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New shot could tame rare 'Fat Blood' disease and prevent pancreatitis

NCT ID NCT05089084

Disease control Sponsor: Arrowhead Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 study tested a drug called plozasiran (ARO-APOC3) in 75 adults with familial chylomicronemia syndrome, a rare genetic disorder causing extremely high blood fat levels and risk of pancreatitis. Participants received four injections of the drug or a placebo over several months. The main goal was to see if the drug safely lowers triglycerides and reduces pancreatitis attacks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Plozasiran (ARO-APOC3) injection
What this could lead to
If it works, this could provide a new treatment to dramatically lower triglycerides and reduce the risk of painful, life-threatening pancreatitis attacks in people with familial chylomicronemia syndrome.
What could go wrong
This is a completed Phase 3 trial with only 75 participants, so results may not apply to everyone. The drug requires ongoing injections and may have side effects; long-term safety and effectiveness are still being studied in an open-label extension.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

75 people

The number who actually took part.

Started

Dec 2021

Finished

Apr 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Fasting triglycerides (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

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Conditions

The condition(s) this trial relates to.

familial chylomicronemia syndrome familial lipoprotein lipase deficiency

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Familial chylomicronemia

Contacts and locations

Locations

  • Clinical Site 1

    Boca Raton, Florida, 33434, United States

  • Clinical Site 10

    Córdoba, X5003DCE, Argentina

  • Clinical Site 11

    Formosa, 3600, Argentina

  • Clinical Site 12

    Melbourne, 3081, Australia

  • Clinical Site 13

    Nedlands, 6009, Australia

  • Clinical Site 14

    Camperdown, New South Wales, 2050, Australia

  • Clinical Site 15

    St Leonards, New South Wales, 2065, Australia

  • Clinical Site 16

    Melbourne, Victoria, 3004, Australia

  • Clinical Site 17

    Graz, 8036, Austria

  • Clinical Site 18

    Edegem, 2650, Belgium

  • Clinical Site 19

    Ghent, 9000, Belgium

  • Clinical Site 2

    Suwanee, Georgia, 30024, United States

  • Clinical Site 20

    Leuven, 3000, Belgium

  • Clinical Site 21

    Liège, 4000, Belgium

  • Clinical Site 22

    London, Ontario, N6A 5B7, Canada

  • Clinical Site 23

    Toronto, Ontario, M5G 2C4, Canada

  • Clinical Site 24

    Chicoutimi, Quebec, G7H 7K9, Canada

  • Clinical Site 25

    Montreal, Quebec, H2W 1R7, Canada

  • Clinical Site 26

    Québec, Quebec, G1V 4W2, Canada

  • Clinical Site 27

    Zagreb, 10000, Croatia

  • Clinical Site 28

    Paris, 75013, France

  • Clinical Site 29

    Marseille, Cedez 05, 13385, France

  • Clinical Site 3

    Indianapolis, Indiana, 46290, United States

  • Clinical Site 30

    Jena, 7740, Germany

  • Clinical Site 31

    Leipzig, 4103, Germany

  • Clinical Site 32

    Galway, H91 YR71, Ireland

  • Clinical Site 33

    Jerusalem, 9112001, Israel

  • Clinical Site 34

    Chiba, 260-8677, Japan

  • Clinical Site 35

    Ishikawa, 920-8641, Japan

  • Clinical Site 36

    Osaka, 598-0048, Japan

  • Clinical Site 37

    Tochigi, 329-0498, Japan

  • Clinical Site 38

    Tokyo, 113-8655, Japan

  • Clinical Site 39

    Tokyo, Japan

  • Clinical Site 4

    Elkridge, Maryland, 21075, United States

  • Clinical Site 40

    Mexico City, 11650, Mexico

  • Clinical Site 41

    Tlalpan, Mexico DF, 14000, Mexico

  • Clinical Site 42

    Cuernavaca, Morelos, 62250, Mexico

  • Clinical Site 43

    Auckland, 2025, New Zealand

  • Clinical Site 44

    Auckland, 1010, New Zealand

  • Clinical Site 45

    Christchurch, 8011, New Zealand

  • Clinical Site 46

    Muscat, Oman

  • Clinical Site 47

    Lodz, 93-338, Poland

  • Clinical Site 48

    Belgrade, 11000, Serbia

  • Clinical Site 49

    Niš, 18000, Serbia

  • Clinical Site 5

    St Louis, Missouri, 63110, United States

  • Clinical Site 50

    Singapore, 119074, Singapore

  • Clinical Site 51

    Gwangju, 61469, South Korea

  • Clinical Site 52

    Seoul, 03080, South Korea

  • Clinical Site 53

    A Coruña, 15001, Spain

  • Clinical Site 54

    Granada, 18012, Spain

  • Clinical Site 55

    Madrid, 28007, Spain

  • Clinical Site 56

    Santiago de Compostela, 15706, Spain

  • Clinical Site 57

    Izmir, 35100, Turkey (Türkiye)

  • Clinical Site 58

    Melikgazi, Kayseri, 38030, Turkey (Türkiye)

  • Clinical Site 6

    New York, New York, 10029, United States

  • Clinical Site 7

    New York, New York, 10016, United States

  • Clinical Site 8

    Austin, Texas, 78731, United States

  • Clinical Site 9

    Norfolk, Virginia, 23510, United States

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