Clinical trial NCT02658175: The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Participants With Familial Chylomicronemia Syndrome
Official title: ISIS 304801-CS7 The APPROACH Open Label Study Volanesorsen (ISIS 304801) An Open-Label Study of Volanesorsen Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Familial Chylomicronemia Syndrome
Lipoprotein Lipase Deficiency
Hyperlipoproteinemia Type 1
Lead sponsor: Akcea Therapeutics
Sponsor class: INDUSTRY
Collaborators: name: Ionis Pharmaceuticals, Inc.
First posted: 2016-01-18
Enrollment: 68
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: An open-label study of volanesorsen (ISIS 304801) administered subcutaneously to participants with FCS.

Detailed description: This is a multi-center, open-label study for FCS participants rolling over from the ISIS 304801-CS6 (NCT02211209) index study, FCS participants rolling over from the ISIS 304801-CS16 (NCT02300233) index study and Treatment-naïve group. All participants were to receive volanesorsen 300 milligrams (mg) once per week for 52 weeks. Participants were allowed dose adjustment/dose reduction based on monitoring rules. Participants had the option of continuing dosing for an additional 52 weeks (France: up to an additional 104 weeks for a total of 156 weeks) until an expanded access program was approved and available in their country. Participants who were not participating in an expanded access program were to enter a 13-week (France: 26-week) post-treatment evaluation period.

Eligibility criteria: Inclusion Criteria:

* Must give written informed consent to participate in the study (signed and dated) and any authorization required by law.
* Able and willing to participate in a 65-week study.

Group 1 and 2:

* Satisfactory completion of ISIS 304801-CS6 (NCT02211209) or ISIS 304801-CS16 (NCT02300233) index studies with an acceptable safety profile, per Sponsor and Investigator judgment.

Group 3:

* Participants who did not participate in the CS6 or CS16 index studies and meet additional inclusion criteria of FCS may enroll in the study.
* History of chylomicronemia.
* A diagnosis of FCS (Type 1 Hyperlipoproteinemia.)
* Fasting triglycerides greater than or equal to (≥)750 milligrams per deciliter \[mg/dL\] (8.4 millimoles per liter \[mmol/L\]) at Screening.

Exclusion Criteria:

* Unwilling to comply with lifestyle requirements for the duration of the study.

Group 1 and 2:

* Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the participant participating in or completing the study.

Group 3:

* Diabetes mellitus if newly diagnosed or if hemoglobin A1c (HbA1c)≥ 9.0%.
* Active pancreatitis within 4 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.
* Have any other conditions in the opinion of the investigator which could interfere with the participant participating in or completing the study.

Primary outcomes:
measure: Mean Percent Change From Baseline in Fasting Triglyceride (TG)
timeFrame: Baseline and Months 3, 6, and 12
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: From first dose of study drug to end of follow-up period [Up to Week 182]
Secondary outcomes:
Other outcomes:
Locations:
city: Huntington Beach
country: United States
facility: IONIS Investigative Site
state: California
city: San Francisco
country: United States
facility: IONIS Investigative Site
state: California
city: Boca Raton
country: United States
facility: IONIS Investigative Site
state: Florida
city: Boston
country: United States
facility: IONIS Investigative Site
state: Massachusetts
city: Philadelphia
country: United States
facility: IONIS Investigative Site
state: Pennsylvania
city: Houston
country: United States
facility: IONIS Investigative Site
state: Texas
city: Norfolk
country: United States
facility: IONIS Investigative Site
state: Virginia
city: Seattle
country: United States
facility: IONIS Investigative Site
state: Washington
city: Sao Paulo
country: Brazil
facility: IONIS Investigative Site
city: Sao Paulo
country: Brazil
facility: IONIS Investigative Site
city: Vancouver
country: Canada
facility: IONIS Investigative Site
state: British Columbia
city: Chicoutimi
country: Canada
facility: IONIS Investigative Site
state: Quebec
city: Montreal
country: Canada
facility: IONIS Investigative Site
state: Quebec
city: Quebec
country: Canada
facility: IONIS Investigative Site
city: Paris
country: France
facility: IONIS Investigative Site
state: Cedex 13
city: Marseille Cedex 05
country: France
facility: IONIS Investigative Site
city: Nantes cedex 1
country: France
facility: IONIS Investigative Site
city: Berlin
country: Germany
facility: IONIS Investigative Site
city: Cologne
country: Germany
facility: IONIS Investigative Site
city: Safed
country: Israel
facility: IONIS Investigative Site
city: Palermo
country: Italy
facility: IONIS Investigative Site
city: Roma
country: Italy
facility: IONIS Investigative Site
city: Rome
country: Italy
facility: IONIS Investigative Site
city: Amsterdam-Zuidoost
country: Netherlands
facility: IONIS Investigative Site
city: Cape Town
country: South Africa
facility: IONIS Investigative Site
city: Barcelona
country: Spain
facility: IONIS Investigative Site
city: La Coruna
country: Spain
facility: IONIS Investigative Site
city: Madrid
country: Spain
facility: IONIS Investigative Site
city: Sevilla
country: Spain
facility: IONIS Investigative Site
city: Zaragoza
country: Spain
facility: IONIS Investigative Site
city: Birmingham
country: United Kingdom
facility: IONIS Investigative Site
city: London
country: United Kingdom
facility: IONIS Investigative Site
city: Manchester
country: United Kingdom
facility: IONIS Investigative Site
city: Manchester
country: United Kingdom
facility: IONIS Investigative Site
Study officials:
References: