A Randomized Double-Blind Placebo-Controlled Phase 3 Study of AKCEA-APOCIII-LRx Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS)

Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions:
    Familial Chylomicronemia
  • Age: Between 18 - 99 Years
  • Gender: Male or Female
  • Other Inclusion Criteria:
    1. Must have given written informed consent
    2. A diagnosis of Familial Chylomicronemia Syndrome (type 1 Hyperlipoproteinemia)
    3. History of pancreatitis within 10 years prior
    4. Willing to follow a diet comprising = 20g fat per day during the study
    5. Willing to complete the FCS Symptoms and diet questionnaire on a daily basis for FCS symptoms/diet
    6. Females must be non-pregnant and non-lactating and either surgically sterile and/or post-menopausal or abstinent.
    7. Males are surgically sterile or uses highly effective contraceptive method if engaged in sexual relations.
    8. Participants on stable doses of atypical antipsychotic medications for at least 3 months prior
    9. Participants on stable doses of concomitant medications listed in the protocol for at least 4 weeks prior.

You may not be eligible for this study if the following are true:

    1. Clinically significant abnormalities in medical history or physical examination
    2. Active pancreatitis
    3. Uncontrolled arterial hypertension
    4. History of bleeding diathesis or coagulopathy or clinically significant abnormality in coagulation parameters
    5. Active infection requiring systemic antiviral or antimicrobial therapy
    6. Active infection with human immunodeficiency virus (HIV), hepatitis C or hepatitis C
    7. Malignancy within 5 years
    8. Blood donation of 50 to 499 mL within 30 days
    9. Treatment with another investigational drug (non-oligonucleotide), biological agent, or device within 1 month
    10. Previous treatment with an oligonucleotide (including siRNA) within 4 months
    11. Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator

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