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Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Liposomal siRNA in Subjects With High Cholesterol

Primary Purpose

Hypercholesterolemia

Status

Terminated

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

PRO-040201

Placebo

About this trial

This is an interventional treatment trial for Hypercholesterolemia focused on measuring cholesterol, coronary artery disease

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesAccepts Healthy Volunteers

Inclusion Criteria:

Fasting, stable LDL-C ≥ 160 mg/dL
Fasting, stable triglyceride < 400 mg/dL
BMI between 22 and 35 kg/m2, inclusive
Females must be of non-child bearing potential
Males of reproductive potential must agree to practice effective contraception throughout the study and for 3 months following infusion

Exclusion Criteria:

Clinically significant endocrine, hematologic, renal, hepatic, pulmonary, uncontrolled psychiatric, or neurologic disease
Cancer within 5 years prior to screening
History of congestive heart failure or chronic heart failure
Uncontrolled cardiac arrhythmias
History of coronary heart disease
Clinically significant abnormal baseline ECG
History of additional risk factors for torsades de pointes
Hepatitis B, C, or HIV positive
Current diagnosis or known history of liver disease
A marked baseline prolongation of QT/QTc interval
Known history of fibromyalgia, myopathy, myositis, rhabdomyolysis, any unexplained muscle pain, or a creatine phosphokinase (CPK) >3 x upper limit of normal (ULN) at screening
Alanine aminotransferase, AST, GGT, or total bilirubin >2 x ULN at screening
Serum creatinine > 1.5 mg/dL
Systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg on 2 occasions during screening
Concomitant use of medications that prolongs the QT/QTc interval
Treatment with lipid lowering therapy within 30 days prior to screening
Use of investigational drug within 3 months prior to screening

Sites / Locations

Medpace Clinical Pharmacology Unit

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

PRO-040201

Placebo

Arm Description

PRO-040201 with placebo control in each cohort

Outcomes

Primary Outcome Measures

Safety and tolerability of PRO-040201

Secondary Outcome Measures

Pharmacokinetics of PRO-040201 in Humans

Pharmacodynamics of PRO-040201 in Humans

Full Information

NCT ID

NCT00927459

First Posted

June 23, 2009

Last Updated

January 21, 2010

Sponsor

Arbutus Biopharma Corporation

1. Study Identification

Unique Protocol Identification Number

NCT00927459

Brief Title

Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Liposomal siRNA in Subjects With High Cholesterol

Official Title

A Placebo-Controlled, Single-Blind, Single-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PRO-040201 in Male and Female Subjects With Hypercholesterolemia

Study Type

Interventional

2. Study Status

Record Verification Date

January 2010

Overall Recruitment Status

Terminated

Why Stopped

Potential for immune stimulation to interfere with further dose escalation.

Study Start Date

June 2009 (undefined)

Primary Completion Date

December 2009 (Actual)

Study Completion Date

January 2010 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor

Arbutus Biopharma Corporation

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This study is a Phase 1, single-center, placebo-controlled, single-blind, first-in-human, single-ascending dose study in male and female subjects with high cholesterol. A maximum of 32 subjects is planned for enrollment in this study.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Hypercholesterolemia

Keywords

cholesterol, coronary artery disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

Participant

Allocation

Randomized

Enrollment

23 (Actual)

8. Arms, Groups, and Interventions

Arm Title

PRO-040201

Arm Type

Experimental

Arm Description

PRO-040201 with placebo control in each cohort

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

PRO-040201 with placebo control in each cohort

Intervention Type

Drug

Intervention Name(s)

PRO-040201

Intervention Description

Single dose IV infusion

Intervention Type

Drug

Intervention Name(s)

Placebo

Intervention Description

placebo

Primary Outcome Measure Information:

Title

Safety and tolerability of PRO-040201

Time Frame

29 days

Secondary Outcome Measure Information:

Title

Pharmacokinetics of PRO-040201 in Humans

Time Frame

48 hours

Title

Pharmacodynamics of PRO-040201 in Humans

Time Frame

29 days

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Fasting, stable LDL-C ≥ 160 mg/dL Fasting, stable triglyceride < 400 mg/dL BMI between 22 and 35 kg/m2, inclusive Females must be of non-child bearing potential Males of reproductive potential must agree to practice effective contraception throughout the study and for 3 months following infusion Exclusion Criteria: Clinically significant endocrine, hematologic, renal, hepatic, pulmonary, uncontrolled psychiatric, or neurologic disease Cancer within 5 years prior to screening History of congestive heart failure or chronic heart failure Uncontrolled cardiac arrhythmias History of coronary heart disease Clinically significant abnormal baseline ECG History of additional risk factors for torsades de pointes Hepatitis B, C, or HIV positive Current diagnosis or known history of liver disease A marked baseline prolongation of QT/QTc interval Known history of fibromyalgia, myopathy, myositis, rhabdomyolysis, any unexplained muscle pain, or a creatine phosphokinase (CPK) >3 x upper limit of normal (ULN) at screening Alanine aminotransferase, AST, GGT, or total bilirubin >2 x ULN at screening Serum creatinine > 1.5 mg/dL Systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg on 2 occasions during screening Concomitant use of medications that prolongs the QT/QTc interval Treatment with lipid lowering therapy within 30 days prior to screening Use of investigational drug within 3 months prior to screening

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Douglas Logan, MD

Organizational Affiliation

Medpace Clinical Pharmacology Unit

Official's Role

Principal Investigator

Facility Information:

Facility Name

Medpace Clinical Pharmacology Unit

City

Cincinnati

State/Province

Ohio

ZIP/Postal Code

45212

Country

United States

12. IPD Sharing Statement

Learn more about this trial

Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Liposomal siRNA in Subjects With High Cholesterol

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