Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Liposomal siRNA in Subjects With High Cholesterol
Status and phase
Terminated
Phase 1
Conditions
Hypercholesterolemia
Treatments
Drug: Placebo
Drug: PRO-040201
Details and patient eligibility
About
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.
Enrollment
23 patients
Sex
All
Ages
18 to 65 years old
Volunteers
Accepts 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
Trial design
Primary purpose
Treatment
Allocation
Randomized
Interventional model
Single Group Assignment
Masking
Single Blind
23 participants in 2 patient groups, including a placebo group
PRO-040201
Experimental group
Description:
PRO-040201 with placebo control in each cohort
Treatment:
Drug: PRO-040201
Placebo
Placebo Comparator group
Description:
PRO-040201 with placebo control in each cohort
Treatment:
Drug: Placebo
Trial contacts and locations
1
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Data sourced from clinicaltrials.gov
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