At a glance
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Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Adults With Occipital Horn Syndrome: Double-blind Placebo-controlled Randomized Crossover Clinical Trial
In Brief
A Phase 2 clinical trial evaluating Droxidopa and Placebo for Menkes Disease and Occipital Horn Syndrome. Completed, enrolled 3 participants across 1 site.
Detailed 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.
Study Details
Timeline
Interventions
Subjects will self-administer capsules of Droxidopa by mouth twice daily for six weeks.
Subjects will self-administer capsules of placebo by mouth twice daily for six weeks.