Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia

Phase 2

Completed

• Conditions: Hypertriglyceridemia; Hyperlipoproteinemia Type I; Chylomicronemia, Familial; Lipoprotein Lipase Deficiency, Familial
• Interventions: Drug: CAT-2003; Drug: Placebo

• Registration Number: NCT02098278
• Lead Sponsor: Catabasis Pharmaceuticals

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2014-03
• Study First Submitted: 2014-03-24
• Study First Submitted QC: 2014-03-27
• Study First Posted: 2014-03-28
• Primary Completion: 2015-04
• Study Completion: 2015-04
• Status Verified: 2016-07
• Disposition First Submitted: 2016-07-19
• Disposition First Submitted QC: 2016-07-19
• Last Update Submitted: 2016-07-19
• Disposition First Posted: 2016-07-21
• Last Update Posted: 2016-07-21

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 13

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CAT-2003 or Placebo	Placebo	All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.
CAT-2003 or Placebo	CAT-2003	All patients will receive placebo for 1 week, and CAT-2003 for 12 weeks during the 13 week treatment period.

Primary Outcome Measures

Name	Time	Method
Percent change from baseline in fasting triglycerides in patients with chylomicronemia	12 Weeks

Secondary Outcome Measures

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