Overview

NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome

Status:
Recruiting
Trial end date:
2026-06-30
Target enrollment:
Participant gender:
Summary
The purpose of this study is to evaluate whether Northera (Droxidopa) is safe and effective in young adults with Menkes disease who survived the most severe complications of their illness or adults with occipital horn syndrome (OHS), who have trouble with intermittent low blood pressure and other symptoms of dysautonomia. The outcomes and information from this study may help adult survivors of Menkes disease and individuals with OHS lead more normal day-to-day lives.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Stephen G. Kaler, MD
Treatments:
Droxidopa