A Single and Multiple Ascending Dose Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of CAT-2003

Study Overview
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Brief Summary
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This is a two-part Phase 1 placebo-controlled, double-blind, randomized single and multiple ascending dose study. In Part A, CAT-2003 is administered as a single dose; at two doses (1000 and 2000 mg), subjects will return for a second dose of CAT-2003 or placebo after a high fat meal. In Part B, CAT-2003 is administered for 14 consecutive days.

Official Title
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A Two-Part, Phase 1, Single and Multiple Ascending Dose Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of CAT-2003 in Healthy Subjects

Conditions
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Dyslipidemia

Intervention / Treatment
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* Drug: CAT 2003
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Major Inclusion Criteria: Provision of written informed consent prior to any study-specific procedure; Good health as determined by medical history, physical examination, vital signs, ECG, and clinical laboratory measurements; Satisfies one of the following: Females not of childbearing potential: non-pregnant and non-lactating; surgically sterile or postmenopausal for 2 years or at least 1 year with a follicle stimulating hormone assessment (FSH) greater than or equal to 40 IU/L; OR Males: surgically sterile, abstinent, or subject or partner is utilizing an acceptable contraceptive method during and 3 months after the last study dose; Major Exclusion Criteria: Clinically significant disease that requires a physician's care and/or would interfere with study evaluations Clinically significant electrocardiogram (ECG) abnormalities or laboratory results as assessed by the investigator, such as QTcF >450; Use of any investigational drug or participation in any investigational study within 30 days prior to screening or 5 half-lives of the study agent, whichever is longer;

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 55 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cohort A1, Dose Level 1: CAT 2003 or placebo fasting<br>Single dose | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort A2, Dose Level 2: CAT 2003 or placebo fasting<br>Single dose | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort A3, Dose Level 3: CAT 2003 or placebo fasting<br>Single dose | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort A4, Dose Level 4: CAT 2003 or placebo fasting<br>Single dose | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort A5, Dose Level 5: CAT 2003 or placebo fasting<br>Single dose | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort A2: Dose Level 2: CAT 2003 or placebo fed<br>Single dose | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort A3: Dose level 3:CAT 2003 or placebo fed<br>Single dose | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort B1: Dose level 6: CAT 2003 or placebo<br>Multiple dose for 14 days | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort B2: Dose level 7: CAT 2003 or placebo<br>Multiple dose for 14 days | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort B3: Dose level 8: CAT 2003 or placebo<br>Multiple dose for 14 days | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |
| Experimental: Cohort B4: Dose level 9: CAT 2003 or placebo<br>Multiple dose for 14 days | Drug: CAT 2003<br> <br> Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency and severity of adverse events |  | Screening to End of study (up to 4 weeks following randomization) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| AUCinf of CAT-2003 |  | Days 1, 7 and 14 | 
| Cmax of CAT-1004 |  | Days 1, 7 and14 | 
| Changes from baseline for hematology, chemistry, coagulation and urinalysis |  | Baseline through End of study (up to 4 weeks) | 
| Changes from baseline for Physical exams |  | Baseline through end of study (up to 4 weeks) | 
| Changes from baseline for ECGs |  | Baseline through end of study (up to 4 weeks) | 
| Changes from baseline in vital signs |  | Baseline through end of study (up to 4 weeks) | 
| Assess the pharmacodynamic effects of multiple doses of CAT-2003 on triglycerides and other lipids (LDL-C, VLDL-C, VLDL-triglycerides, non-HDL-C, total cholesterol, HDL-C), apoB, lipoprotein(a), and PCSK9 in healthy subjects with mild dyslipidemia |  | Baseline to end of study (up to 4 weeks) |