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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT02098278

Enrollment

Registered

2014-03-28

Start date

2014-03-31

Completion date

2015-04-30

Last updated

2016-07-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Chylomicronemia, Familial, Hyperlipoproteinemia Type I, Hypertriglyceridemia, Lipoprotein Lipase Deficiency, Familial

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.

Interventions

DRUGCAT-2003

DRUGPlacebo

Study design

Allocation

NON_RANDOMIZED

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

SINGLE (Subject)

Eligibility

Sex/Gender

ALL

Age

18 Years to 74 Years

Healthy volunteers

Inclusion criteria

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

Design outcomes

Primary

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

Secondary

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

Countries

Canada

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026