Can a blood pressure drug help kids with menkes disease?
NCT ID NCT07398508
First seen Jun 27, 2026 · Last updated Jul 21, 2026 · Updated 2 times
Summary
This study tests whether Northera (droxidopa) is safe and can improve symptoms like dizziness, fainting, and low blood pressure in children aged 7 to 17 with Menkes disease. Six to ten participants will receive either the drug or a placebo in two four-week periods, with careful dose adjustments. The goal is to see if the drug raises blood pressure and improves quality of life.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Northera (droxidopa)
- What this could lead to
- If it works, this could point toward a treatment that improves daily life for children with Menkes disease who have symptoms like dizziness and fainting.
- What could go wrong
- This is a very small early-phase trial with only 6 participants, so results may not apply broadly. The drug may not work better than placebo, and side effects are unknown in this group.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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About 6 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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Jun 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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7 to 17 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
1 site. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Vagelos College of Physicians and Surgeons, Columbia University
RECRUITINGNew York, New York, 10032, United States
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