Northera (droxidopa) for dysautonomia in children who survived Menkes disease
Official title: Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease: Double-blind Placebo-controlled Randomized Crossover Clinical Trial
Run by Columbia University · NCT07398508 · Data updated 2026-11-12
This trial will test whether the oral medicine Northera (droxidopa) is safe and helps reduce fainting, dizziness, low blood pressure, and other dysautonomia symptoms in children and adolescents (ages 7–17) who survived Menkes disease after early copper treatment.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 7 Years to 17 Years |
| Sex | All |
| Enrollment | 6 (estimated) |
| Sponsor | Columbia University Academic / other |
| Locations | 1 site (New York, New York) |
| Status | Recruiting |
| Started | August 10, 2026 |
| Main results expected | June 30, 2030 |
| Study ends | June 30, 2030 |
| Registry record updated | October 5, 2026 |
| Trial ID | NCT07398508 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are ambulatory children or adolescents aged 7–17 with Menkes disease who received early copper histidinate, have a documented ATP7A mutation, attend school, and have recurrent symptoms of dysautonomia such as orthostatic hypotension, frequent dizziness, or chronic diarrhea.
✕Probably not a fit
Patients without dysautonomia symptoms, those outside the 7–17 age range, nonambulatory individuals, or those with contraindications to droxidopa or without prior early copper treatment are unlikely to benefit from this specific trial.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This Phase I/II, placebo-controlled trial will enroll about six to ten pediatric Menkes disease survivors to study safety, tolerability, dosing, and preliminary efficacy of oral droxidopa. Participants must be aged 7–17, have a documented ATP7A mutation, have survived following early copper histidinate treatment, and show clinical signs of dysautonomia such as orthostatic hypotension, frequent dizziness, or chronic diarrhea. The study will compare droxidopa versus placebo, monitor adverse events, measure blood neurochemistry and systolic blood pressure, and record symptomatic and quality-of-life changes. Results will inform whether droxidopa corrects neurochemical deficits, raises blood pressure, and produces clinically meaningful symptom improvement in this population.
Why it matters
Potential benefit: If successful, Northera could raise blood pressure, reduce episodes of fainting and dizziness, and improve daily functioning and quality of life for pediatric Menkes survivors with dysautonomia.
How similar studies have gone: Droxidopa has demonstrated benefit for neurogenic orthostatic hypotension in adults, but its use specifically in pediatric Menkes survivors is novel and has not been established in larger studies.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Children or adolescents with Menkes disease who survived beyond the expected natural history, attained independent ambulation, and attend school after early CuHis treatment for three years, who manifest clinical signs and symptoms of dysautonomia, e.g., orthostatic hypotension: specifically, a decrease in systolic or diastolic blood pressure of at least 20 or 10 mm Hg, respectively, within three minutes after standing, and/or chronic diarrhea: production of loose stools with or without increased stool frequency for more than four weeks immediately preceding enrollment. 2. History of at least thrice weekly occurrence of dizziness/feeling lightheaded while standing upright and/or thrice weekly episodes of diarrhea or an urgent need to defecate after food ingestion for more than four weeks immediately preceding enrollment. 3. Documented mutation in ATP7A. 4. One parent must sign and date an Informed Consent Form (ICF) and patient must also assent. 5. Age 7 to 17 years. (Enrollment will be staggered so that at least the first two children enrolled are aged 12-17 years.) 6. Ability to adhere to the prescribed oral Northera (Droxidopa) regimen. 7. Willingness to comply with all study visits and procedures. Exclusion Criteria: 1. Pre-existing liver (e.g., hepatitis, biliary atresia, cirrhosis) or kidney disease (i.e., calculated glomerular filtration rate \<30 ml/min). 2. History of age-adjusted stage 1 hypertension (≥ 95th percentile) \[1\] (Also see Attachment A). 3. History of anti-hypertensive therapy, heart failure (or decreased ejection fraction), cardiac arrhythmia, or bleeding diatheses. 4. Any disease or condition that, in the opinion of the Investigator, has a high probability of precluding the subject from completing the study or where the subject cannot or will not appropriately comply with study requirements. 5. Any alpha-1 adrenoreceptor agonist, beta-blocker, DOPA decarboxylase inhibitor, midodrine, ephedrine, or any triptan medication as a concomitant medication.
Where it's running
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Vagelos College of Physicians and Surgeons, Columbia University
New York, New York, United States Recruiting
Study contacts
- Principal investigator: Stephen G Kaler, MD, MPH — Columbia University
- Study coordinator: Stephen G Kaler, MD, MPH
- Email: sgk2141@cumc.columbia.edu
- Phone: 212 305-3669
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.