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

NCT ID NCT05089084

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

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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