Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS)
- Conditions
- Familial Chylomicronemia
- Interventions
- Drug: Placebo
- Registration Number
- NCT05089084
- Lead Sponsor
- Arrowhead Pharmaceuticals
- Brief Summary
The purpose of AROAPOC3-3001 is to evaluate the efficacy and safety of ARO-APOC3 plozasiran) in adult participants with familial chylomicronemia syndrome (FCS). Participants who have met all eligibility criteria will be randomized to receive 4 doses of plozasiran or matching placebo administered subcutaneously. Participants who complete the randomized period will continue in a 2-year open-label extension period where all participants will receive plozasiran.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 75
- 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
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo calculated volume to match active treatment by sc injection (randomized period) ARO-APOC3 (plozasiran) Plozasiran 4 doses of plozasiran by subcutaneous (sc) injection (randomized period) 8 doses of plozasiran by sc injection (open-label period)
- Primary Outcome Measures
Name Time Method Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10 Baseline, Month 10
- Secondary Outcome Measures
Name Time Method Percent Change from Baseline in Fasting APOC3 at Month 12 Baseline, Month 12 Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10 Baseline, Month 10 Percent Change from Baseline in Fasting TG at Month 10 and Month 12 (Averaged) Baseline, Month 10, Month 12 Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10 Baseline, Month 10 Percent Change from Baseline in Fasting Non-HDL-C at Month 12 Baseline, Month 12 Proportion of Patients Achieving TG of < 500 mg/dL at Month 12 Month 12 Percent Change from Baseline in Fasting TG Over Time Baseline, up through Month 12 Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs) From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period) Number of Participants with Positively Adjudicated Events of Acute Pancreatitis From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period) Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10 Baseline, Month 10 Percent Change from Baseline in Fasting TG at Month 12 Baseline, Month 12 Percent Change from Baseline in Fasting HDL-C at Month 12 Baseline, Month 12 Proportion of Patients Achieving TG of < 500 mg/dL at Month 10 Month 10 Change from Baseline in Fasting TG Over Time Baseline, up through Month 12
Trial Locations
- Locations (58)
Instytut Centrum Zdrowia Matki Polki
🇵🇱Łódź, Poland
Excel Medical Clinical Trials, LLC
🇺🇸Boca Raton, Florida, United States
Ascension St. Vincent Cardiovascular Research Institute
🇺🇸Indianapolis, Indiana, United States
Centennial Medical Group
🇺🇸Elkridge, Maryland, United States
Icahn School of Medicine at Mt. Sinai
🇺🇸New York, New York, United States
Austin Health
🇦🇺Melbourne, Australia
Hadassah Medical Center Ein Karem
🇮🇱Jerusalem, Israel
National Institute of Medical Sciences and Nutrition - Salvador Zubiran (INCMNSZ)
🇲🇽Tlalpan, Mexico DF, Mexico
New York University Langone Medical Center
🇺🇸New York, New York, United States
Instituto de Diabetes Obesidad y Nutrición S.C.
🇲🇽Cuernavaca, Morelos,, Mexico
Washington University School of Medicine, Division of Endocrinology, Metabolism and Lipid Research
🇺🇸Saint Louis, Missouri, United States
Robarts Research Institute
🇨🇦London, Ontario, Canada
Toronto General Hospital
🇨🇦Toronto, Ontario, Canada
York Clinical Research, LLC
🇺🇸Norfolk, Virginia, United States
Linear Clinical Research Ltd
🇦🇺Nedlands, Australia
Complejo Hospitalario Universitario de Granada - Hospital Universitario Virgen de las Nieves
🇪🇸Granada, Spain
Centro Especializado en Diabetes Obesidad y Prevención de entermedades Cardiovasculares (CEDOPEC)
🇲🇽Mexico City, Mexico
NZCR OPCO Ltd
🇳🇿Auckland, New Zealand
Hospital Abente y Lago
🇪🇸A Coruña, Spain
Hospital General Universitario Gregorio Maranon
🇪🇸Madrid, Spain
Herman Clinical Research, LLC
🇺🇸Suwanee, Georgia, United States
Baker Heart and Diabetes Institute
🇦🇺Melbourne, Victoria, Australia
Royal Prince Alfred Hospital
🇦🇺Camperdown, New South Wales, Australia
Royal North Shore Hospital
🇦🇺St. Leonards, New South Wales, Australia
Universitair Ziekenhuis Antwerpen (UZA)
🇧🇪Edegem, Belgium
Universitaire Ziekenhuizen Leuven
🇧🇪Leuven, Belgium
Ecogene-21
🇨🇦Chicoutimi, Quebec, Canada
Clinique des Maladies Lipidiques de Quebec Inc.
🇨🇦Québec, Quebec, Canada
Universitaetsklinikum Leipzig
🇩🇪Leipzig, Germany
University Hospital Galway
🇮🇪Galway, Ireland
Rinku General Medical Center
🇯🇵Osaka, Japan
Middlemore Clinical Trials
🇳🇿Auckland, New Zealand
Seoul National University Hospital
🇰🇷Seoul, Korea, Republic of
Chonnam National University Hospital
🇰🇷Gwangju, Korea, Republic of
Hospital Clinico Universitario de Santiago
🇪🇸Santiago De Compostela, Spain
Erciyes University Faculty of Medicine
🇹🇷Melikgazi, Kayseri, Turkey
Ege University Hospital Department Of Infectious Diseases
🇹🇷İzmir, Turkey
Clinical Centre Nis
🇷🇸Niš, Serbia
Medizinische Universitaet Graz
🇦🇹Graz, Austria
Institute de Recherches Cliniques de Montreal
🇨🇦Montreal, Quebec, Canada
New Zealand Clinical Research
🇳🇿Christchurch, New Zealand
University Hospital Ghent
🇧🇪Ghent, Belgium
Tokyo University Hospital
🇯🇵Tokyo, Japan
Universitaetsklinikum Jena
🇩🇪Jena, Germany
Nippon Medical School Hospital
🇯🇵Tokyo, Japan
Instituto Medico DAMIC
🇦🇷Córdoba, Argentina
Instituto Modelo de Gastroenterologia
🇦🇷Formosa, Argentina
Centre Hospitalier Universitaire (CHU) de Liege
🇧🇪Liège, Belgium
AP-HM-Hopital de La Conception
🇫🇷Marseille, Cedez 05, France
Chiba University Hospital
🇯🇵Chiba, Japan
Kanazawa University Hospital
🇯🇵Ishikawa, Japan
Jichi Medical University Hospital
🇯🇵Tochigi, Japan
Sultan Qaboos University Hospital
🇴🇲Muscat, Oman
Clinical Center of Serbia, Institute of Endocrinology, Diabetes and Metabolic Diseases
🇷🇸Belgrade, Serbia
National University Hospital
🇸🇬Singapore, Singapore
University Hospital Center Zagreb -Rebro, Department for Metabolic Diseases
🇭🇷Zagreb, Croatia
AP-HP Hopital Pitie-Salpetriere
🇫🇷Paris, France
Texas Diabetes and Endocrinology
🇺🇸Austin, Texas, United States