NCT03360747

Brief Summary

This is a single center, open-label study to evaluate the efficacy of AKCEA-ANGPTL3-LRx for reduction of triglyceride (TG) levels in participants with FCS.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
3

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2017

Shorter than P25 for phase_2

Geographic Reach
1 country

1 active site

Status
completed

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

November 18, 2017

Completed
16 days until next milestone

First Posted

Study publicly available on registry

December 4, 2017

Completed
17 days until next milestone

Study Start

First participant enrolled

December 21, 2017

Completed
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 12, 2018

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 4, 2018

Completed
2.3 years until next milestone

Results Posted

Study results publicly available

January 7, 2021

Completed
Last Updated

January 7, 2021

Status Verified

December 1, 2020

Enrollment Period

6 months

First QC Date

November 18, 2017

Results QC Date

December 11, 2020

Last Update Submit

December 11, 2020

Conditions

Familial Chylomicronemia SyndromeLipoprotein Lipase DeficiencyHyperlipoproteinemia Type 1

Keywords

Familial Lipoprotein Lipase DeficiencyHyperlipoproteinemiasFamilial Hyperlipoproteinemia Type 1Hyperlipoproteinemia Type IHyperchylomicronemia, FamilialLipoprotein Lipase Deficiency, FamilialHyperlipidemiasDyslipidemiasLipid Metabolism DisordersLipid Metabolism, Inborn ErrorsMetabolic DiseasesMetabolism, Inborn ErrorsGenetic Diseases, Inborn

Outcome Measures

Primary Outcomes (2)

  • Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)

    Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

    Baseline to Month 3

  • Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)

    Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

    Baseline to Month 3

Secondary Outcomes (8)

  • Absolute Change From Baseline to Month 3 in Fasting Angiopoietin-Like 3 (ANGPTL3)

    Baseline to Month 3

  • Percent Change From Baseline to Month 3 in Fasting Angiopoietin-like 3 (ANGPTL3)

    Baseline to Month 3

  • Fasting Lipid and Lipoprotein Measurements at Month 3

    Month 3

  • Absolute Change From Baseline to Month 3 in Other Fasting Lipid Parameters

    Baseline to Month 3

  • Percent (%) Change From Baseline to Month 3 in Other Fasting Lipid Parameters

    Baseline to Month 3

  • +3 more secondary outcomes

Study Arms (1)

AKCEA-ANGPTL3-LRx 20 mg

EXPERIMENTAL

Participants received a subcutaneous (SC) injection of AKCEA-ANGPTL3-LRx, 20 milligrams (mg), weekly (QW) for 13-weeks of treatment period. Participants were followed up to Week 26.

Drug: AKCEA-ANGPTL3-LRx

Interventions

AKCEA-ANGPTL3-LRxDRUG

AKCEA-ANGPTL3-LRx at dose 20 mg, administered via SC injection QW.

Also known as: ISIS 703802, IONIS-ANGPTL3-LRx, Vupanorsen
AKCEA-ANGPTL3-LRx 20 mg

Eligibility Criteria

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

You may qualify if:

  • Genetically confirmed chylomicronemia syndrome.
  • Fasting triglycerides greater than or equal to (\>=) 750 milligrams per deciliter (mg/dL) \[8.4 millimoles per liter (mmol/L)\] at Screening.

You may not qualify if:

  • Diabetes mellitus if newly diagnosed or if glycated hemoglobin (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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Investigative Site

Montreal, Quebec, H2W 1R7, Canada

Location

MeSH Terms

Conditions

Familial hyperchylomicronemia syndromeHyperlipoproteinemia Type IHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersLipid Metabolism, Inborn ErrorsMetabolic DiseasesMetabolism, Inborn ErrorsGenetic Diseases, Inborn

Interventions

IONIS-ANGPTL3-LRxvupanorsen

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesNutritional and Metabolic Diseases

Results Point of Contact

Title
Study Director
Organization
Akcea Therapeutics
clinicalstudies@akceatx.com
617-207-0289

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
LTE60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 18, 2017

First Posted

December 4, 2017

Study Start

December 21, 2017

Primary Completion

June 12, 2018

Study Completion

September 4, 2018

Last Updated

January 7, 2021

Results First Posted

January 7, 2021

Record last verified: 2020-12

Locations

CA(1)