Actively Recruiting
Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease: Double-blind Placebo-controlled Randomized Crossover Clinical Trial
Led by Stephen G. Kaler · Updated on 2026-02-10
6
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, dosing, and effectiveness of Northera (Droxidopa) in children aged 7 to 17 years who survived the major neurodegenerative effects of Menkes disease after early treatment with Copper Histidinate. This clinical trial focuses on pediatric survivors who experience symptoms of dysautonomia, such as dizziness, orthostatic hypotension, and bowel or bladder dysfunction, caused by a deficiency of the enzyme dopamine-beta-hydroxylase. The study aims to improve blood neurochemical levels, increase systolic blood pressure, and enhance quality of life. Participants will receive oral doses of Droxidopa or matching placebo capsules in a double-blind, randomized crossover design. Doses will be carefully adjusted for each child, with the medication given twice daily in a liquid suspension form prepared from capsules and simple syrup. Each treatment period lasts four weeks, with two treatment phases assessing active drug and placebo effects. The study will also validate a questionnaire assessing orthostatic hypotension symptoms for this population. During the trial, children will be closely monitored for safety and treatment effects through blood tests measuring neurochemical levels, blood pressure readings, and physical tests such as standing time and six-minute walk distance. Researchers will track any adverse events over ten weeks and evaluate symptoms through questionnaires. Participants will attend multiple study visits and are expected to follow the prescribed medication schedule while undergoing assessments of their physical and neurological function.
CONDITIONS
Brief Title
Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Children or adolescents aged 7 to 17 years with Menkes disease who survived beyond expected natural history after early Copper Histidinate treatment
- Participants must have clinical signs and symptoms of dysautonomia, including orthostatic hypotension or chronic diarrhea lasting more than four weeks
- History of at least thrice weekly dizziness or diarrhea episodes for more than four weeks
- Documented ATP7A gene mutation
- One parent must sign informed consent and patient must assent
- Ability to adhere to oral Northera (Droxidopa) regimen
- Willingness to comply with all study visits and procedures
You will not qualify if you...
- Pre-existing liver diseases such as hepatitis, biliary atresia, or cirrhosis
- Pre-existing kidney disease with glomerular filtration rate below 30 ml/min
- History of age-adjusted stage 1 hypertension (≥ 95th percentile)
- History of anti-hypertensive therapy, heart failure, cardiac arrhythmia, or bleeding disorders
- Use of medications including alpha-1 adrenoreceptor agonists, beta-blockers, DOPA decarboxylase inhibitors, midodrine, ephedrine, or triptans
- Any condition likely to prevent completion of the study or compliance with study requirements
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 4 weeks per treatment period
Participants receive oral doses of Droxidopa or placebo twice daily in a randomized crossover design to evaluate safety and efficacy for dysautonomia symptoms.
1 baseline visit and 1 follow-up visit per treatment period
Trial Site Locations
Total: 1 location
1
Vagelos College of Physicians and Surgeons, Columbia University
New York, New York, United States, 10032
Actively Recruiting
Research Team
S
Stephen G Kaler, MD, MPH
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
OTHER
Number of Arms
2
Frequently Asked Questions
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