Clinical trial NCT04720534: Study to Evaluate ARO-APOC3 in Adults With Severe Hypertriglyceridemia
Official title: A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Severe Hypertriglyceridemia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Severe Hypertriglyceridemia
Lead sponsor: Arrowhead Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2021-01-22
Enrollment: 229
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

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

Primary outcomes:
measure: Percent Change from Baseline in Fasting Triglycerides (TG) at Week 24
timeFrame: Baseline, Week 24
Secondary outcomes:
measure: Percent Change from Baseline in Fasting TG Over Time Through Week 48
timeFrame: Baseline, up to Week 48
measure: Percent Change from Baseline in Apolipoprotein (Apo)C-III at Week 24
timeFrame: Baseline, Week 24
measure: Percent Change from Baseline in ApoC-III Over Time Through Week 48
timeFrame: Baseline, up to Week 48
measure: Percent Change from Baseline in Fasting Non-High-Density Lipoprotein-Cholesterol (Non-HDL-C) at Week 24
timeFrame: Baseline, Week 24
measure: Percent Change from Baseline in Fasting Non-HDL-C Over Time Through Week 48
timeFrame: Baseline, up to Week 48
measure: Percent Change from Baseline in Fasting High-Density Lipoprotein-Cholesterol (HDL-C) at Week 24
timeFrame: Baseline, Week 24
measure: Percent Change from Baseline in Fasting HDL-C Over Time Through Week 48
timeFrame: Baseline, up to Week 48
measure: Percent Change from Baseline in Fasting Total Apolipoprotein B (ApoB) at Week 24
timeFrame: Baseline, Week 24
measure: Percent Change from Baseline in Fasting Total ApoB Over Time Through Week 48
timeFrame: Baseline, up to Week 48
measure: Percent Change from Baseline in Fasting Low-density Lipoprotein-Cholesterol (LDL-C) Using Ultracentrifugation
timeFrame: Baseline, Week 24
measure: Percent Change from Baseline in Fasting LDL-C Using Ultracentrifugation Over Time Through Week 48
timeFrame: Baseline, up to Week 48
measure: Change from Baseline in Plasma Concentration of ARO-APOC3 Over Time Through Week 48
timeFrame: Baseline, up to Week 48
measure: Number of Participants with Adverse Events (AEs) and/or Serious Adverse Events (SAEs) Possibly or Probably Related to Treatment
timeFrame: up to Week 48
Other outcomes:
Locations:
city: Beverly Hills
country: United States
facility: Westside Medical Associates of Los Angeles
state: California
city: Palm Springs
country: United States
facility: Desert Oasis Healthcare (Desert Medical Group, Inc.)
state: California
city: Boca Raton
country: United States
facility: Preventive Cardiology Inc.
state: Florida
city: Fort Lauderdale
country: United States
facility: Invesclinic U.S., LLC
state: Florida
city: Miami Springs
country: United States
facility: Ocean Blue Medical Research Center Inc.
state: Florida
city: Miami
country: United States
facility: A Positive Research, Inc.
state: Florida
city: Miami
country: United States
facility: Y&L Advance Health care, Inc. D/B/A Elite Clinical Research
state: Florida
city: Miami
country: United States
facility: AppleMed Research Group
state: Florida
city: Miami
country: United States
facility: HeartWell LLP
state: Florida
city: Port Orange
country: United States
facility: Progressive Medical Research
state: Florida
city: Dunwoody
country: United States
facility: Alta Pharmaceutical Research Center
state: Georgia
city: Arlington Heights
country: United States
facility: Northwest Heart Clinical Research
state: Illinois
city: Lexington
country: United States
facility: The Research Group of Lexington, LLC
state: Kentucky
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Tupelo
country: United States
facility: Cardiology Associates Research, LLC
state: Mississippi
city: Kalispell
country: United States
facility: Glacier View Research Institute
state: Montana
city: Omaha
country: United States
facility: Methodist Physicians Clinic Heart Consultants
state: Nebraska
city: Las Vegas
country: United States
facility: Clinical Research of South Nevada
state: Nevada
city: Long Island City
country: United States
facility: NYC Research, Inc.
state: New York
city: New Windsor
country: United States
facility: Mid Hudson Medical Research, PLLC
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai (ISMMS)
state: New York
city: Morehead City
country: United States
facility: Lucas Research, Inc
state: North Carolina
city: Fargo
country: United States
facility: Lillestol Research LLC
state: North Dakota
city: Dayton
country: United States
facility: PriMed Clinical Research
state: Ohio
city: Franklin
country: United States
facility: Prestige Clinical Research
state: Ohio
city: Maumee
country: United States
facility: Advanced Medical Research, LLC
state: Ohio
city: Oklahoma City
country: United States
facility: South Oklahoma Heart Research, LLC
state: Oklahoma
city: Greenville
country: United States
facility: Tribe Clinical Research
state: South Carolina
city: Chattanooga
country: United States
facility: WR-CllinSearch, LLC
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Houston
country: United States
facility: Pioneer Research Solutions, Inc.
state: Texas
city: San Antonio
country: United States
facility: BFHC Research
state: Texas
city: Manassas
country: United States
facility: Manassas Clinical Research Center
state: Virginia
city: Perth
country: Australia
facility: Linear Clinical Research
state: Nedlands
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: Brisbane
country: Australia
facility: Core Research Group
state: Queensland
city: Sippy Downs
country: Australia
facility: University of the Sunshine Coast
state: Queensland
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
state: South Australia
city: Clayton
country: Australia
facility: Monash Health
state: Victoria
city: Concord
country: Canada
facility: LMC Diabetes & Endocrinology
state: Ontario
city: London
country: Canada
facility: Lawson Health Research Institute
state: Ontario
city: Toronto
country: Canada
facility: LMC Manna Research - Bayview
state: Ontario
city: Chicoutimi
country: Canada
facility: Centre d'Etudes Cliniques ECOGENE-21
state: Quebec
city: Greenfield
country: Canada
facility: ViaCar Recherches Cliniques Inc.
state: Quebec
city: Montréal
country: Canada
facility: Institut de Recherches Cliniques de Montreal
state: Quebec
city: Aachen
country: Germany
facility: Uniklinik RWTH
city: Leipzig
country: Germany
facility: Universitatsklinikum Leipzig
city: Baja
country: Hungary
facility: Lausmed Kft.
city: Baja
country: Hungary
facility: Principal SMO Kft
city: Balatonföldvár
country: Hungary
facility: DRC Gyogyszervizsgalo Kozpont Balatonfured
city: Bekescsaba
country: Hungary
facility: Belgyogyaszati - Kardiologiai Maganrendelo
city: Debrecen
country: Hungary
facility: Debreceni Egyetem
city: Gyöngyös
country: Hungary
facility: Pharma4trial Ltd
city: Komárom
country: Hungary
facility: Selye Janos Korhaz
city: Nyiregyhaza
country: Hungary
facility: Borbanya Praxis Kft.
city: Nyíregyháza
country: Hungary
facility: Medifarma-98 Kft.
city: Amsterdam
country: Netherlands
facility: Uni Van Amsterdam Academisch Medisch Centrum
city: Dordrecht
country: Netherlands
facility: Albert Schweitzer Ziekenhuis
city: Sneek
country: Netherlands
facility: D&A Research
city: Grafton
country: New Zealand
facility: Auckland Clinical Studies
state: Auckland
city: Christchurch
country: New Zealand
facility: Lipid and Diabetes Research Group
city: Hamilton
country: New Zealand
facility: Lakeland Clinical Trials - Waikato
city: Papatoetoe
country: New Zealand
facility: Middlemore Clinical Trials
city: Rotorua
country: New Zealand
facility: Lakeland Clinical Trials - Rotorua
city: Bydgoszcz
country: Poland
facility: Spolka Lekarzy Intercor Sp.z.o.o.
city: Elbląg
country: Poland
facility: NZOZ TWOJE ZDROWIE EL Sp.z.o.o.
city: Gdynia
country: Poland
facility: Indywidualna Specjalistyczna Praktyka Lekarska w Dziedzinie Kardiologii lek.
city: Leczyca
country: Poland
facility: Zaklad Opieki Zdrowotnej Leczyca
city: Oświęcim
country: Poland
facility: Medicome Sp. z.o.o.
city: Poznań
country: Poland
facility: Praktyka Lekarska Ewa Krzyzagorska
city: Puławy
country: Poland
facility: KO-MED M Konieczny Cent. Wielosp. Opieki
city: Rzeszów
country: Poland
facility: Centrum Medyczne Medyk
city: Wołomin
country: Poland
facility: Centrum Medyczne K2J2
city: Łódź
country: Poland
facility: All-MED Centrum Medyczne
Study officials:
References: