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

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

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.

Study Overview

• Status: Completed
• Conditions: Dyslipidemia
• Intervention / Treatment: Drug: Placebo; Drug: CAT 2003

• Study Type: Interventional
• Enrollment (Actual): 99
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Ohio
    • Cincinnati, Ohio, United States
      • Medpace CPU

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

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:

  1. 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
  2. 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;

Study Plan

How is the study designed?

Design Details

• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

11

Arms and Interventions

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

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	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)

Collaborators and Investigators

• Sponsor: Catabasis Pharmaceuticals
• Investigators: Principal Investigator: Lukasz Biernat, M.D, Medpace, Inc.

Study record dates

Study Major Dates

• Study Start: November 1, 2012
• Primary Completion (Actual): May 1, 2013
• Study Completion (Actual): May 1, 2013

Study Registration Dates

• First Submitted: November 6, 2012
• First Submitted That Met QC Criteria: November 8, 2012
• First Posted (Estimate): November 14, 2012

Study Record Updates

• Last Update Posted (Estimate): July 11, 2013
• Last Update Submitted That Met QC Criteria: July 9, 2013
• Last Verified: July 1, 2013

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Lipid Metabolism Disorders; Dyslipidemias
• Other Study ID Numbers: CAT-2003-101