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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05079919




Registration number
NCT05079919
Ethics application status
Date submitted
4/10/2021
Date registered
15/10/2021

Titles & IDs
Public title
A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia
Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of ISIS 678354 Administered Subcutaneously to Patients With Severe Hypertriglyceridemia
Secondary ID [1] 0 0
2021-002192-19
Secondary ID [2] 0 0
ISIS 678354-CS5
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Severe Hypertriglyceridemia 0 0
Condition category
Condition code
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional(has expanded access)
Description of intervention(s) / exposure
Treatment: Drugs - Olezarsen
Treatment: Drugs - Placebo

Experimental: Olezarsen - Olezarsen will be administered once every 4 weeks by subcutaneous (SC) injection from Week 1 through Week 49.

Placebo comparator: Placebo - Olezarsen-matching placebo will be administered once every 4 weeks by SC injection from Week 1 through Week 49.


Treatment: Drugs: Olezarsen
Olezarsen will be administered by SC injection.

Treatment: Drugs: Placebo
Olezarsen-matching placebo will be administered by SC injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Change from Baseline in Fasting TG at 6 Months (Average of Weeks 25 and 27) Compared to Placebo
Timepoint [1] 0 0
Baseline and Month 6
Secondary outcome [1] 0 0
Percent Change from Baseline in Fasting TG at 12 Months (Average of Weeks 51 and 53) Compared to Placebo
Timepoint [1] 0 0
Baseline and Month 12
Secondary outcome [2] 0 0
Proportion of Participants Who Achieve Fasting TG Less Than (<) 500 milligrams per deciliter (mg/dL) (5.7 millimoles per liter [mmol/L]) at 6 and 12 Months Compared to Placebo
Timepoint [2] 0 0
Month 6 and 12
Secondary outcome [3] 0 0
Proportion of Participants Who Achieve Fasting TG < 880 mg/dL (10 mmol/L) at 6 and 12 Months Compared to Placebo in the Subgroup of Participants with Baseline TG = 880 mg/dL
Timepoint [3] 0 0
Month 6 and 12
Secondary outcome [4] 0 0
Proportion of Participants Who Achieve Fasting TG < 1000 mg/dL (11.29 mmol/L) at 6 and 12 Months Compared to Placebo in the Subgroup of Participants with Baseline TG = 1000 mg/dL
Timepoint [4] 0 0
Month 6 and Month 12
Secondary outcome [5] 0 0
Percent Change from Baseline in Fasting Apolipoprotein C-III (ApoC-III) at 6 and 12 Months Compared to Placebo
Timepoint [5] 0 0
Baseline, Month 6 and 12
Secondary outcome [6] 0 0
Percent Change from Baseline in Fasting Very Low-Density Lipoprotein Cholesterol (VLDL-C) at 6 and 12 Months Compared to Placebo
Timepoint [6] 0 0
Baseline, Month 6 and 12
Secondary outcome [7] 0 0
Percent Change from Baseline in Fasting Non-High-Density Lipoprotein-Cholesterol (Non-HDL-C) at 6 and 12 Months Compared to Placebo
Timepoint [7] 0 0
Baseline, Month 6 and 12
Secondary outcome [8] 0 0
Percent Change from Baseline in Fasting High-Density Lipoprotein-Cholesterol (HDL-C) at 6 and 12 Months Compared to Placebo
Timepoint [8] 0 0
Baseline, Month 6 and 12
Secondary outcome [9] 0 0
Adjudicated Acute Pancreatitis Event Rate During the Treatment Period Compared to Placebo, in Participants with = 2 Events of Adjudicated Acute Pancreatitis in 5 Years Prior to Enrollment
Timepoint [9] 0 0
Week 1 through Week 53
Secondary outcome [10] 0 0
Adjudicated Acute Pancreatitis Event Rate from Week 13 to Week 53 Compared to Placebo in Participants with = 2 Events of Adjudicated Acute Pancreatitis in 5 Years Prior to Enrollment
Timepoint [10] 0 0
Week 13 through Week 53
Secondary outcome [11] 0 0
Adjudicated Acute Pancreatitis Event Rate During the Treatment Period Compared to Placebo
Timepoint [11] 0 0
Week 1 through Week 53
Secondary outcome [12] 0 0
Adjudicated Acute Pancreatitis Event Rate from Week 13 to Week 53 Compared to Placebo
Timepoint [12] 0 0
Week 13 through Week 53

Eligibility
Key inclusion criteria
Key

* Fasting TG = 500 mg/dL (5.65 mmol/L) at Screening and Qualification
* Patients must be on lipid-lowering therapy that should adhere to standard of care (SOC) per local guidelines. Lipid-lowering medications should be optimized and stabilized for at least 4 weeks prior to Screening to minimize changes in these medications during the study.

Key
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Hemoglobin A1c (HbA1c) = 9.5% at Screening
* Alanine aminotransferase or aspartate aminotransferase > 3.0 × upper limit of normal
* Total bilirubin > upper limit of normal unless due to Gilbert's syndrome
* Estimated GFR < 30 mL/min/1.73 m^2

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
25/10/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2025
Actual
Sample size
Target
Accrual to date
Final
617
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Royal North Shoe Hospital - Saint Leonards
Recruitment hospital [3] 0 0
Dr. Heart Pty Ltd at Cholesterol Care Australia - Woolloongabba
Recruitment hospital [4] 0 0
Monash Medical Centre - Clayton S.
Recruitment hospital [5] 0 0
The Alfred Hospital - Melbourne
Recruitment hospital [6] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2065 - Saint Leonards
Recruitment postcode(s) [3] 0 0
4102 - Woolloongabba
Recruitment postcode(s) [4] 0 0
3168 - Clayton S.
Recruitment postcode(s) [5] 0 0
3004 - Melbourne
Recruitment postcode(s) [6] 0 0
5000 - Adelaide
Recruitment outside Australia
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England
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Northamptonshire
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West Bromwich
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Cornwell
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Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Ionis Pharmaceuticals, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Ionis may share anonymized individual participant data, aggregated clinical data, and other types of data that support the results in this study. Data requests from qualified researchers will be considered once all three of the following criteria are met: (1) 12 months from marketing approval of the study drug in both the United States and European Union; (2) 18 months from conclusion of the study; and (3) 6 months from publication of study article. Access would be via a secure environment and is contingent upon approval of a research proposal and entry into an appropriate data use agreement. Requests to access data can be submitted via the website https://vivli.org/ourmember/ionis/.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://vivli.org/ourmember/ionis/


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05079919