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Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia

Primary Purpose

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

Status

Completed

Phase

Phase 2

Locations

Canada

Study Type

Interventional

Intervention

CAT-2003

Placebo

About this trial

This is an interventional treatment trial for Lipoprotein Lipase Deficiency, Familial

Eligibility Criteria

18 Years - 74 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

CAT-2003 or Placebo

Arm Description

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

Outcomes

Primary Outcome Measures

Percent change from baseline in fasting triglycerides in patients with chylomicronemia

Secondary Outcome Measures

Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels

Absolute and percent change from baseline on chylomicron triglyceride clearance

Absolute and percent change from baseline in plasma non-HDL-C

Frequency of adverse events

Full Information

NCT ID

NCT02098278

First Posted

March 24, 2014

Last Updated

July 19, 2016

Sponsor

Catabasis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT02098278

Brief Title

Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2016

Overall Recruitment Status

Completed

Study Start Date

March 2014 (undefined)

Primary Completion Date

April 2015 (Actual)

Study Completion Date

April 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Catabasis Pharmaceuticals

4. Oversight

Data Monitoring Committee

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

Participant

Allocation

Non-Randomized

Enrollment

13 (Actual)

8. Arms, Groups, and Interventions

Arm Title

CAT-2003 or Placebo

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

CAT-2003

Intervention Type

Drug

Intervention Name(s)

Placebo

Primary Outcome Measure Information:

Title

Percent change from baseline in fasting triglycerides in patients with chylomicronemia

Time Frame

12 Weeks

Secondary Outcome Measure Information:

Title

Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels

Time Frame

12 Weeks

Title

Absolute and percent change from baseline on chylomicron triglyceride clearance

Time Frame

12 Weeks

Title

Absolute and percent change from baseline in plasma non-HDL-C

Time Frame

12 Weeks

Title

Frequency of adverse events

Time Frame

13 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

74 Years

Accepts Healthy Volunteers

Eligibility 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

Facility Information:

City

Chicoutimi

State/Province

Quebec

ZIP/Postal Code

G7H 7K9

Country

Canada

City

Quebec City

State/Province

Quebec

ZIP/Postal Code

G1V 4M6

Country

Canada

12. IPD Sharing Statement

Learn more about this trial

Pilot Study To Assess CAT-2003 in Patients With Chylomicronemia

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