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

An Open-Label Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2015-003755-21-GB

Enrollment

Registered

2016-02-26

Start date

2016-06-06

Completion date

Unknown

Last updated

2020-07-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial Chylomicronemia Syndrome (FCS) MedDRA version: 20.0 Level: LLT Classification code 10017339 Term: Fredrickson Type I lipidaemia System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10060593 Term: Fredrickson Type I lipidemia System Organ Class: 100000004850

Interventions

Product Name: Volanesorsen Product Code: ISIS 304801 Pharmaceutical Form: Solution for injection INN or Proposed INN: Volanesorsen CAS Number: 915430-78-3 Current Sponsor code: ISIS 304801 Other descr

Eligibility

Sex/Gender

All

Inclusion criteria

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

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.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the safety and efficacy of dosing and of extended dosing with volanesorsen (volanesorsen sodium 300 mg) in patients with FCS;Secondary Objective: Not applicable;Primary end point(s): Efficacy Endpoints: • Percent change and absolute change from baseline in fasting TG • Frequency and severity of patient reported abdominal pain during the treatment period • Percent change and change from baseline in other fasting lipid measurements including total cholesterol, non-HDL-C, apoB, HDL-C, apolipoprotein A-1 (apoA-1), VLDL-C, and LDL-C • Percent change from baseline in fasting total apolipoprotein C-III • Quality of Life questionnaires (EQ-5D, SF-36) • Adjudicated acute pancreatitis event rate • Other symptoms: eruptive xanthoma, lipemia retinalis Safety Endpoints: • Adverse events including adjudicated events of pancreatitis and MACE • Vital signs and weight • Physical examinations • Clinical laboratory tests (serum chemistry, hematology, coagulation, urinalysis) • Echocardiography • Electrocardiograms (ECGs) • Use of concomitant medications • MRIs;Timepoint(s) of evaluation of this end point: Fasting lipid measurements: Month 3 (average of W12 (D78) and W13 (D85) fasting assessments), Month 6 (average of W25 (D169) and W26 (D176) fasting assessments), and Month 12 (average of W51 (D351) and W52 (D358) fasting assessments) - For Extended Treatment Period: Month 17 (average of W70 (D484) and W74 (D512) fasting assessments), Month 21 (average of W86 (D596) and W90 (D624) fasting assessments). Abdominal pain: Weekly from Qualification to W65 Quality of Life questionnaires: W1, 13, 26, 52, 65 AEs including acute pancreatitis, eruptive xanthoma: at each visit Lipemia retinalis: Qualification and W52 Clinical laboratory tests: every week Echocardiography: W26 and 52 Electrocardiograms: W13, 26, 38, 52, 65 - If Extended Treatment Period: W76 and 104 MRI: W52	—

Measure

Time frame

Main Objective: To evaluate the safety and efficacy of dosing and of extended dosing with volanesorsen (volanesorsen sodium 300 mg) in patients with FCS;Secondary Objective: Not applicable;Primary end point(s): Efficacy Endpoints: • Percent change and absolute change from baseline in fasting TG • Frequency and severity of patient reported abdominal pain during the treatment period • Percent change and change from baseline in other fasting lipid measurements including total cholesterol, non-HDL-C, apoB, HDL-C, apolipoprotein A-1 (apoA-1), VLDL-C, and LDL-C • Percent change from baseline in fasting total apolipoprotein C-III • Quality of Life questionnaires (EQ-5D, SF-36) • Adjudicated acute pancreatitis event rate • Other symptoms: eruptive xanthoma, lipemia retinalis Safety Endpoints: • Adverse events including adjudicated events of pancreatitis and MACE • Vital signs and weight • Physical examinations • Clinical laboratory tests (serum chemistry, hematology, coagulation, urinalysis) • Echocardiography • Electrocardiograms (ECGs) • Use of concomitant medications • MRIs;Timepoint(s) of evaluation of this end point: Fasting lipid measurements: Month 3 (average of W12 (D78) and W13 (D85) fasting assessments), Month 6 (average of W25 (D169) and W26 (D176) fasting assessments), and Month 12 (average of W51 (D351) and W52 (D358) fasting assessments) - For Extended Treatment Period: Month 17 (average of W70 (D484) and W74 (D512) fasting assessments), Month 21 (average of W86 (D596) and W90 (D624) fasting assessments). Abdominal pain: Weekly from Qualification to W65 Quality of Life questionnaires: W1, 13, 26, 52, 65 AEs including acute pancreatitis, eruptive xanthoma: at each visit Lipemia retinalis: Qualification and W52 Clinical laboratory tests: every week Echocardiography: W26 and 52 Electrocardiograms: W13, 26, 38, 52, 65 - If Extended Treatment Period: W76 and 104 MRI: W52

—

Secondary

Measure	Time frame
Secondary end point(s): None;Timepoint(s) of evaluation of this end point: None	—

Countries

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

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026