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

Primary Purpose

Familial Chylomicronemia Syndrome

Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Olezarsen
Sponsored by
Ionis Pharmaceuticals, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Familial Chylomicronemia Syndrome focused on measuring FCS

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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).

Sites / Locations

  • Diabetes/Lipid Management & Research CenterRecruiting
  • Excel Medical Clinical Trials, LLCRecruiting
  • Department of PharmacyRecruiting
  • University of Kansas Medical Center (KUMC)Recruiting
  • CTSI Investigational PharmacyRecruiting
  • Milstein HospitalRecruiting
  • IDS CentralRecruiting
  • Ecogene-21Recruiting
  • Institute de Recherches Cliniques de MontrealRecruiting
  • Nathalie Saint-PierreRecruiting
  • Clinique des Maladies Lipidiques de Quebec Inc.Recruiting
  • Groupement Hospitalier Est- Pharmacie Secteur Essais CliniqueRecruiting
  • Hôpital BicêtreRecruiting
  • Pharmacie Hopital de la ConceptionRecruiting
  • Via Sergio Pansini 5Recruiting
  • UOC di Farmacia AOUP PAOLO GIACCONERecruiting
  • Azienda Ospedaliero Universitaria Policlinico Umberto IRecruiting
  • Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. KenniscentrumRecruiting
  • Oslo Hospital Pharmacy RikshospitaletRecruiting
  • Hospital da Senhora da Oliveira GuimaraesRecruiting
  • Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas MonizRecruiting
  • Metabolicke centrum MURecruiting
  • Hospital Clínic Barcelona C/ VillarroelRecruiting
  • Hospital Universitario 12 de OctubreRecruiting
  • Hospital Universitario Virgen del RocíoRecruiting
  • Apokteket ABRecruiting
  • Royal Manchester Children's HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Olezarsen

Arm Description

Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 153.

Outcomes

Primary Outcome Measures

Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline

Secondary Outcome Measures

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)
Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 6 Months
Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 12, 24, 36 Months
Percent Change From Baseline in Fasting Apolipoprotein 48 (apoB48) at 6 Months
Percent Change From Baseline in Fasting apoB48 at 12, 24, 36 Months
Percentage of Participants Who Achieve Fasting TG ≤ 750 milligrams per deciliter (mg/dL) at 6 Months
Percentage of Participants Who Achieve Fasting TG ≤ 750 mg/dL at 12, 24, 36 months
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 Olezarsen
Adjudicated Acute Pancreatitis Event Rate During the Treatment Period
Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 6 Months
Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 12, 24, 36 Months
Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 6 Months
Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 12, 24, 36 Months

Full Information

First Posted
November 11, 2021
Last Updated
October 18, 2023
Sponsor
Ionis Pharmaceuticals, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05130450
Brief Title
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

2. Study Status

Record Verification Date
October 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 18, 2021 (Actual)
Primary Completion Date
January 2025 (Anticipated)
Study Completion Date
April 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ionis Pharmaceuticals, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Chylomicronemia Syndrome
Keywords
FCS

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Olezarsen
Arm Type
Experimental
Arm Description
Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 153.
Intervention Type
Drug
Intervention Name(s)
Olezarsen
Other Intervention Name(s)
ISIS 678354, AKCEA -APOCIII-LRx
Intervention Description
Olezarsen will be administered by SC injection.
Primary Outcome Measure Information:
Title
Percent Change From Baseline in Fasting TG at 6 Months (Average of Weeks 23, 25, and 27) Compared to Baseline
Time Frame
Baseline and 6 months
Secondary Outcome Measure Information:
Title
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)
Time Frame
Baseline and 36 months
Title
Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 6 Months
Time Frame
At 6 months
Title
Percentage of Participants Who Achieve ≥ 40% Reduction in Fasting TG From Baseline at 12, 24, 36 Months
Time Frame
At 12, 24, 36 months
Title
Percent Change From Baseline in Fasting Apolipoprotein 48 (apoB48) at 6 Months
Time Frame
At 6 months
Title
Percent Change From Baseline in Fasting apoB48 at 12, 24, 36 Months
Time Frame
At 12, 24, 36 months
Title
Percentage of Participants Who Achieve Fasting TG ≤ 750 milligrams per deciliter (mg/dL) at 6 Months
Time Frame
At 6 months
Title
Percentage of Participants Who Achieve Fasting TG ≤ 750 mg/dL at 12, 24, 36 months
Time Frame
At 12, 24, 36 months
Title
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 Olezarsen
Time Frame
Week 1 through Week 157
Title
Adjudicated Acute Pancreatitis Event Rate During the Treatment Period
Time Frame
Week 1 through Weeks 53, 105, or 157
Title
Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 6 Months
Time Frame
At 6 months
Title
Percentage of Participants Who Achieve ≥ 70% Reduction in Fasting TG From Baseline at 12, 24, 36 Months
Time Frame
At 12, 24, 36 months
Title
Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 6 Months
Time Frame
At 6 months
Title
Percentage of Participants Who Achieve Fasting TG ≤ 500 mg/dL at 12, 24, 36 Months
Time Frame
At 12, 24, 36 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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).
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Ionis Pharmaceuticals
Phone
(844) 621-1320
Email
ionisFCS2study@clinicaltrialmedia.com
Facility Information:
Facility Name
Diabetes/Lipid Management & Research Center
City
Huntington Beach
State/Province
California
ZIP/Postal Code
92648
Country
United States
Individual Site Status
Recruiting
Facility Name
Excel Medical Clinical Trials, LLC
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33434
Country
United States
Individual Site Status
Recruiting
Facility Name
Department of Pharmacy
City
Park Ridge
State/Province
Illinois
ZIP/Postal Code
60068
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Kansas Medical Center (KUMC)
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Name
CTSI Investigational Pharmacy
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Individual Site Status
Recruiting
Facility Name
Milstein Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Individual Site Status
Recruiting
Facility Name
IDS Central
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
Ecogene-21
City
Chicoutimi
State/Province
Quebec
ZIP/Postal Code
G7H 7K9
Country
Canada
Individual Site Status
Recruiting
Facility Name
Institute de Recherches Cliniques de Montreal
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H2W 1R7
Country
Canada
Individual Site Status
Recruiting
Facility Name
Nathalie Saint-Pierre
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H2W 1R7
Country
Canada
Individual Site Status
Recruiting
Facility Name
Clinique des Maladies Lipidiques de Quebec Inc.
City
Québec
State/Province
Quebec
ZIP/Postal Code
G1V 4W2
Country
Canada
Individual Site Status
Recruiting
Facility Name
Groupement Hospitalier Est- Pharmacie Secteur Essais Clinique
City
Bron
ZIP/Postal Code
69677
Country
France
Individual Site Status
Recruiting
Facility Name
Hôpital Bicêtre
City
Le Kremlin-Bicêtre
ZIP/Postal Code
94270
Country
France
Individual Site Status
Recruiting
Facility Name
Pharmacie Hopital de la Conception
City
Marseille
ZIP/Postal Code
05 13385
Country
France
Individual Site Status
Recruiting
Facility Name
Via Sergio Pansini 5
City
Napoli
ZIP/Postal Code
80131
Country
Italy
Individual Site Status
Recruiting
Facility Name
UOC di Farmacia AOUP PAOLO GIACCONE
City
Palermo
ZIP/Postal Code
90127
Country
Italy
Individual Site Status
Recruiting
Facility Name
Azienda Ospedaliero Universitaria Policlinico Umberto I
City
Rome
ZIP/Postal Code
00161
Country
Italy
Individual Site Status
Recruiting
Facility Name
Academisch Medisch Centrum Goederenontvangst Apotheek t.a.v. Kenniscentrum
City
Amsterdam
State/Province
Noor-Holland
ZIP/Postal Code
1105 AZ
Country
Netherlands
Individual Site Status
Recruiting
Facility Name
Oslo Hospital Pharmacy Rikshospitalet
City
Oslo
ZIP/Postal Code
0372
Country
Norway
Individual Site Status
Recruiting
Facility Name
Hospital da Senhora da Oliveira Guimaraes
City
Creixomil
ZIP/Postal Code
4835-044
Country
Portugal
Individual Site Status
Recruiting
Facility Name
Dra Margarida Falcao Centro Hospitalar Lisboa Ocidental Hospital Egas Moniz
City
Lisboa
ZIP/Postal Code
1340-019
Country
Portugal
Individual Site Status
Recruiting
Facility Name
Metabolicke centrum MU
City
Bratislava
ZIP/Postal Code
83101
Country
Slovakia
Individual Site Status
Recruiting
Facility Name
Hospital Clínic Barcelona C/ Villarroel
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Virgen del Rocío
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Individual Site Status
Recruiting
Facility Name
Apokteket AB
City
Malmö
ZIP/Postal Code
211 24
Country
Sweden
Individual Site Status
Recruiting
Facility Name
Royal Manchester Children's Hospital
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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