Pilot Study To Assess CAT-2003 in Patients With Severe Hypertriglyceridemia

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Catabasis Pharmaceuticals
ClinicalTrials.gov Identifier:
NCT01968720
First received: October 21, 2013
Last updated: March 7, 2014
Last verified: March 2014
  Purpose

The purpose of this study is to determine the safety and efficacy of multiple doses of CAT-2003 in patients with severe hypertriglyceridemia either naive or refractory to current therapy. The study will evaluate effects of CAT-2003 on fasting and postprandial total triglycerides and chylomicron triglyceride levels in patients with severe hypertriglyceridemia.

This is a single-blind study. All patients will receive placebo for a 14 day treatment period and CAT-2003 for a 28 day treatment period.


Condition Intervention Phase
Hyperlipidemias
Hypertriglyceridemia
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Drug: CAT-2003
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Single Blind (Subject)
Primary Purpose: Treatment
Official Title: A Phase 2 Pilot Study to Assess the Safety and Efficacy of CAT-2003 in Patients With Severe Hypertriglyceridemia

Resource links provided by NLM:


Further study details as provided by Catabasis Pharmaceuticals:

Primary Outcome Measures:
  • Percent change from baseline in fasting triglycerides in patients with severe hypertriglyceridemia [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Frequency of adverse events [ Time Frame: 4 weeks ] [ Designated as safety issue: Yes ]

    Safety will be assessed for all enrolled patients from the time the patient signs the informed consent through post-treatment follow-up.

    Safety parameters include physical exam, vital signs, clinical laboratory tests, ECGs and concomitant medications.


  • Absolute and percent change from baseline on postprandial total and chylomicron triglyceride levels [ Time Frame: 4 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 12
Study Start Date: October 2013
Estimated Study Completion Date: May 2014
Estimated Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: CAT-2003 or Placebo
All patients will receive placebo for a 14 day treatment period and CAT-2003 for a 28 day treatment period.
Drug: CAT-2003 Drug: Placebo

  Eligibility

Ages Eligible for Study:   18 Years to 74 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Between 18 and 74 years at Screening
  • Severe Hypertriglyceridemia (TG ≥ 500 mg/dL). If on lipid lowering therapy including statins, fibrates, and/or omega-3 fatty acids, stable dose for at least 4 weeks prior to Screening
  • Body mass index (BMI) ≤ 45 kg/m2

Exclusion Criteria:

  • History of any major cardiovascular event within 6 months of Screening
  • Type I diabetes mellitus or use of insulin
  • History of pancreatitis
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01968720

Locations
United States, Florida
Miami, Florida, United States, 33143
United States, Indiana
Indianapolis, Indiana, United States, 46260
United States, Maine
Auburn, Maine, United States, 04210
United States, Ohio
Cincinnati, Ohio, United States, 45227
Sponsors and Collaborators
Catabasis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Catabasis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01968720     History of Changes
Other Study ID Numbers: CAT-2003-202
Study First Received: October 21, 2013
Last Updated: March 7, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Dyslipidemias
Hyperlipidemias
Hypertriglyceridemia
Lipid Metabolism Disorders
Metabolic Diseases
Sphingolipidoses
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Central Nervous System Diseases
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Lipidoses
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Metabolism, Inborn Errors
Nervous System Diseases

ClinicalTrials.gov processed this record on October 20, 2014