Pilot Study To Assess CAT-2003 in Patients With Hyperlipidemia (PATHWAYS I)

A Randomized, Double-Blind, Placebo-Controlled Pilot Study to Assess the Safety, Efficacy and Pharmacodynamics of CAT-2003 Alone and In Combination With A Statin in Patients With Hyperlipidemia (PATHWAYS I)

The purpose of this study is to determine the safety and efficacy of different doses of CAT-2003 in patients with hyperlipidemia when CAT-2003 is taken for 4 weeks. The study will evaluate effects of CAT-2003 on (1) fasting triglycerides and non-HDL-C in patients with moderate hypertriglyceridemia and (2) fasting LDL-C levels in combination with a statin in patients with hypercholesterolemia who are on a statin.

Study Overview

• Status: Completed
• Conditions: Dyslipidemia; Hypercholesterolemia; Hypertriglyceridemia
• Intervention / Treatment: Drug: Placebo; Drug: CAT-2003; Drug: Statin

• Study Type: Interventional
• Enrollment (Actual): 99
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Sainte-Foy, Quebec, Canada, G1V 4M6
• United States
  • Alabama
    • Muscle Shoals, Alabama, United States, 35662
  • California
    • Los Angeles, California, United States, 90057
  • Florida
    • Jacksonville, Florida, United States, 32223
    • Miami, Florida, United States, 33143
    • Winter Park, Florida, United States, 32792
  • Kansas
    • Kansas City, Kansas, United States, 66160
  • Kentucky
    • Louisville, Kentucky, United States, 40213
  • Maine
    • Auburn, Maine, United States, 04120
  • North Carolina
    • Raleigh, North Carolina, United States, 27609
    • Raleigh, North Carolina, United States, 27612
  • Ohio
    • Cincinnati, Ohio, United States, 45219
    • Cincinnati, Ohio, United States, 45227
    • Cincinnati, Ohio, United States, 45246

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 69 years (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Between 18 and 69 years at Screening
• Hypertriglyceridemia (TG ≥ 200 mg/dL and < 500 mg/dL and non-HDL-C ≥ 100 mg/dL and < 220 mg/dL) OR
• Hypercholesterolemia (LDL-C ≥ 100 mg/dL and < 190 mg/dL and TG value < 200 mg/dL) plus a stable dose of statin for at least 4 weeks prior to Screening.
• Body mass index (BMI) ≤ 45 kg/m2

Exclusion Criteria:

• History of any major cardiovascular event within 6 months of Screening
• Type I diabetes mellitus
• Any condition that may predispose the patient to secondary hyperlipidemia, such as uncontrolled hypothyroidism
• Any statin at the highest approved dose
• Non-statin lipid-altering drugs other than cholesterol absorption inhibitors.
• Active peptic ulcer disease or a history of muscle disease or myopathy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: CAT-2003 or Placebo Dose 1; Daily for 28 days in patients with moderate hypertriglyceridemia	Drug: Placebo; Drug: CAT-2003
EXPERIMENTAL: CAT-2003 or Placebo Dose 2; Daily for 28 days in patients with moderate hypertriglyceridemia	Drug: Placebo; Drug: CAT-2003
EXPERIMENTAL: CAT-2003 or Placebo Dose 3; Daily for 28 days in patients with moderate hypertriglyceridemia	Drug: Placebo; Drug: CAT-2003
EXPERIMENTAL: CAT-2003 or Placebo Dose 4; Daily for 28 days in patients with hypercholesterolemia who are on a statin	Drug: Placebo; Drug: CAT-2003; Drug: Statin; All patients being treated with a stable dose of a statin prior to enrollment continue on their dosing regimen.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Absolute and percent change from baseline in plasma triglycerides in patients with hypertriglyceridemia	4 weeks
Absolute and percent change from baseline in plasma low density lipoprotein-cholesterol in patients with hypercholesterolemia	4 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Absolute and percent change from baseline in plasma non-high-density lipoprotein cholesterol		4 weeks
Pharmacodynamic effects of CAT-2003 on other lipid biomarkers in patients with hyperlipidemia		4 weeks
Frequency of adverse events	Safety and tolerability will be assessed for all enrolled patients from the time the patient signs the informed consent through post-treatment follow-up. Safety parameters include physical exam, vital signs, clinical laboratory tests, ECGs and concomitant medications.	4 weeks

Collaborators and Investigators

• Sponsor: Catabasis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start: July 1, 2013
• Primary Completion (ACTUAL): December 1, 2013
• Study Completion (ACTUAL): December 1, 2013

Study Registration Dates

• First Submitted: July 25, 2013
• First Submitted That Met QC Criteria: July 29, 2013
• First Posted (ESTIMATE): July 31, 2013

Study Record Updates

• Last Update Posted (ESTIMATE): July 28, 2016
• Last Update Submitted That Met QC Criteria: July 26, 2016
• Last Verified: July 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Lipid Metabolism Disorders; Dyslipidemias; Hypercholesterolemia; Hyperlipidemias; Hypertriglyceridemia; Hyperlipoproteinemias
• Other Study ID Numbers: CAT-2003-201