Last updated on June 2020

Adrabetadex for Patients With Nerve Symptoms of Niemann-Pick Type C Disease (NPC)

Brief description of study

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.

Clinical Study Identifier: NCT03643562

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University of Arkansas System

Little Rock, AR United States
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Recruitment Status: Open

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