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Can a new shot tame a rare fat disorder in kids?

NCT ID NCT07727538

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 27, 2026 · Last updated Aug 27, 2026 · Updated 7 times

Summary

This trial tests whether olezarsen, an experimental drug given as a weekly injection, can lower dangerously high blood fat levels in children with familial chylomicronemia syndrome (FCS). FCS is a rare genetic condition that prevents the body from breaking down fats, leading to severe pancreatitis risk. The study enrolls children aged 2 to 17 and measures changes in triglyceride levels and safety over time.

What this could mean

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

Active substance
an experimental drug called olezarsen, given as a shot under the skin
What this could lead to
If it works, this could offer a treatment option for children with a rare, severe fat-processing disorder that currently has few therapies.
What could go wrong
This is a small, early-phase trial with only 12 participants, so results may not apply broadly. The drug may cause side effects or fail to lower triglycerides enough.

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

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2026

Expected to finish

Aug 2031

An estimate. End dates often move.

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

2 to 17 years

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.

Key Inclusion Criteria: 1. Parental or legally authorized representative consent must be obtained, and the participants must provide age-appropriate or cognition-appropriate assent, as determined by the Investigator. The parent or legal guardian must be able to understand and comply with the study visit schedule and all other study procedures. 2. Must be able to comply with all study procedures. 3. Age 12 to less than 18 years at the time of informed consent/assent (Cohort 1); age 2 to less than 12 years at time of informed consent/assent (Cohort 2). 4. Willing to fast for at least 10 hours before visits requiring fasted blood sampling. 5. A diagnosis of Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia) by documentation of confirmed homozygote, compound heterozygote or double heterozygote for loss-of-function mutations in type 1-causing genes. 6. Fasting TGs ≥880 mg/dL at screening. If fasting TG is \< 880 mg/dL, up to two additional tests may be performed during the screening period with any single test used to qualify. Key Exclusion Criteria: 1. Diabetes mellitus with any of the following: 1. Newly diagnosed within 12 weeks prior to screening or during the screening period. 2. Hemoglobin A1c (HbA1c) ≥9.5% at screening. 3. Change in basal insulin regimen \>20% within 3 months prior to screening or during the screening period. 4. Type 1 diabetes. 2. History of bleeding, diathesis, or coagulopathy. 3. Major surgery within 3 months of screening. 4. Plasma apheresis within 4 weeks prior to screening or planned during the study. 5. Treatment with another investigational drug, biological agent, or device within one month of screening, or 5 half-lives of investigational agent, whichever is longer. 6. Active pancreatitis within 4 weeks prior to screening or during the screening period. 7. Malignancy diagnosed or treated within 5 years prior to screening or during the screening period. Note: Other protocol-specified inclusion/exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

familial chylomicronemia syndrome Familial hyperchylomicronemia syndrome

As listed by the trial registrant

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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    4 sites. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Cook Children's Medical Center

    RECRUITING

    Fort Worth, Texas, 76104, United States

  • UCSF Benioff Children's Hospital

    RECRUITING

    San Francisco, California, 94143, United States

  • University of Alabama

    RECRUITING

    Birmingham, Alabama, 35294, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

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