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

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

Status

Terminated

Phases

Phase 1

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00927459

Enrollment

Registered

2009-06-25

Start date

2009-06-30

Completion date

2010-01-31

Last updated

2010-01-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hypercholesterolemia

Keywords

cholesterol, coronary artery disease

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.

Interventions

DRUGPRO-040201

Single dose IV infusion

DRUGPlacebo

placebo

Study design

Allocation

RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

SINGLE (Subject)

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

Yes

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

Design outcomes

Primary

Measure	Time frame
Safety and tolerability of PRO-040201	29 days

Secondary

Measure	Time frame
Pharmacokinetics of PRO-040201 in Humans	48 hours
Pharmacodynamics of PRO-040201 in Humans	29 days

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026