An open-label clinical trial of volanesorsen in patients with Familial Chylomicronemia Syndrome (FCS)

Phase 1

• Conditions: Familial Chylomicronemia Syndrome (FCS); MedDRA version: 20.0Level: LLTClassification code 10017339Term: Fredrickson Type I lipidaemiaSystem Organ Class: 100000004850; MedDRA version: 20.0Level: LLTClassification code 10060593Term: Fredrickson Type I lipidemiaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: EUCTR2015-003755-21-GB
• Lead Sponsor: Akcea Therapeutics

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-02-26
• Last Update Posted: 13 July 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

1. Able and willing to participate in a 65-week study
2. 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
3. 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.
3a. History of chylomicronemia
3b. A diagnosis of Familial Chylomicronemia Syndrome (Type 1 Hyperlipoproteinemia)
3c. Fasting triglycerides = 750 mg/dL (8.4 mmol/L) at Screening
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 70
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

For Group 1 and Group 2:
1. 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
2. Unwilling to comply with lifestyle requirements for the duration of the study
For Group 3:
a. Diabetes mellitus if newly diagnosed or if HbA1c = 9.0%
b. Active pancreatitis within 4 weeks of screening
c. Acute Coronary Syndrome within 6 months of screening
d. Major surgery within 3 months of screening
e. Treatment with Glybera therapy within 2 years of screening
f. Have any other conditions in the opinion of the investigator which could interfere with the patient participating in or completing the study.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified