NCT02098278

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Mar 2014

Shorter than P25 for phase_2

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2014

Completed
23 days until next milestone

First Submitted

Initial submission to the registry

March 24, 2014

Completed
4 days until next milestone

First Posted

Study publicly available on registry

March 28, 2014

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2015

Completed
Last Updated

July 21, 2016

Status Verified

July 1, 2016

Enrollment Period

1.1 years

First QC Date

March 24, 2014

Last Update Submit

July 19, 2016

Conditions

Outcome Measures

Primary Outcomes (1)

  • Percent change from baseline in fasting triglycerides in patients with chylomicronemia

    12 Weeks

Secondary Outcomes (4)

  • 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

Study Arms (1)

CAT-2003 or Placebo

EXPERIMENTAL

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

Drug: CAT-2003Drug: Placebo

Interventions

CAT-2003 or Placebo
CAT-2003 or Placebo

Eligibility Criteria

Age18 Years - 74 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Unknown Facility

Chicoutimi, Quebec, G7H 7K9, Canada

Location

Unknown Facility

Québec, Quebec, G1V 4M6, Canada

Location

MeSH Terms

Conditions

Hyperlipoproteinemia Type IHypertriglyceridemia

Condition Hierarchy (Ancestors)

Lipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 24, 2014

First Posted

March 28, 2014

Study Start

March 1, 2014

Primary Completion

April 1, 2015

Study Completion

April 1, 2015

Last Updated

July 21, 2016

Record last verified: 2016-07

Locations