CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 3 enrolled
Drug / intervention
Droxidopa +1 moredrug
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT04977388
NCT04977388Phase 2Completed

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

Stephen G. Kaler, MD·interventional·Posted Jul 26, 2021·Updated Apr 13, 2026

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

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
20222023202420252026
First PostedJul 26, 2021
Enrollment StartJul 12, 2021
Primary CompletionOct 30, 2023
Study CompletionJun 29, 2024
TodayJul 2, 2026
Enrollment to primary: 2.3 yearsPosted 4.9 years ago

Interventions

Droxidopadrug

Subjects will self-administer capsules of Droxidopa by mouth twice daily for six weeks.

Placeboother

Subjects will self-administer capsules of placebo by mouth twice daily for six weeks.