The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome

• ClinicalTrials.gov Identifier: NCT02658175
• Recruitment Status: Completed
• First Posted: January 18, 2016
• Last Update Posted: December 24, 2020
• Sponsor: Ionis Pharmaceuticals, Inc.
• Collaborator: Akcea Therapeutics
• Information provided by (Responsible Party): Ionis Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: January 12, 2016
• First Posted Date: January 18, 2016
• Last Update Posted Date: December 24, 2020
• Actual Study Start Date: December 31, 2015
• Actual Primary Completion Date: January 15, 2020 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: January 4, 2017): Efficacy of extended dosing of volanesorsen as measured by the percent change in fasting triglycerides from baseline. [ Time Frame: 117 Weeks ]
• Original Primary Outcome Measures (submitted: January 15, 2016): Efficacy of extended dosing of volanesorsen as measured by the percent change in fasting triglycerides from baseline. [ Time Frame: 52 Weeks ]
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients 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)
• Brief Summary: An Open-Label Study of Volanesorsen (IONIS 304801) Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)
• Detailed Description: This is a multi-center, open-label study for Group 1: ISIS 304801-CS6 (index study) roll-over FCS patients, Group 2: ISIS 304801-CS16 (index study) roll-over FCS patients, and Group 3: FCS patients who did not participate in the ISIS 304801-CS6 or ISIS 304801-CS16 index studies. All patients will receive volanesorsen 300 mg once per week for 52 weeks. Patients will have the option of continuing dosing for an additional 52 weeks until an expanded access program is approved and available in their country.
• Study Type: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

• Familial Chylomicronemia Syndrome
• Lipoprotein Lipase Deficiency
• Hyperlipoproteinemia Type 1

Intervention

Drug: Volanesorsen

300 mg volanesorsen

Other Names:

• IONIS-APOCIIIRx
• ISIS 304801

Study Arms

Experimental: Volanesorsen

Intervention: Drug: Volanesorsen

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: February 13, 2018): 69
• Original Estimated Enrollment (submitted: January 15, 2016): 60
• Actual Study Completion Date: January 15, 2020
• Actual Primary Completion Date: January 15, 2020 (Final data collection date for primary outcome measure)

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 or ISIS 304801-CS16 (index studies) with an acceptable safety profile, per Sponsor and Investigator judgment

Group 3:

• Patients who did not participate in the CS6 or CS16 index studies and meet additional inclusion criteria of Familial Chylomicronemia Syndrome (FCS) may enroll in the study.
• History of chylomicronemia
• A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
• Fasting triglycerides ≥ 750 mg/dL (8.4mmol/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 patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study.

Group 3:

• Diabetes mellitus if newly diagnosed or if 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 patient participating in or completing the study.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Brazil, Canada, France, Germany, Israel, Italy, Netherlands, South Africa, Spain, United Kingdom, United States

Administrative Information

• NCT Number: NCT02658175
• Other Study ID Numbers: ISIS 304801-CS7
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes

• IPD Sharing Statement: Not Provided
• Responsible Party: Ionis Pharmaceuticals, Inc.
• Study Sponsor: Ionis Pharmaceuticals, Inc.
• Collaborators: Akcea Therapeutics
• Investigators: Not Provided
• PRS Account: Ionis Pharmaceuticals, Inc.
• Verification Date: December 2020