Study of Northera Droxidopa for Treating Dysautonomia in Children and Teens with Menkes Disease Phase III, Double-blind, Placebo-controlled, Randomized Crossover Trial

What this Trial Is About

Researchers are evaluating the safety, tolerability, dosing, and efficacy of Northera Droxidopa in children aged 7 to 17 years with Menkes disease who survived major neurodegenerative effects through early Copper Histidinate treatment. The study focuses on pediatric survivors experiencing dysautonomia symptoms such as dizziness, fainting, low blood pressure, and bowel or bladder issues caused by dopamine-beta-hydroxylase deficiency. This Phase III clinical trial aims to address an important unmet need in this rare disease population. Participants receive oral Northera Droxidopa or placebo capsules indistinguishable from each other in a double-blind, randomized, crossover design. The study uses individualized dose titration of droxidopa, taken twice daily in a freshly prepared liquid suspension. Two four-week treatment periods alternate between active drug and placebo to assess effects on blood neurochemicals, blood pressure, and physical performance. The Orthostatic Hypotension Symptom Assessment OHSA questionnaire is validated during each treatment period. During the trial, participants undergo safety monitoring for serious adverse events over 10 weeks. Researchers measure plasma norepinephrine and dihydroxyphenylglycol levels, systolic blood pressure, daily bowel movements, standing time, and six-minute walk distance at baseline and after each intervention period. The study involves multiple visits to ensure adherence and collect data to determine whether droxidopa improves symptoms of dysautonomia and quality of life in pediatric Menkes disease survivors.

NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease