sPIF CLINICAL STUDY PROTOCOL IN AUTOIMMUNE HEPATITIS

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02239562
• Date of registration: 10/09/2014
• Prospective Registration: Yes
• Primary sponsor: Christopher O'Brien, MD
• Public title: sPIF CLINICAL STUDY PROTOCOL IN AUTOIMMUNE HEPATITIS
• Scientific title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability and Pharmacokinetics of Synthetic PreImplantation Factor (sPIF) in Autoimmune Hepatitis
• Date of first enrolment: November 14, 2014
• Target sample size: 36
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02239562
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Christopher B. O'Brien, MD
• Affiliation: University of Miami

Key inclusion & exclusion criteria

Inclusion Criteria:

- Female, aged from 18 to 70 years old, of non-child bearing potential (to avoid the
possibility if antibodies are formed to sPIF this could put the patient at risk for
future fertility)

- Females must be either

- Postmenopausal for greater than two years,

- Postmenopausal for less than two years with an follicle stimulating hormone (FSH)
level greater > 40 million international units per milliliter (mIU/mL )

- Or documented as surgically sterile (bilateral tubal ligation, bilateral
oophorectomy or post-hysterectomy) at least three months prior to the screening
evaluation

- Autoimmune hepatitis as documented by a:

- Pretreatment score =15

- Or a post-treatment score of =17 on the International Criteria for the Diagnosis
of Autoimmune Hepatitis (Appendix 2)

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

- Stable ALT levels with a fixed dose of their immunosuppressant medications

- Subjects do not have to have had 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 mofetil equal to/or less 3000 mg per day,

- Prednisone equal to/or less than 10 mg per day

- Ursodeoxycholic acid equal to/or less than 1000 mg per day

- In the judgment of the Investigator, be in reasonable general health, based on review
of the results of a screening evaluation (to include physical examination, measurement
of vital signs, 12-lead ECG trace and the collection of blood and urine for routine
clinical laboratory testing), performed no more than 30 days prior to Day 1 of study.

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

- Alanine aminotransferase (ALT) levels of no more than five times the upper limit of
normal (reference) range (ULN) at the screening evaluation.

- Normal renal function as determined by a serum creatinine

- A female of childbearing potential who is documented as either surgically sterile
(bilateral tubal ligation, bilateral oophorectomy or post-hysterectomy at least 3
months prior to the screening evaluation) or post-menopausal for = 2 years.

Exclusion Criteria:

- Any other forms of chronic liver disease.

- 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).

- Subjects 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 subject 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.

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

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



Age minimum: 18 Years
Age maximum: 70 Years
Gender: Female

Health Condition(s) or Problem(s) studied

Autoimmune Hepatitis

Intervention(s)

Drug: Placebo

Drug: sPIF

Primary Outcome(s)

Safety/Tolerability Measured by Clinical Laboratory Tests, Metabolics, Cytokines, Anti-PIF Antibody, Periodic Physical Examination, Including Vital Signs Measurements and 12-lead ECG [Time Frame: 29 Day]

Secondary Outcome(s)

Number of Participants With Anti-sPIF Antibodies and Drug Interactions [Time Frame: 29 Day]

Secondary ID(s)

20140114

119219

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

BioIncept LLC

Results

• Results available: Yes
• Date Posted: 20/11/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02239562