Niemann-Pick Type C Disease

Condition Description

Niemann-Pick is a rare, inherited disease that affects the body's ability to metabolize fat (cholesterol and lipids) within cells. These cells malfunction and, over time, die. Niemann-Pick disease can affect the brain, nerves, liver, spleen, bone marrow and, in severe cases, lungs.

People with this condition experience symptoms related to progressive loss of function of nerves, the brain and other organs.

Active Trials

Upcoming Trials

Past Trials

  • TRIAL TITLE: Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

    DESCRIPTION: This study was amended from extended access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks.

    Participants who were already taking adrabetadex will receive their stable dose. Participants who have not ever taken it will start by receiving 400 mg.

    The study design will be like the expanded access by Rush University, but for children at least 4 years of age at screening.

    Participants will receive treatment every 2 weeks until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason. Participants will not receive additional study treatment after their participation in this protocol.

    SPONSOR: Mandos LLC

    INDICATION: Niemann Pick Type C Disease

    STUDY PROTOCOL: VTS-270-001

    PHASE: 4

    STATUS: Completed

    RECRUITING PATIENTS: No