A Phase 1 Study of PPI-668 in Healthy Volunteers and Patients With Hepatitis C Virus (HCV) Genotype 1

• Register: ClinicalTrials.gov
• Last refreshed on: 19 November 2012
• Main ID: NCT01448200
• Date of registration: 05/10/2011
• Primary sponsor: Presidio Pharmaceuticals, Inc.
• Public title: A Phase 1 Study of PPI-668 in Healthy Volunteers and Patients With Hepatitis C Virus (HCV) Genotype 1
• Scientific title: A Phase 1 Dose-Ranging Study to Assess the Safety, Pharmacokinetics and Antiviral Efficacy of PPI-668 in Healthy Volunteers and Patients With HCV Genotype-1 Infection
• Date of first enrolment: October 2011
• Target sample size: 82
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT01448200
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment

Countries of recruitment

Australia, New Zealand, United States

Contacts

• Name: Nathaniel Brown, M.D.
• Affiliation: Presidio Pharmaceuticals, Inc.

Key inclusion & exclusion criteria

In order to participate in the study, volunteers for Part I and patients for Part II must
meet all of the following key entry criteria, as well as other entry criteria specified in
the full protocol:

Key Inclusion Criteria

1. Male or female, between 18 and 65 years of age. Female patients must be surgically
sterile or two years post-menopausal.

2. Body Mass Index (BMI) 18 - 35 kg/m2

3. In good health, in the judgment of the Principal Investigator

4. Able and willing to comply with all protocol requirements and to sign an informed
consent.

Key Exclusion Criteria:

1. Seropositive for HIV antibody, or HBV surface antigen (HBsAg) at Screen. Volunteer
subjects for Part I must also be negative for HCV antibody.

2. Any medical condition that may interfere with the absorption, distribution or
elimination of study drug (PPI-668), or with the clinical and laboratory assessments
in this study.

3. Poorly controlled or unstable hypertension; or sustained systolic BP > 150 or
diastolic BP > 95 at Screen.

4. History of Diabetes Mellitus treated with insulin or hypoglycemic agents

5. History of alcohol abuse or illicit drug use which, in the investigator's judgment,
could interfere with a patient's compliance, with the protocol requirements or with
the safety or efficacy assessments of the study

6. History of malignancy unless the malignancy has been in complete remission and
without additional medical or surgical interventions during the preceding three years

7. No clinically significant laboratory abnormalities at Screen for healthy volunteers
in Part I. For Screen laboratory parameters for HCV patients in Part II, refer to the
'Additional Criteria for HCV Patients' below.

Additional Key Entry Criteria for HCV patients (Part II):

1. Clinical diagnosis of chronic hepatitis C, documented by:

1. Clinical findings compatible with chronic hepatitis C, and absence of other
known liver disease

2. Seropositive for HCV antibody or HCV RNA at least once previously, and at Screen

3. Serum HCV RNA > 5 log10 IU/mL at Screen, by the PCR assay at the central study
laboratory

4. HCV genotype-1 (1a or 1b, or non-subtypable genotype-1), or HCV genotype-2a or
genotype-3a

2. ALT must be <5 x ULN at screen

3. No previous treatment with interferon, pegIFN, or ribavirin for genotype-1 patients

4. No history of signs or symptoms of decompensated liver disease

5. Any of the following laboratory values at Screening will be exclusionary for study
participation:

- Hgb <11 g/dL in women or 12 g/dL in men.

- White blood cell count < 4,000/mm3.

- Absolute neutrophil count (ANC) < 1800 per mm3.

- Platelet count < 100,000 per mm3.

- Serum creatinine >ULN at the central study laboratory.

- Serum albumin < 3.4 g/dL.

- Total bilirubin > 2.0 mg/dL

- Clinically significant abnormality in the electrocardiograms (ECGs) at Screen



Age minimum: 18 Years
Age maximum: 65 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Hepatitis C, Chronic

Intervention(s)

Drug: Placebo

Drug: PPI-668

Primary Outcome(s)

Safety and tolerability, as measured by clinical adverse events and laboratory assessments [Time Frame: Part I, up to day 12; and Part II, up to day 17]

Secondary Outcome(s)

PPI-668 plasma levels [Time Frame: Part I, up to day 12; and Part II, up to day 17]

serum HCV RNA levels [Time Frame: Part II, up to day 17]

Secondary ID(s)

PPI-668-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors