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Open-Label Study of plozasiran in Adolescents Aged 12 to <18 Years with Familial Chylomicronemia Syndrome

Verified siteRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

The study focuses on Familial Chylomicronemia Syndrome, a rare inherited condition in which the body cannot break down a type of fat called triglycerides. Very high levels of this fat can cause painful inflammation of the pancreas and other complications. The investigational medicine being tested is plozasiran, an experimental drug given by subcutaneous injection. The aim of the trial is to evaluate how the drug works, how it moves through the body, and its safety in adolescents aged 12 to under 18 years.

Participants will receive a series of scheduled injections over approximately one year. At regular visits, blood will be drawn after an overnight fast (no food for at least 10 hours) to measure changes in triglyceride levels and other blood fats. Imaging of the liver and spleen will be performed using ultrasound to check for any signs of fat buildup or inflammation. Questionnaires will be completed to assess pain and overall quality of life throughout the study.

The research process

The trial runs in 8 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment and Consent

    You agree to join the open‑label study that evaluates plozasiran in adolescents with familial chylomicronemia syndrome.

    You sign a consent form that explains the study purpose, procedures, and your rights.

  2. Step 2

    Baseline Evaluations

    You undergo a fasting blood draw to measure triglycerides and other lipid levels.

    An ultrasound of the liver and spleen is performed to assess steatosis, steatohepatitis, and hepato‑splenomegaly.

    You complete questionnaires about pain and quality of life (PedsQL).

  3. Step 3

    First Dose Administration

    You receive a 25 mg dose of plozasiran as a subcutaneous injection using a pre‑filled syringe.

    The injection is given by a qualified study staff member according to the study schedule.

  4. Step 4

    Ongoing Treatment and Monitoring

    You continue to receive the same dose of plozasiran (25 mg, subcutaneous) at each scheduled visit throughout the study period.

    At Months 6, 10, and 12 you have fasting blood draws to assess triglyceride levels and other lipids.

    At Month 12 you have a repeat ultrasound to evaluate liver and spleen status.

  5. Step 5

    Lipid and Triglyceride Assessments

    Your fasting triglyceride (TG) level is measured at baseline and again at Months 6, 10, and 12.

    Additional lipid measurements (non‑HDL‑C, HDL‑C, total cholesterol, LDL‑C, VLDL‑C, apolipoproteins, etc.) are taken at the same time points.

  6. Step 6

    Pain and Quality‑of‑Life Evaluations

    You complete the PedsQL 4.0 Generic Core Scales and the PedsQL Pediatric Pain Questionnaire at baseline, Month 6, and Month 12 to track changes in pain and overall well‑being.

  7. Step 7

    Safety Monitoring

    Throughout the study you are monitored for signs of pancreatitis, abdominal pain, and any hospitalizations related to these conditions.

    Any adverse events are recorded and evaluated by the study team.

  8. Step 8

    Study Completion

    At Month 12 you undergo final blood tests, the final ultrasound, and complete the last set of questionnaires.

    The study concludes for you after the final assessments are completed.

Who can join the trial?

7 criteria

  • You must be between 12 years old and under 18 years old, and you can be a boy or a girl who is not pregnant, not planning to become pregnant, and not breastfeeding.
  • You and a parent or legal guardian must be able and willing to sign written permission forms to take part in the study.
  • Your blood must show very high triglycerides (at least 880 mg/dL, which is about 10 mmol/L) even after you have been on stable cholesterol‑lowering medicines for at least three months; up to two additional tests can be done to confirm this level.
  • You need a confirmed genetic test showing specific mutations that cause Familial Chylomicronemia Syndrome (such as changes in the LPL gene or related genes). If another inherited disorder that affects fats is suspected, the doctor must get special approval before you can join.
  • You must agree to follow a low‑fat diet as advised by the study doctor, keeping your daily fat intake at 20 grams or less.
  • If you are taking medicines for type 2 diabetes or other restricted drugs, the dose must have been unchanged for at least three months before the screening blood test.
  • Women who could become pregnant must use a highly effective method of birth control during the study and for at least 90 days after the last dose; men must use condoms and must not donate sperm for at least 90 days after the study ends.

Who cannot join the trial?

29 criteria

  • Use of any liver‑targeted siRNA medicine that lowers fats or triglycerides in the last 365 days.
  • Use of any other liver‑targeted siRNA or antisense oligonucleotide (ASO) within 60 days or five drug half‑lives before the study.
  • Body weight less than 35 kg (about 77 lb).
  • Having diabetes that was diagnosed within the past 12 weeks or that shows a high HbA1c level (9 % or higher), which means poor blood‑sugar control over the last three months.
  • Having pancreatitis (inflammation of the pancreas) that started within the past 12 weeks.
  • Having had a major surgery (large operation) within the past 12 weeks.
  • Having any of the following lab results at screening:
    • ALT or AST (liver enzymes) three times higher than the normal range.
    • Total bilirubin 1.5 times higher than normal (if you have Gilbert’s syndrome, it must be 3 mg/dL or less).
    • Kidney function measured as eGFR less than 30 mL/min/1.73 m².
    • Persistent protein in the urine (protein/creatinine ratio over 200 mg/day on two tests a week apart).
    • Abnormal clotting tests such as prothrombin time, partial thromboplastin time (PTT), or INR.
    • LDL cholesterol higher than 130 mg/dL.
    • Persistent blood in the urine (more than 5 red blood cells per high‑power field on two tests a week apart).
    • Uncontrolled high blood pressure that is above the 95th percentile for your age, gender, and height; you may be re‑screened after it is treated and controlled.
    • Use of any of the following within 4 weeks before the study:
      • Systemic corticosteroids or anabolic steroids.
      • Plasma apheresis (a procedure that filters blood).
      • Donation of 50–499 mL of blood within 4 weeks, or more than 500 mL within 8 weeks.
      • Being on anti‑retroviral therapy for HIV infection or having a known history of HIV.
      • Having a known history of hepatitis B (positive surface antigen) or hepatitis C (positive antibody confirmed by an RNA test).
      • Having uncontrolled thyroid disease:
        • Primary hypothyroidism (high TSH with low free T4).
        • Secondary hypothyroidism (abnormal TSH levels).
        • History of bleeding problems or a clotting disorder (bleeding diathesis or coagulopathy).
        • Current diagnosis of nephrotic syndrome or chronic kidney disease.
        • History of cancer (malignancy).
        • History of alcoholism or drug abuse within the past year.
        • Active infection that needs antibacterial or antiviral medication and has not been finished before the study starts.
        • Use of any investigational drug or device within 30 days (or five half‑lives) before the study, or participation in another interventional study; if you have previously received plozasiran or ARO‑ANG3, you must wait at least one year.
        • Any other medical, psychiatric, or social condition that the doctor believes would make it difficult to follow the study rules or increase safety risk.
        • Known allergy (hypersensitivity) to plozasiran or any of its ingredients.
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Investigated drugs

plozasiran is an experimental medication being studied for teenagers with familial chylomicronemia syndrome, a rare condition that causes very high blood fats. In this trial, participants receive the drug as a small amount of liquid that is injected just under the skin (subcutaneous injection). The study is looking at how well the medicine works to lower the blood fat levels, how it moves through the body (pharmacokinetics), how it affects the body’s processes (pharmacodynamics), and whether it is safe for young people. This medication is given in a pre‑filled syringe, and it is being tested as a potential new treatment for this serious genetic disorder.

What is already known about the treatment

Redemplo 25 mg solution for injection in pre‑filled syringe - This medication is a 25 mg liquid that is injected under the skin with a ready‑to‑use pre‑filled syringe. It is still an investigational drug, currently being studied in clinical trials and designated as an orphan drug for a rare disease. It is meant to treat familial chylomicronemia syndrome, a condition that causes very high triglyceride levels. The drug uses a small piece of RNA to block the production of a protein that raises triglycerides, and it belongs to the class of RNA‑based (siRNA) therapies.

Investigated diseases

Familial chylomicronemia syndrome - It is a rare inherited disorder in which the body cannot properly break down large fat particles called chylomicrons, resulting in extremely high triglyceride levels. The condition usually appears in childhood or adolescence and leads to episodes of abdominal pain, nausea, and sometimes inflammation of the pancreas. Persistent high triglycerides cause fat to accumulate in the liver and spleen over time. Skin lesions known as eruptive xanthomas may develop on the arms and legs. The disease course is characterized by recurrent flare‑ups that can become more frequent if triglyceride levels remain elevated.
Trial detailsLast updated 11 Nov 2026
Age0-17PhasePhase IIITrial ID2026-526244-12-00Protocol codeAROAPOC3-3007Estimated enrolment9 patientsSponsorArrowhead Pharmaceuticals Inc.

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