Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia

Catabasis Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Hyperlipoproteinemia Type I; Chylomicronemia, Familial; Hypertriglyceridemia; Lipoprotein Lipase Deficiency, Familial

Treatments: Drug: Placebo; Drug: CAT-2003

Study type: Interventional

Funder types: Industry

Identifiers

NCT02098278

CAT-2003-203

Details and patient eligibility

About

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.

Enrollment: 13 patients

Sex: All

Ages: 18 to 74 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: Single Blind

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13 participants in 1 patient group

CAT-2003 or Placebo

Experimental group

Description:

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

Treatment:

Drug: Placebo

Drug: CAT-2003