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NCT07398508 · ClinicalTrials.gov registry record · Phase 1
Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease.
A Phase 1 study of Menkes Disease, sponsored by Stephen G. Kaler.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 6
- Enrollment target
- 1
- Study location
NCT07398508 is a Phase 1 study of Menkes Disease that is actively recruiting participants, run by Stephen G. Kaler. The registered enrollment target is 6 participants. The trial reports 1 study location across 1 state.
The verdict
NCT07398508, a Phase 1 study of Menkes Disease, is actively recruiting participants, sponsored by Stephen G. Kaler.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 6 participants
- Enrollment target
- 1
- Study location
Study Summary
This clinical trial will evaluate the safety, tolerability, dosing, and efficacy of Northera (Droxidopa) in children with Menkes disease aged 7 to 17 years who survived the major neurodegenerative and neurocognitive effects of Menkes disease through early Copper Histidinate treatment. The investigator hypothesizes that Northera (Droxidopa) treatment in pediatric Menkes disease survivors with symptoms of dysautonomia (e.g., syncope, dizziness, orthostatic hypotension, abnormal sinoatrial conduction, and bowel or bladder dysfunction) from deficiency of the cuproenzyme, dopamine-beta-hydroxylase, will be safe and will correct or improve blood neurochemical levels, raise systolic blood pressure, and produce symptomatic improvement and a better quality of life. The investigator will test this hypothesis, in six to ten child or adolescent Menkes disease survivors through a placebo-controlled trial to evaluate adverse event rates and whether oral administration of Northera (Droxidopa) at doses established for individual subjects by careful dose titration improves plasma norepinephrine and dihydroxyphenylglycol (DHPG) levels, raises systolic blood pressure, and improves performance on tests of physical exertion. As an exploratory outcome measure, the study will validate the Orthostatic Hypotension Symptom Assessment (OHSA) questionnaire for this population for two four-week periods of either active or placebo treatment. Aim 1. Determine the safety of Droxidopa in Menkes disease pediatric survivors. Aim 2. Determine the efficacy of Droxidopa in Menkes disease survivors. The investigator hypothesizes that low-dose Droxidopa treatment in classic Menkes disease survivors aged 7 to 17 will improve orthostatic hypotension and ameliorate other signs and symptoms of dysautonomia. This pilot study will employ an ascending dose paradigm in a double-blind placebo-controlled randomized crossover design to optimize statistical power and rigorously discern treatment effects on 1) tilt
Conditions Studied
Interventions
- DRUG Droxidopa Oral Product
- OTHER Placebo Control
Study Locations (1)
New York
- Vagelos College of Physicians and Surgeons, Columbia University - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 6 participants |
| Start Date | 2026-06-01 |
| Est. Completion | 2030-06-30 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT07398508
The ClinicalTrials.gov registry entry for NCT07398508 describes a study currently listed as recruiting, categorized as Phase 1. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Stephen G. Kaler, which has 1 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Menkes Disease appearing as the primary indexed condition, and to 2 interventions - of which Droxidopa Oral Product is the first listed.
NCT07398508 reports 1 study location spanning 1 distinct geographic area - top geographies include New York.
Frequently Asked Questions
What is clinical trial NCT07398508 about?
NCT07398508 is a clinical study titled "Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease.". This clinical trial will evaluate the safety, tolerability, dosing, and efficacy of Northera (Droxidopa) in children with Menkes disease aged 7 to 17 years who survived the major neurodegenerative and neurocognitive effects of Menkes disease through early Copper Histidinate treatment. The investigat...
What is the current status of trial NCT07398508?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2026-06-01. Estimated completion is 2030-06-30.
What conditions does trial NCT07398508 study?
This clinical trial studies the following conditions: Menkes Disease.
What interventions are being tested in trial NCT07398508?
The interventions under investigation include: Droxidopa Oral Product (DRUG), Placebo Control (OTHER).
Who is sponsoring clinical trial NCT07398508?
This trial is sponsored by Stephen G. Kaler, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT07398508 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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