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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02658175
• Date of registration: 12/01/2016
• Prospective Registration: No
• Primary sponsor: Ionis Pharmaceuticals, Inc.
• Public title: The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome
• Scientific 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)
• Date of first enrolment: December 31, 2015
• Target sample size: 69
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02658175
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Brazil, Canada, France, Germany, Israel, Italy, Netherlands, South Africa, Spain, United Kingdom, United States

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Familial Chylomicronemia Syndrome

Hyperlipoproteinemia Type 1

Lipoprotein Lipase Deficiency

Intervention(s)

Drug: Volanesorsen

Primary Outcome(s)

Efficacy of extended dosing of volanesorsen as measured by the percent change in fasting triglycerides from baseline. [Time Frame: 117 Weeks]

Secondary ID(s)

ISIS 304801-CS7

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Akcea Therapeutics