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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05089084




Registration number
NCT05089084
Ethics application status
Date submitted
11/10/2021
Date registered
22/10/2021

Titles & IDs
Public title
Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)
Scientific title
A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome
Secondary ID [1] 0 0
AROAPOC3-3001
Universal Trial Number (UTN)
Trial acronym
PALISADE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Familial Chylomicronemia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Plozasiran
Treatment: Drugs - Placebo

Experimental: ARO-APOC3 (plozasiran) - 4 doses of plozasiran by subcutaneous (sc) injection (randomized period)

8 doses of plozasiran by sc injection (open-label period)

Placebo comparator: Placebo - calculated volume to match active treatment by sc injection (randomized period)


Treatment: Drugs: Plozasiran
ARO-APOC3 injection

Treatment: Drugs: Placebo
sterile normal saline (0.9% NaCl)
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10
Timepoint [1] 0 0
Baseline, Month 10
Secondary outcome [1] 0 0
Percent Change from Baseline in Fasting TG at Month 10 and Month 12 (Averaged)
Timepoint [1] 0 0
Baseline, Month 10, Month 12
Secondary outcome [2] 0 0
Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10
Timepoint [2] 0 0
Baseline, Month 10
Secondary outcome [3] 0 0
Percent Change from Baseline in Fasting APOC3 at Month 12
Timepoint [3] 0 0
Baseline, Month 12
Secondary outcome [4] 0 0
Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10
Timepoint [4] 0 0
Baseline, Month 10
Secondary outcome [5] 0 0
Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10
Timepoint [5] 0 0
Baseline, Month 10
Secondary outcome [6] 0 0
Percent Change from Baseline in Fasting TG at Month 12
Timepoint [6] 0 0
Baseline, Month 12
Secondary outcome [7] 0 0
Percent Change from Baseline in Fasting Non-HDL-C at Month 12
Timepoint [7] 0 0
Baseline, Month 12
Secondary outcome [8] 0 0
Percent Change from Baseline in Fasting HDL-C at Month 12
Timepoint [8] 0 0
Baseline, Month 12
Secondary outcome [9] 0 0
Proportion of Patients Achieving TG of < 500 mg/dL at Month 10
Timepoint [9] 0 0
Month 10
Secondary outcome [10] 0 0
Proportion of Patients Achieving TG of < 500 mg/dL at Month 12
Timepoint [10] 0 0
Month 12
Secondary outcome [11] 0 0
Change from Baseline in Fasting TG Over Time
Timepoint [11] 0 0
Baseline, up through Month 12
Secondary outcome [12] 0 0
Percent Change from Baseline in Fasting TG Over Time
Timepoint [12] 0 0
Baseline, up through Month 12
Secondary outcome [13] 0 0
Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs)
Timepoint [13] 0 0
From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period)
Secondary outcome [14] 0 0
Number of Participants with Positively Adjudicated Events of Acute Pancreatitis
Timepoint [14] 0 0
From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period)

Eligibility
Key inclusion criteria
* Fasting TG = 10 mmol/L (= 880 mg/dL) at screening refractory to standard lipid lowering therapy
* Diagnosis of FCS
* Willing to follow dietary counseling as per investigator judgement based on local standard of care
* Participants of childbearing potential (males & females) must use highly-effective contraception during the study and for at least 24 weeks following the last dose of study medication. Males must not donate sperm during the study and for at least 24 weeks following the last dose of study medication
* Women of childbearing potential must have a negative pregnancy test at Screening and cannot be breastfeeding
* Women of childbearing potential on hormonal contraceptives must be stable on the medication for = 2 menstrual cycles prior to Day 1
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Current use or use within the last 365 Days from Day 1 of any hepatocyte-targeted siRNA or antisense oligonucleotide molecule
* Diabetes mellitus newly diagnosed within 12 weeks of Screening or where HbA1c = 9.0% at Screening
* Active pancreatitis within 12 weeks before Day 1
* History of acute coronary syndrome event within 24 weeks of Day 1
* History of major surgery within 12 weeks of Day 1
* Uncontrolled hypertension
* On treatment with human immunodeficiency virus (HIV) antiretroviral therapy
* Seropositive for hepatitis B virus (HBV) or hepatitis C virus (HCV)
* New York Heart Association (NYHA) Clas II, III, or IV heart failure

Note: Additional Inclusion/Exclusion criteria may apply per protocol

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 assessing the outcomes
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
11/01/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/04/2026
Actual
Sample size
Target
Accrual to date
Final
75
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Royal North Shore Hospital - St. Leonards
Recruitment hospital [3] 0 0
Baker Heart and Diabetes Institute - Melbourne
Recruitment hospital [4] 0 0
Austin Health - Melbourne
Recruitment hospital [5] 0 0
Linear Clinical Research Ltd - Nedlands
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
2065 - St. Leonards
Recruitment postcode(s) [3] 0 0
3004 - Melbourne
Recruitment postcode(s) [4] 0 0
3081 - Melbourne
Recruitment postcode(s) [5] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Texas
Country [8] 0 0
United States of America
State/province [8] 0 0
Virginia
Country [9] 0 0
Argentina
State/province [9] 0 0
Córdoba
Country [10] 0 0
Argentina
State/province [10] 0 0
Formosa
Country [11] 0 0
Austria
State/province [11] 0 0
Graz
Country [12] 0 0
Belgium
State/province [12] 0 0
Edegem
Country [13] 0 0
Belgium
State/province [13] 0 0
Ghent
Country [14] 0 0
Belgium
State/province [14] 0 0
Leuven
Country [15] 0 0
Belgium
State/province [15] 0 0
Liège
Country [16] 0 0
Canada
State/province [16] 0 0
Ontario
Country [17] 0 0
Canada
State/province [17] 0 0
Quebec
Country [18] 0 0
Croatia
State/province [18] 0 0
Zagreb
Country [19] 0 0
France
State/province [19] 0 0
Cedez 05
Country [20] 0 0
France
State/province [20] 0 0
Paris
Country [21] 0 0
Germany
State/province [21] 0 0
Jena
Country [22] 0 0
Germany
State/province [22] 0 0
Leipzig
Country [23] 0 0
Ireland
State/province [23] 0 0
Galway
Country [24] 0 0
Israel
State/province [24] 0 0
Jerusalem
Country [25] 0 0
Japan
State/province [25] 0 0
Chiba
Country [26] 0 0
Japan
State/province [26] 0 0
Ishikawa
Country [27] 0 0
Japan
State/province [27] 0 0
Osaka
Country [28] 0 0
Japan
State/province [28] 0 0
Tochigi
Country [29] 0 0
Japan
State/province [29] 0 0
Tokyo
Country [30] 0 0
Korea, Republic of
State/province [30] 0 0
Gwangju
Country [31] 0 0
Korea, Republic of
State/province [31] 0 0
Seoul
Country [32] 0 0
Mexico
State/province [32] 0 0
Mexico DF
Country [33] 0 0
Mexico
State/province [33] 0 0
Morelos,
Country [34] 0 0
Mexico
State/province [34] 0 0
Mexico City
Country [35] 0 0
New Zealand
State/province [35] 0 0
Auckland
Country [36] 0 0
New Zealand
State/province [36] 0 0
Christchurch
Country [37] 0 0
Oman
State/province [37] 0 0
Muscat
Country [38] 0 0
Poland
State/province [38] 0 0
Lódz
Country [39] 0 0
Serbia
State/province [39] 0 0
Belgrade
Country [40] 0 0
Serbia
State/province [40] 0 0
Niš
Country [41] 0 0
Singapore
State/province [41] 0 0
Singapore
Country [42] 0 0
Spain
State/province [42] 0 0
A Coruña
Country [43] 0 0
Spain
State/province [43] 0 0
Granada
Country [44] 0 0
Spain
State/province [44] 0 0
Madrid
Country [45] 0 0
Spain
State/province [45] 0 0
Santiago De Compostela
Country [46] 0 0
Turkey
State/province [46] 0 0
Kayseri
Country [47] 0 0
Turkey
State/province [47] 0 0
Izmir

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Arrowhead Pharmaceuticals
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)?
No
No/undecided IPD sharing reason/comment


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/NCT05089084