Active — Not RecruitingPhase 3

A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen

United States, Canada24 participantsStarted 2022-02-25

Plain-language summary

The purpose of the study is to evaluate the safety, tolerability, pharmacokinetic (PK) and pharmacodynamic (PD) effects of olezarsen (formerly known as AKCEA -APOCIII-LRX) in participants with FCS previously treated with volanesorsen.

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Participants with FCS (clinical or genetic diagnosis) currently on or previously treated with volanesorsen (ISIS 304801)
✓. The following concomitant medications will be allowed if dosing regimen is expected to remain constant through the end of the study (occasional or intermittent use of over-the-counter (OTC) medications will be allowed at Investigator's discretion):

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
✕. Concomitant medication/procedure restrictions:
✕. Systemic corticosteroids or anabolic steroids within 6 weeks prior to Screening and during the study unless approved by the Sponsor Medical Monitor
✕. Plasma apheresis within 4 weeks prior to Screening or planned during the study

What they're measuring

Proportion of Participants With Decrease in Platelet Count by >30% or >50%, or With Platelet Count Value <50,000/cubic millimeter (mm^3)

Timeframe: Baseline to Week 209

Proportion of Participants With Clinical Bleeding Events

Timeframe: Baseline to Week 209

Proportion of Participants With Decrease in Estimated Glomerular Filtration Rate (eGFR) by ≥30% or ≥50%

Timeframe: Baseline to Week 209

Proportion of Participants With Urine Protein/Creatinine Ratio (UPCR) ≥1000 milligram (mg)/gram (g) or with Urine/Albumin Creatinine Ratio (UACR) ≥500 mg/g

Timeframe: Baseline to Week 209

Proportion of Participants With Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥5 x Upper Limit of Normal (ULN)

Timeframe: Baseline to Week 209

Proportion of Participants With ALT or AST ≥3 x ULN and Total Bilirubin > 2 x ULN

Timeframe: Baseline to Week 209

Proportion of Participants With Total Bilirubin ≥2 mg/deciliter (dL)

Timeframe: Baseline to Week 209

Trial details

NCT IDNCT05185843

SponsorIonis Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-06

View on ClinicalTrials.gov More Familial Chylomicronemia Syndrome trials

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

✓. Participants with FCS (clinical or genetic diagnosis) currently on or previously treated with volanesorsen (ISIS 304801)
✓. The following concomitant medications will be allowed if dosing regimen is expected to remain constant through the end of the study (occasional or intermittent use of over-the-counter (OTC) medications will be allowed at Investigator's discretion):

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
✕. Concomitant medication/procedure restrictions:
✕. Systemic corticosteroids or anabolic steroids within 6 weeks prior to Screening and during the study unless approved by the Sponsor Medical Monitor
✕. Plasma apheresis within 4 weeks prior to Screening or planned during the study

What they're measuring

Proportion of Participants With Decrease in Platelet Count by >30% or >50%, or With Platelet Count Value <50,000/cubic millimeter (mm^3)

Timeframe: Baseline to Week 209

Proportion of Participants With Clinical Bleeding Events

Timeframe: Baseline to Week 209

Proportion of Participants With Decrease in Estimated Glomerular Filtration Rate (eGFR) by ≥30% or ≥50%

Timeframe: Baseline to Week 209

Proportion of Participants With Urine Protein/Creatinine Ratio (UPCR) ≥1000 milligram (mg)/gram (g) or with Urine/Albumin Creatinine Ratio (UACR) ≥500 mg/g

Timeframe: Baseline to Week 209

Proportion of Participants With Alanine Aminotransferase (ALT) or Aspartate Aminotransferase (AST) ≥5 x Upper Limit of Normal (ULN)

Timeframe: Baseline to Week 209

Proportion of Participants With ALT or AST ≥3 x ULN and Total Bilirubin > 2 x ULN

Timeframe: Baseline to Week 209

Proportion of Participants With Total Bilirubin ≥2 mg/deciliter (dL)

Timeframe: Baseline to Week 209