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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04720534




Registration number
NCT04720534
Ethics application status
Date submitted
19/01/2021
Date registered
22/01/2021
Date last updated
18/04/2024

Titles & IDs
Public title
Study to Evaluate ARO-APOC3 in Adults With Severe Hypertriglyceridemia
Scientific title
A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Severe Hypertriglyceridemia
Secondary ID [1] 0 0
AROAPOC3-2001
Universal Trial Number (UTN)
Trial acronym
SHASTA-2
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
Description of intervention(s) / exposure
Treatment: Drugs - ARO-APOC3
Treatment: Drugs - Placebo

Experimental: ARO-APOC3 10 mg, Day 1 and Week 12 - 2 doses of ARO-APOC3 by subcutaneous (sc) injection

Experimental: ARO-APOC3 25 mg, Day 1 and Week 12 - 2 doses of ARO-APOC3 by sc injection

Experimental: ARO-APOC3 50 mg, Day 1 and Week 12 - 2 doses of ARO-APOC3 by sc injection

Placebo comparator: Placebo, Day 1 and Week 12 - calculated volume to match active treatment by sc injection


Treatment: Drugs: ARO-APOC3
2 doses of ARO-APOC3 by subcutaneous (sc) injection

Treatment: Drugs: Placebo
calculated volume to match active treatment 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 Triglycerides (TG) at Week 24
Timepoint [1] 0 0
Baseline, Week 24
Secondary outcome [1] 0 0
Percent Change from Baseline in Fasting TG Over Time Through Week 48
Timepoint [1] 0 0
Baseline, up to Week 48
Secondary outcome [2] 0 0
Percent Change from Baseline in Apolipoprotein (Apo)C-III at Week 24
Timepoint [2] 0 0
Baseline, Week 24
Secondary outcome [3] 0 0
Percent Change from Baseline in ApoC-III Over Time Through Week 48
Timepoint [3] 0 0
Baseline, up to Week 48
Secondary outcome [4] 0 0
Percent Change from Baseline in Fasting Non-High-Density Lipoprotein-Cholesterol (Non-HDL-C) at Week 24
Timepoint [4] 0 0
Baseline, Week 24
Secondary outcome [5] 0 0
Percent Change from Baseline in Fasting Non-HDL-C Over Time Through Week 48
Timepoint [5] 0 0
Baseline, up to Week 48
Secondary outcome [6] 0 0
Percent Change from Baseline in Fasting High-Density Lipoprotein-Cholesterol (HDL-C) at Week 24
Timepoint [6] 0 0
Baseline, Week 24
Secondary outcome [7] 0 0
Percent Change from Baseline in Fasting HDL-C Over Time Through Week 48
Timepoint [7] 0 0
Baseline, up to Week 48
Secondary outcome [8] 0 0
Percent Change from Baseline in Fasting Total Apolipoprotein B (ApoB) at Week 24
Timepoint [8] 0 0
Baseline, Week 24
Secondary outcome [9] 0 0
Percent Change from Baseline in Fasting Total ApoB Over Time Through Week 48
Timepoint [9] 0 0
Baseline, up to Week 48
Secondary outcome [10] 0 0
Percent Change from Baseline in Fasting Low-density Lipoprotein-Cholesterol (LDL-C) Using Ultracentrifugation
Timepoint [10] 0 0
Baseline, Week 24
Secondary outcome [11] 0 0
Percent Change from Baseline in Fasting LDL-C Using Ultracentrifugation Over Time Through Week 48
Timepoint [11] 0 0
Baseline, up to Week 48
Secondary outcome [12] 0 0
Change from Baseline in Plasma Concentration of ARO-APOC3 Over Time Through Week 48
Timepoint [12] 0 0
Baseline, up to Week 48
Secondary outcome [13] 0 0
Number of Participants with Adverse Events (AEs) and/or Serious Adverse Events (SAEs) Possibly or Probably Related to Treatment
Timepoint [13] 0 0
up to Week 48

Eligibility
Key inclusion criteria
* Based on medical history, evidence of TG = 500 mg/dL and = 4000 mg/dL at Screening
* Fasting TG = 500 mg/dL at Screening
* Willing to follow diet counseling per Investigator judgment based on local standard of care
* Women of childbearing potential must have a negative pregnancy test, cannot be breastfeeding, and must be willing to use contraception
* Willing to provide written informed consent and to comply with study requirements
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Active pancreatitis within 12 weeks prior to first dose
* Any planned bariatric surgery or similar procedures to induce weight loss from consent to end of study
* Acute coronary syndrome event within 24 weeks of first dose
* Major surgery within 12 weeks of first dose
* Planned coronary intervention (e.g., stent placement or heart bypass) or any non-cardiac major surgical procedure throughout the study
* Uncontrolled hypertension
* Human immunodeficiency virus (HIV) infection, seropositive for Hepatitis B (HBV), seropositive for Hepatitis C (HCV)
* Uncontrolled hypothyroidism or hyperthyroidism
* Hemorrhagic stroke within 24 weeks of first dose
* Malignancy within the last 2 years prior to date of consent requiring systemic treatment (some exceptions apply)

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 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/05/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
31/08/2023
Sample size
Target
Accrual to date
Final
229
Recruitment in Australia
Recruitment state(s)
NedlandsNSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
Linear Clinical Research - Perth
Recruitment hospital [2] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [3] 0 0
Core Research Group - Brisbane
Recruitment hospital [4] 0 0
University of the Sunshine Coast - Sippy Downs
Recruitment hospital [5] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [6] 0 0
Monash Health - Clayton
Recruitment postcode(s) [1] 0 0
6009 - Perth
Recruitment postcode(s) [2] 0 0
2050 - Camperdown
Recruitment postcode(s) [3] 0 0
4064 - Brisbane
Recruitment postcode(s) [4] 0 0
4556 - Sippy Downs
Recruitment postcode(s) [5] 0 0
5000 - Adelaide
Recruitment postcode(s) [6] 0 0
3168 - Clayton
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
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United States of America
State/province [2] 0 0
Florida
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United States of America
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Georgia
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United States of America
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Illinois
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United States of America
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Kentucky
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United States of America
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Minnesota
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United States of America
State/province [7] 0 0
Mississippi
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United States of America
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Montana
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United States of America
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Nebraska
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United States of America
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Nevada
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United States of America
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New York
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United States of America
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North Carolina
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United States of America
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North Dakota
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United States of America
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Ohio
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United States of America
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Oklahoma
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United States of America
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South Carolina
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United States of America
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Tennessee
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United States of America
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Texas
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United States of America
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Virginia
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Canada
State/province [20] 0 0
Ontario
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Canada
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Quebec
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Germany
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Aachen
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Germany
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Leipzig
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Hungary
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Baja
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Hungary
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Balatonföldvár
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Hungary
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Bekescsaba
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Hungary
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Debrecen
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Hungary
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Gyöngyös
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Hungary
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Komárom
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Hungary
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Nyiregyhaza
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Hungary
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Nyíregyháza
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Netherlands
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Amsterdam
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Netherlands
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Dordrecht
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Netherlands
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Sneek
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New Zealand
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Auckland
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New Zealand
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Christchurch
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New Zealand
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Hamilton
Country [38] 0 0
New Zealand
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Papatoetoe
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New Zealand
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Rotorua
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Poland
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Bydgoszcz
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Poland
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Elblag
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Poland
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Gdynia
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Leczyca
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Oswiecim
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Poznan
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Pulawy
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Rzeszów
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Poland
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Wolomin
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Poland
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Lódz

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Arrowhead Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of AROAPOC3-2001 is to evaluate the efficacy and safety of ARO-APOC3 in participants with severe hypertriglyceridemia. Participants will receive 2 subcutaneous injections of ARO-APOC3.
Trial website
https://clinicaltrials.gov/study/NCT04720534
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04720534