Study of ARO-APOC3 (Plozasiran) in Adults With Familial Chylomicronemia Syndrome (FCS) (PALISADE)
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| ClinicalTrials.gov Identifier: NCT05089084 |
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Recruitment Status :
Active, not recruiting
First Posted : October 22, 2021
Last Update Posted : May 2, 2024
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Sponsor:
Arrowhead Pharmaceuticals
Information provided by (Responsible Party):
Arrowhead Pharmaceuticals
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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.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Familial Chylomicronemia | Drug: Plozasiran Drug: Placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 75 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults With Familial Chylomicronemia Syndrome |
| Actual Study Start Date : | January 11, 2022 |
| Actual Primary Completion Date : | April 29, 2024 |
| Estimated Study Completion Date : | April 2026 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Familial lipoprotein lipase deficiency
MedlinePlus related topics:
Family Issues
| Arm | Intervention/treatment |
|---|---|
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Experimental: ARO-APOC3 (plozasiran)
4 doses of plozasiran by subcutaneous (sc) injection (randomized period) 8 doses of plozasiran by sc injection (open-label period) |
Drug: Plozasiran
ARO-APOC3 injection
Other Name: ARO-APOC3 |
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Placebo Comparator: Placebo
calculated volume to match active treatment by sc injection (randomized period)
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Drug: Placebo
sterile normal saline (0.9% NaCl) |
Primary Outcome Measures :
- Percent Change from Baseline in Fasting Triglycerides (TG) at Month 10 [ Time Frame: Baseline, Month 10 ]
Secondary Outcome Measures :
- Percent Change from Baseline in Fasting TG at Month 10 and Month 12 (Averaged) [ Time Frame: Baseline, Month 10, Month 12 ]
- Percent Change from Baseline in Apolipoprotein C-III (APOC3) at Month 10 [ Time Frame: Baseline, Month 10 ]
- Percent Change from Baseline in Fasting APOC3 at Month 12 [ Time Frame: Baseline, Month 12 ]
- Percent Change from Baseline in Non-High Density Lipoprotein Cholesterol (Non-HDL-C) at Month 10 [ Time Frame: Baseline, Month 10 ]
- Percent Change from Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Month 10 [ Time Frame: Baseline, Month 10 ]
- Percent Change from Baseline in Fasting TG at Month 12 [ Time Frame: Baseline, Month 12 ]
- Percent Change from Baseline in Fasting Non-HDL-C at Month 12 [ Time Frame: Baseline, Month 12 ]
- Percent Change from Baseline in Fasting HDL-C at Month 12 [ Time Frame: Baseline, Month 12 ]
- Proportion of Patients Achieving TG of < 500 mg/dL at Month 10 [ Time Frame: Month 10 ]
- Proportion of Patients Achieving TG of < 500 mg/dL at Month 12 [ Time Frame: Month 12 ]
- Change from Baseline in Fasting TG Over Time [ Time Frame: Baseline, up through Month 12 ]
- Percent Change from Baseline in Fasting TG Over Time [ Time Frame: Baseline, up through Month 12 ]
- Number of Participants with Treatment-Emergent Adverse Events (AEs) and/or Serious Adverse Events (SAEs) [ Time Frame: 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 [ Time Frame: From first dose of study drug through Month 12 (Randomized Period) and through Month 36 (Open-label Period) ]
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| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
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
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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05089084
Locations
Show 58 study locations
Sponsors and Collaborators
Arrowhead Pharmaceuticals
| Responsible Party: | Arrowhead Pharmaceuticals |
| ClinicalTrials.gov Identifier: | NCT05089084 |
| Other Study ID Numbers: |
AROAPOC3-3001 |
| First Posted: | October 22, 2021 Key Record Dates |
| Last Update Posted: | May 2, 2024 |
| Last Verified: | April 2024 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Additional relevant MeSH terms:
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Hyperlipoproteinemia Type I Lipid Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Hyperlipoproteinemias |
Hyperlipidemias Dyslipidemias Lipid Metabolism Disorders Metabolic Diseases |