Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia

A Phase 2 Pilot Study to Assess the Safety and Efficacy of CAT-2003 in Patients With Chylomicronemia

The purpose of this study is to assess safety and efficacy of CAT-2003 in patients with chylomicronemia. The study will evaluate the effects of CAT-2003 on fasting total and chylomicron triglyceride levels, as well as postprandial total and chylomicron triglyceride clearance.

This is a single-blind study. All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.

Study Overview

• Status: Completed
• Conditions: Hypertriglyceridemia; Hyperlipoproteinemia Type I; Lipoprotein Lipase Deficiency, Familial; Chylomicronemia, Familial
• Intervention / Treatment: Drug: Placebo; Drug: CAT-2003

• Study Type: Interventional
• Enrollment (Actual): 13
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Quebec
    • Chicoutimi, Quebec, Canada, G7H 7K9
    • Quebec City, Quebec, Canada, G1V 4M6

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 74 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Key Inclusion Criteria:

• Familial Chylomicronemia (Fasting triglycerides ≥ 880 mg/dL at Screening and documented history of plasma post-heparin LDL activity < 20% of normal or genetic confirmation of homozygosity or compound heterozygosity for loss-of-function mutations in familial chylomicronemia-causing genes) OR
• Non-familial Chylomicronemia (Fasting triglycerides ≥ 440 mg/dL at Screening and documented history of fasting triglycerides ≥ 880 mg/dL)

Key Exclusion Criteria:

• History of any major cardiovascular event within 6 months of Screening
• Type I diabetes mellitus or use of insulin
• History of pancreatitis within 3 month of Screening

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: Single

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CAT-2003 or Placebo; All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.	Drug: Placebo; Drug: CAT-2003

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percent change from baseline in fasting triglycerides in patients with chylomicronemia	12 Weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels	12 Weeks
Absolute and percent change from baseline on chylomicron triglyceride clearance	12 Weeks
Absolute and percent change from baseline in plasma non-HDL-C	12 Weeks
Frequency of adverse events	13 weeks

Collaborators and Investigators

• Sponsor: Catabasis Pharmaceuticals

Study record dates

Study Major Dates

• Study Start: March 1, 2014
• Primary Completion (Actual): April 1, 2015
• Study Completion (Actual): April 1, 2015

Study Registration Dates

• First Submitted: March 24, 2014
• First Submitted That Met QC Criteria: March 27, 2014
• First Posted (Estimate): March 28, 2014

Study Record Updates

• Last Update Posted (Estimate): July 21, 2016
• Last Update Submitted That Met QC Criteria: July 19, 2016
• Last Verified: July 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Dyslipidemias; Lipid Metabolism, Inborn Errors; Hyperlipidemias; Hypertriglyceridemia; Hyperlipoproteinemias; Hyperlipoproteinemia Type I
• Other Study ID Numbers: CAT-2003-203