Clinical trial NCT05130450: A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
Official title: An Open-Label Extension Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Familial Chylomicronemia Syndrome
Keywords: FCS
Lead sponsor: Ionis Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2021-11-23
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the effect of olezarsen (formerly known as AKCEA-APOCIII-LRx) on the percent change in fasting triglycerides (TG) from baseline.

Detailed description: This is a multi-center, open-label extension (OLE) study of up to 60 participants with FCS rolling-over from Study ISIS 678354-CS3 (NCT04568434). Participants will receive olezarsen during a 157-week treatment period, followed by a 13-week post-treatment follow-up period. The length of participation in this study is approximately 201 weeks, which includes an up to 31-day qualification period, a 157-week treatment period, and a 13-week post-treatment evaluation period.

Treatment has been extended to obtain additional safety assessments and efficacy data and to provide patients with continued access to ISIS 678354 until the drug may be available commercially.

Eligibility criteria: Inclusion Criteria:

• Satisfactory completion of treatment with olezarsen in the index study (ISIS 678354-CS3, last dose as scheduled at Week 49) with an acceptable safety profile, per Investigator judgement.

Exclusion Criteria:

• Have any new condition or worsening of existing condition which in the opinion of the Investigator would make the participant unsuitable for enrollment, or could interfere with the patient participating in or completing the study, including need for treatment with medications disallowed in the index study (ISIS 678354-CS3).

Primary outcomes:
measure: Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline
timeFrame: Baseline and 6 months
Secondary outcomes:
measure: Percent Change From Baseline in Fasting TG at 12 Months (Average of Weeks 51 and 53) at 24 Months (Average of Week 103 and Week 105) and 36 Months (Average of Week 155 and Week 157)
timeFrame: Baseline and 36 months
measure: Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 6 Months
timeFrame: At 6 months
measure: Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 12, 24, 36 Months
timeFrame: At 12, 24, 36 months
measure: Percent Change in Fasting apoC-III from Baseline at Month 6
timeFrame: Baseline and 6 months
measure: Percent Change in Fasting apoC-III from Month 12, 24, 36
timeFrame: At 12, 24, 36 months
measure: Percent Change From Baseline in Fasting Apolipoprotein 48 (apoB48) at 6 Months
timeFrame: At 6 months
measure: Percent Change From Baseline in Fasting apoB48 at 12, 24, 36 Months
timeFrame: At 12, 24, 36 months
measure: Percent Change in Fasting non-HDL-C from Baseline at Month 6
timeFrame: Baseline and 6 months
measure: Percent Change in Fasting non-HDL-C from Months 12, 24, 36
timeFrame: At 12, 24, 36 months
measure: Percentage of Participants Who Achieve Fasting TG ≤ 880 milligrams per deciliter (mg/dL) at 6 Months
timeFrame: At 6 months
measure: Percentage of Participants Who Achieve Fasting TG ≤ 880 mg/dL at 12, 24, 36 months
timeFrame: At 12, 24, 36 months
measure: Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Participants With ≥ 2 Events of Adjudicated Acute Pancreatitis in 5 Years Prior to Treatment With Study Drug in the Index Study
timeFrame: Week 1 through Week 157
measure: Adjudicated Acute Pancreatitis Event Rate During the Treatment Period
timeFrame: Week 1 through Weeks 53, 105, or 157
measure: Adjudicated Acute Pancreatitis Event Rate During the Treatment Period in Patients with a Prior History of Pancreatitis within 10 Years Prior to Screening in the Index Study
timeFrame: Week 1 through Weeks 53, 105, or 157
measure: Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 6 Months
timeFrame: At 6 months
measure: Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 12, 24, 36 Months
timeFrame: At 12, 24, 36 months
measure: Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 6 Months
timeFrame: At 6 months
measure: Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 12, 24, 36 Months
timeFrame: At 12, 24, 36 months
Other outcomes:
Locations:
city: Huntington Beach
country: United States
facility: Diabetes/Lipid Management & Research Center
state: California
city: Boca Raton
country: United States
facility: Excel Medical Clinical Trials, LLC
state: Florida
city: Park Ridge
country: United States
facility: Department of Pharmacy
state: Illinois
city: Kansas City
country: United States
facility: University of Kansas Medical Center (KUMC)
state: Kansas
city: New York
country: United States
facility: CTSI Investigational Pharmacy
state: New York
city: New York
country: United States
facility: Milstein Hospital
state: New York
city: Philadelphia
country: United States
facility: IDS Central
state: Pennsylvania
city: Chicoutimi
country: Canada
facility: Ecogene-21
state: Quebec
city: Montreal
country: Canada
facility: Institute de Recherches Cliniques de Montreal
state: Quebec
city: Montréal
country: Canada
facility: Nathalie Saint-Pierre
state: Quebec
city: Québec
country: Canada
facility: Clinique des Maladies Lipidiques de Quebec Inc.
state: Quebec
city: Bron
country: France
facility: Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique
city: Le Kremlin-Bicêtre
country: France
facility: Hôpital Bicêtre
city: Marseille
country: France
facility: Pharmacie Hopital de la Conception
city: Napoli
country: Italy
facility: Via Sergio Pansini 5
city: Palermo
country: Italy
facility: UOC di Farmacia AOUP PAOLO GIACCONE
city: Rome
country: Italy
facility: Azienda Ospedaliero Universitaria Policlinico Umberto I
city: Amsterdam
country: Netherlands
facility: Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. Kenniscentrum
state: Noor-Holland
city: Oslo
country: Norway
facility: Oslo Hospital Pharmacy Rikshospitalet
city: Creixomil
country: Portugal
facility: Hospital da Senhora da Oliveira Guimaraes
city: Lisboa
country: Portugal
facility: Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas Moniz
city: Bratislava
country: Slovakia
facility: Metabolicke centrum MU
city: Barcelona
country: Spain
facility: Hospital Clínic Barcelona C/ Villarroel
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocío
city: Malmö
country: Sweden
facility: Apokteket AB
city: Manchester
country: United Kingdom
facility: Royal Manchester Children's Hospital
Study officials:
References: