Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

Summary

This study was amended from expanded 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. 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.

Timeline

Start

2018-06-18

Primary completion

2021-11-02

Completion

2021-11-02

Outcome

Outcome not reported

Stopped: “Sponsor had determined that the benefit-risk profile of adrabetadex was not favorable and the clinical development program for adrabetadex was discontinued.”

Drugs

Indications