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

Menkes Disease Clinical Trial

Official title: 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

Status Recruiting

Clinical Trial 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.

Clinical Trial Description

This pilot clinical trial will evaluate the safety, tolerability, dosing, and preliminary efficacy of Northera (Droxidopa) treatment in young adults who survived the major neurodegenerative and neurocognitive effects of Menkes disease through early Copper Histidinate treatment. We hypothesize that Northera (Droxidopa) in Menkes disease survivors with symptoms of dysautonomia (e.g., syncope, dizziness, orthostatic hypotension, abnormal sinoatrial conduction, nocturnal bradycardia, and bowel or bladder dysfunction) from persistent deficiency of the copper-dependent enzyme, dopamine-β-hydroxylase, will be safe, and correct or improve blood neurochemical levels, raise systolic blood pressure, and produce symptomatic improvement and better overall quality of life. We will test this hypothesis in six to ten Menkes disease survivors or OHS patients in a double-blind placebo-controlled randomized crossover clinical trial. ;

Related Conditions & MeSH terms

• Autonomic Nervous System Diseases
• Cutis Laxa
• Ehlers-Danlos Syndrome
• Menkes Disease
• Occipital Horn Syndrome
• Primary Dysautonomias
• Syndrome

• NCT number: NCT04977388
• Study type: Interventional
• Source: Nationwide Children's Hospital
• Contact: Stephen G. Kaler, MD
• Phone: 614 722-5964
• Email: stephen.kaler@nationwidechildrens.org
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: July 12, 2021
• Completion date: June 30, 2026