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Phase 2 Study of AKCEA-ANGPTL3-LRX (ISIS 703802) in Patients With Familial Chylomicronemia Syndrome (FCS)

This study is currently recruiting participants.
Verified December 2017 by Ionis Pharmaceuticals, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT03360747
First Posted: December 4, 2017
Last Update Posted: December 8, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Akcea Therapeutics
Information provided by (Responsible Party):
Ionis Pharmaceuticals, Inc.
  Purpose
This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRX for reduction of triglyceride (TG) levels in patients with Familial Chylomicronemia Syndrome (FCS) .

Condition Intervention Phase
Familial Chylomicronemia Syndrome Lipoprotein Lipase Deficiency Hyperlipoproteinemia Type 1 Drug: AKCEA-ANGPTL3-LRX Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Open-Label Study to Assess the Pharmacodynamics, Pharmacokinetics, Safety and Tolerability of AKCEA-ANGPTL3-LRX (ISIS 703802) Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

Resource links provided by NLM:


Further study details as provided by Ionis Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Change from baseline in fasting TG [ Time Frame: 3 months ]
    Percent change and change from baseline to Month 3 in fasting TG will be summarized.


Secondary Outcome Measures:
  • Change from baseline in fasting ANGPTL3 [ Time Frame: 3 months ]
    Percent change and change from baseline to Month 3 will be summarized in fasting ANGPTL3

  • Change from baseline in fasting lipid measurements [ Time Frame: 3 months ]
    Percent change and change from baseline to Month 3 will be summarized


Estimated Enrollment: 3
Anticipated Study Start Date: December 6, 2017
Estimated Study Completion Date: July 31, 2018
Estimated Primary Completion Date: April 30, 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: AKCEA-ANGPTL3-LRX Dose 1 Drug: AKCEA-ANGPTL3-LRX
Single open-label cohort
Other Name: ISIS 703802

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Genetically confirmed Chylomicronemia Syndrome
  • Fasting triglycerides ≥ 750 mg/dL (8.4 mmol/L) at Screening

Exclusion Criteria:

  • Diabetes mellitus if newly diagnosed or if HbA1c ≥ 9.0%
  • Active pancreatitis within 2 weeks of screening
  • Acute Coronary Syndrome within 6 months of screening
  • Major surgery within 3 months of screening
  • Treatment with Glybera therapy within 2 years of screening
  • Previous treatment with AKCEA-ANGPTL3-LRX
  • Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03360747


Contacts
Contact: Akcea Therapeutics 617-207-0289 clinicalstudies@akceatx.com

Locations
Canada, Quebec
Investigative Site Recruiting
Montréal, Quebec, Canada, H2W 1R7
Sponsors and Collaborators
Ionis Pharmaceuticals, Inc.
Akcea Therapeutics
  More Information

Responsible Party: Ionis Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03360747     History of Changes
Other Study ID Numbers: ISIS 703802-CS3
First Submitted: November 18, 2017
First Posted: December 4, 2017
Last Update Posted: December 8, 2017
Last Verified: December 2017

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Ionis Pharmaceuticals, Inc.:
Familial Lipoprotein Lipase Deficiency
Hyperlipoproteinemias
Familial Hyperlipoproteinemia Type 1
Hyperlipoproteinemia Type I
Hyperchylomicronemia, Familial
Lipoprotein Lipase Deficiency, Familial
Hyperlipidemias
Dyslipidemias
Lipid Metabolism Disorders
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn

Additional relevant MeSH terms:
Syndrome
Hyperlipoproteinemias
Hyperlipidemias
Hyperlipoproteinemia Type I
Disease
Pathologic Processes
Dyslipidemias
Lipid Metabolism Disorders
Metabolic Diseases
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn