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NCT04977388 · ClinicalTrials.gov registry record · Phase 1
NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome
A Phase 1 study of Menkes Disease and Occipital Horn Syndrome, sponsored by Stephen G. Kaler, MD.
- Completed
- Registry status
- Phase 1
- Development phase
- 3
- Enrollment target
- 1
- Study location
NCT04977388: Completed Phase 1 study of Menkes Disease and Occipital Horn Syndrome, sponsored by Stephen G. Kaler, MD.
NCT04977388 is a Phase 1 study of Menkes Disease and Occipital Horn Syndrome that has completed, run by Stephen G. Kaler, MD. The registered enrollment target is 3 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (95% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04977388, a Phase 1 study of Menkes Disease and Occipital Horn Syndrome, has completed, sponsored by Stephen G. Kaler, MD.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 3 participants
- Enrollment target
- 1
- Study location
Study Summary
The purpose of this study is to evaluate whether Northera (Droxidopa) is safe and effective in young adults with Menkes disease who survived the most severe complications of their illness or adults with occipital horn syndrome (OHS), who have trouble with intermittent low blood pressure and other symptoms of dysautonomia. The outcomes and information from this study may help adult survivors of Menkes disease and individuals with OHS lead more normal day-to-day lives.
Primary Outcome
Treatment related adverse events as assessed by CTCAE v 4.0 by study arm
Conditions Studied
Interventions
- OTHER Placebo
- DRUG Droxidopa
Study Locations (1)
New York
- Vagelos College of Physicians and Surgeons, Columbia University - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2021-07-12 |
| Est. Completion | 2024-06-29 |
| Phase | Phase 1 |
What the finished NCT04977388 record still lists
NCT04977388 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 3 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (95% lower).
The record links to 2 conditions, with Menkes Disease appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT04977388 reports a single indexed study location in New York.
Frequently Asked Questions
What is clinical trial NCT04977388 about?
NCT04977388 is a clinical study titled "NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome". The purpose of this study is to evaluate whether Northera (Droxidopa) is safe and effective in young adults with Menkes disease who survived the most severe complications of their illness or adults with occipital horn syndrome (OHS), who have trouble with intermittent low blood pressure and other sy...
What is the current status of trial NCT04977388?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 3 participants. The study started on 2021-07-12. Estimated completion is 2024-06-29.
What conditions does trial NCT04977388 study?
This clinical trial studies the following conditions: Menkes Disease, Occipital Horn Syndrome.
What interventions are being tested in trial NCT04977388?
The interventions under investigation include: Placebo (OTHER), Droxidopa (DRUG).
Who is sponsoring clinical trial NCT04977388?
This trial is sponsored by Stephen G. Kaler, MD, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04977388 being conducted?
This trial has 1 study location across New York. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04977388's enrollment target sits among peer trials
3 1999th of 2000 higher than 1 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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