2) of: Pretreatment score =15
Post-treatment score of =17

- Treatment with prednisone and/or other oral, immunosuppressive drug(s) must be
stabilized for 6 weeks prior to screening for this study.

- All patients with an abnormal ALT confirmed by two measurements 2 weeks apart at
screening, with the present dose(s) of their immunosuppressant medication(s)

- Patients do not have to have a documented relapse after completion of an initial
course of therapy

- Permitted concomitant immunosuppressant medications will include:

Azathioprine dose equal to/or less 100 mg per day Budesonide dose equal to/or less 9 mg per
day Mycophenolate metil equal to/or less 3000 mg per day Prednisone equal to/or less than
30 mg per day Ursodeoxycholic acid equal to/or less than 1500 mg er day Prograf equal to/or
less than 6 mg per day

- Patients must agree to abstain from alcohol and illicit drugs use during their
participation in the study protocol

- ALT and AST levels greater than 2 times less than five times the upper limit of the
normal (reference) range (ULN) and have no clinical or laboratory evidence of hepatic
decompensation (i.e., platelets = 100,000/mm, total bilirubin = 1.5 × ULN, prothrombin
time = 1.2 × ULN and albumin = 3.0 g/dL) for inclusion.

- Normal renal function as determined by a serum creatinine

Exclusion criteria

Patients fulfilling any of the following criteria are not eligible for inclusion in this
study. No additional exclusions may be applied by the Investigator, to ensure that the
study population will be representative of all eligible patients.

- Any other forms of chronic liver disease that is not under treatment

- Decompensated liver disease defined on the basis of any one of the following
laboratory parameters at the screening evaluation: total bilirubin > 1.5 × ULN,
prothrombin time > 1.2 × ULN, platelets = 100,000/mm3, or albumin < 3 g/dL OR current
or prior history of clinical hepatic decompensation (e.g., ascites, jaundice,
encephalopathy or variceal hemorrhage)

- Hemoglobin < 11 g/dL at the screening evaluation

- Serological evidence of infection with HIV upon review of the medical record

- Evidence of hepatocellular carcinoma (i.e., screening a-fetoprotein > 50 ng/mL or
other standard of care measure and ultrasound)

- Patients with, or a history of clinically significant oncologic, pulmonary, hepatic,
gastrointestinal, renal, other cardiovascular, hematologic, metabolic, endocrine,
neurologic, immunologic or hematologic illness or any other major medical disorder
that, in the judgment of the Investigator, would interfere with patient treatment,
assessment or compliance with the protocol or should otherwise preclude their
participation in this trial

- Have received therapy with potentially hepatotoxic drugs within 3 months (90 days)
prior to day 1 or are expected to receive such therapy during the study

- Patients who are expected to receive a change in their immunosuppressant therapies
during the protocol

- Patients who may receive chemotherapeutic agents (e.g. immunoglobulins and other
immune- or cytokine-based therapies) during the study for any other medical condition

- Use of other investigational drugs within 5 half-lives of enrollment, or within 30
days until the expected pharmacodynamic effect has returned to baseline, whichever is
longer

- History of hypersensitivity to study drug or its excipients

- Hepatitis B and C, infectious hepatitis (documented in medical record may need to
repeat prior to enrollment)

- Tuberculosis (documented in medical record)

- Primary sclerosing cholangitis, primary biliary cholangitis (documented in medical
record and may need to repeat prior to enrollment)

- Alpha-1 antitrypsin deficiency, hemochromatosis, or Wilson's disease (documented in
the medical record)



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hepatitis, Autoimmune

Intervention(s)

Drug: Synthetic PreImplantation Factor

Primary Outcome(s)

Evaluate the pharmacokinetics of sPIF levels in the circulation after multiple ascending, subcutaneously administered doses of sPIF by normalization of liver enzymes, immunoglobulins, and bilirubin levels [Time Frame: 12 weeks]

Evaluate the safety and tolerability of multiple ascending, subcutaneously administered doses of sPIF in patients with autoimmune hepatitis (AIH) by measuring changes in ALT [Time Frame: 12 weeks]

Evaluate the safety and tolerability of the increased sPIF dose and treatment duration by no SAEes greater than grade 3 [Time Frame: 12 weeks]

Secondary ID(s)

sPIF-US-AIH-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

BioIncept LLC