Familial Chylomicronemia Syndrome MedDRA version: 24.1 Level: LLT Classification code 10085051 Term: Familial chylomicronemia syndrome System Organ Class: 100000004850
Conditions
Interventions
Product Name: ISIS 678354
Product Code: ISIS 678354
Pharmaceutical Form: Solution for injection
Current Sponsor code: ISIS 678354
Other descriptive name: ISIS 678354 sodium salt
Concentration unit: mg
Sponsors
Ionis Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients with FCS (clinical or genetic diagnosis) currently on or previously treated with volanesorsen (ISIS 304801). Study participants in countries where Waylivra® is commercially approved and available for the patients should not be deprived of the treatment option withWaylivra®. Participation in this study for such patients will only be allowed when Waylivra® was discontinued due to adverse events (AEs) Other protocol-defined inclusion criteria apply. 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 30 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Treatment with another investigational drug (non-oligonucleotide), biological agent, or device within 4 weeks of Screening, or 5 half-lives of investigational agent, whichever is longer. Have any other conditions including significant medical history which, in the opinion of the Investigator would make the patient unsuitable for inclusion, or could interfere with the patient participating in or completing the Study Other protocol-defined exclusion criteria apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 1. To evaluate the safety of ISIS 678354 in patients with FCS previously treated with volanesorsen 2. To evaluate the tolerability of ISIS 678354 in patients with FCS previously treated with volanesorsen 3. Pharmacokinetic (PK) Objective To evaluate the PK effects of ISIS 678354 in patients with FCS previously treated with volanesorsen 4. Pharmacodynamics (PD) Objectives To evaluate the PD effects of ISIS 678354 in patients with FCS previously treated with volanesorsen;Secondary Objective: Not applicable;Primary end point(s): 1) Safety endpoints include a proportion of patients who show the following changes from Baseline to Week 53, Week 105, and Week 157: • Decrease in platelet count by > 30% • Decrease in platelet count by > 50% • Platelet count value 30% • Decrease in eGFR by > 50% • UPCR = 1000 mg/g • UACR = 500 mg/g • ALT or AST > 5 × ULN • Total bilirubin > 2.0 mg/dL • ALT or AST > 3 × ULN and total bilirubin =2 × ULN 2) Tolerability assessments: AEs, clinical laboratory tests, and use of concomitant medications 3) PK assessments include determination of trough (pre-dose) and post-treatment plasma ISIS 678354 concentrations 4) PD endpoints include: 1. Change and percent change from Baseline to Week 53, Week 105, and Week 157 in fasting: • TG • APOC-III, VLDL-C, chylomicron-TG total cholesterol (TC), non-HDL-C, LDL-C, apoB, apoB48, HDL-C, ApoA-1 2. Event rate of acute pancreatitis (Week 1 through Weeks 53, 105, and 157) ;Timepoint(s) of evaluation of this end point: 1) Baseline, Week 53, Week, 105, Week 157 2) Baseline through EoT 3) Week 1,5,13,25, 53, EoT 4) Baseline, Week, 1,5,9,13,17,21,25,29,33,37,41,45,49,53, 57,61, 105, 157, EoT | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable | — |
Countries
Sweden
Contacts
Public ContactStacy Woeppel
Ionis Pharmaceuticals, Inc.
Outcome results
None listed