A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)

Familial Chylomicronemia Syndrome Clinical Trial

Official title:

An Open-Label Extension Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients With Familial Chylomicronemia Syndrome (FCS)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05130450
• Other study ID #: ISIS 678354-CS13
• Secondary ID: 2021-003280-95
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: November 18, 2021
• Est. completion date: April 2025

Study information

• Verified date: May 2024
• Source: Ionis Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 60
• Est. completion date: April 2025
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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).

Related Conditions & MeSH terms

• Familial Chylomicronemia Syndrome
• Hyperlipoproteinemia Type I
• Syndrome

Intervention

Drug:
• Olezarsen
Olezarsen will be administered by SC injection.

Locations

Country	Name	City	State
Canada	Ecogene-21	Chicoutimi	Quebec
Canada	Institute de Recherches Cliniques de Montreal	Montreal	Quebec
Canada	Nathalie Saint-Pierre	Montréal	Quebec
Canada	Clinique des Maladies Lipidiques de Quebec Inc.	Québec	Quebec
France	Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique	Bron
France	Hôpital Bicêtre	Le Kremlin-Bicêtre
France	Pharmacie Hopital de la Conception	Marseille
Italy	Via Sergio Pansini 5	Napoli
Italy	UOC di Farmacia AOUP PAOLO GIACCONE	Palermo
Italy	Azienda Ospedaliero Universitaria Policlinico Umberto I	Rome
Netherlands	Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. Kenniscentrum	Amsterdam	Noor-Holland
Norway	Oslo Hospital Pharmacy Rikshospitalet	Oslo
Portugal	Hospital da Senhora da Oliveira Guimaraes	Creixomil
Portugal	Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas Moniz	Lisboa
Slovakia	Metabolicke centrum MU	Bratislava
Spain	Hospital Clínic Barcelona C/ Villarroel	Barcelona
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital Universitario Virgen del Rocío	Sevilla
Sweden	Apokteket AB	Malmö
United Kingdom	Royal Manchester Children's Hospital	Manchester
United States	Excel Medical Clinical Trials, LLC	Boca Raton	Florida
United States	Diabetes/Lipid Management & Research Center	Huntington Beach	California
United States	University of Kansas Medical Center (KUMC)	Kansas City	Kansas
United States	CTSI Investigational Pharmacy	New York	New York
United States	Milstein Hospital	New York	New York
United States	Department of Pharmacy	Park Ridge	Illinois
United States	IDS Central	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Ionis Pharmaceuticals, Inc.

Countries where clinical trial is conducted

United States,  Canada,  France,  Italy,  Netherlands,  Norway,  Portugal,  Slovakia,  Spain,  Sweden,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline		Baseline and 6 months
Secondary	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)		Baseline and 36 months
Secondary	Percentage of Participants Who Achieve = 40% Reduction in Fasting TG From Baseline at 6 Months		At 6 months
Secondary	Percentage of Participants Who Achieve = 40% Reduction in Fasting TG From Baseline at 12, 24, 36 Months		At 12, 24, 36 months
Secondary	Percent Change in Fasting apoC-III from Baseline at Month 6		Baseline and 6 months
Secondary	Percent Change in Fasting apoC-III from Month 12, 24, 36		At 12, 24, 36 months
Secondary	Percent Change From Baseline in Fasting Apolipoprotein 48 (apoB48) at 6 Months		At 6 months
Secondary	Percent Change From Baseline in Fasting apoB48 at 12, 24, 36 Months		At 12, 24, 36 months
Secondary	Percent Change in Fasting non-HDL-C from Baseline at Month 6		Baseline and 6 months
Secondary	Percent Change in Fasting non-HDL-C from Months 12, 24, 36		At 12, 24, 36 months
Secondary	Percentage of Participants Who Achieve Fasting TG = 880 milligrams per deciliter (mg/dL) at 6 Months		At 6 months
Secondary	Percentage of Participants Who Achieve Fasting TG = 880 mg/dL at 12, 24, 36 months		At 12, 24, 36 months
Secondary	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		Week 1 through Week 157
Secondary	Adjudicated Acute Pancreatitis Event Rate During the Treatment Period		Week 1 through Weeks 53, 105, or 157
Secondary	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		Week 1 through Weeks 53, 105, or 157
Secondary	Percentage of Participants Who Achieve = 70% Reduction in Fasting TG From Baseline at 6 Months		At 6 months
Secondary	Percentage of Participants Who Achieve = 70% Reduction in Fasting TG From Baseline at 12, 24, 36 Months		At 12, 24, 36 months
Secondary	Percentage of Participants Who Achieve Fasting TG = 500 mg/dL at 6 Months		At 6 months
Secondary	Percentage of Participants Who Achieve Fasting TG = 500 mg/dL at 12, 24, 36 Months		At 12, 24, 36 months