Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia

Hypertriglyceridemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02098278
• Other study ID #: CAT-2003-203
• Status: Completed
• Phase: Phase 2
• First received: March 24, 2014
• Last updated: July 19, 2016
• Start date: March 2014
• Est. completion date: April 2015

Study information

• Verified date: July 2016
• Source: Catabasis Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Health Canada
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 13
• Est. completion date: April 2015
• Est. primary completion date: April 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 74 Years

Eligibility

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 Design

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Single Blind (Subject), Primary Purpose: Treatment

Related Conditions & MeSH terms

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

Intervention

Drug:
• CAT-2003

• Placebo

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Catabasis Pharmaceuticals

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent change from baseline in fasting triglycerides in patients with chylomicronemia		12 Weeks	No
Secondary	Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels		12 Weeks	No
Secondary	Absolute and percent change from baseline on chylomicron triglyceride clearance		12 Weeks	No
Secondary	Absolute and percent change from baseline in plasma non-HDL-C		12 Weeks	No
Secondary	Frequency of adverse events		13 weeks	Yes