Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia

• ClinicalTrials.gov Identifier: NCT02098278
• Recruitment Status: Completed
• First Posted: March 28, 2014
• Last Update Posted: July 21, 2016
• Sponsor: Catabasis Pharmaceuticals
• Information provided by (Responsible Party): Catabasis Pharmaceuticals

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Lipoprotein Lipase Deficiency, Familial; Hyperlipoproteinemia Type I; Chylomicronemia, Familial; Hypertriglyceridemia	Drug: CAT-2003; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 13 participants
• Allocation: Non-Randomized
• Intervention Model: Single Group Assignment
• Masking: Single (Participant)
• Primary Purpose: Treatment
• Official Title: A Phase 2 Pilot Study to Assess the Safety and Efficacy of CAT-2003 in Patients With Chylomicronemia
• Study Start Date: March 2014
• Actual Primary Completion Date: April 2015
• Actual Study Completion Date: April 2015

Arms and Interventions

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: CAT-2003; Drug: Placebo

Outcome Measures

Primary Outcome Measures :

1. Percent change from baseline in fasting triglycerides in patients with chylomicronemia [ Time Frame: 12 Weeks ]

Secondary Outcome Measures :

1. Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels [ Time Frame: 12 Weeks ]
2. Absolute and percent change from baseline on chylomicron triglyceride clearance [ Time Frame: 12 Weeks ]
3. Absolute and percent change from baseline in plasma non-HDL-C [ Time Frame: 12 Weeks ]
4. Frequency of adverse events [ Time Frame: 13 weeks ]

Eligibility Criteria

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

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

Contacts and Locations

Locations

Canada, Quebec

Chicoutimi, Quebec, Canada, G7H 7K9

Quebec City, Quebec, Canada, G1V 4M6

Sponsors and Collaborators

Catabasis Pharmaceuticals

More Information

• Responsible Party: Catabasis Pharmaceuticals
• ClinicalTrials.gov Identifier: NCT02098278
• Other Study ID Numbers: CAT-2003-203
• First Posted: March 28, 2014
• Last Update Posted: July 21, 2016
• Last Verified: July 2016

Additional relevant MeSH terms:

Hyperlipoproteinemia Type I; Hypertriglyceridemia; Hyperlipoproteinemias; Hyperlipidemias; Dyslipidemias; Lipid Metabolism Disorders; Metabolic Diseases; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn