Trial · NCT07398508
NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease
NCT07398508, Phase 1, Phase 2, Recruiting, Stephen G. Kaler
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease. Trials, record NCT07398508. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07398508
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease
- NCT number
- NCT07398508
- Status
- Recruiting
- Phases
- Phase 1, Phase 2
- Enrollment
- 6 participants, estimated
- Lead sponsor
- Stephen G. Kaler
- Conditions
- Menkes Disease
- Interventions
- Droxidopa Oral Product, Placebo Control
- First posted
- 2026-02-10
- Last updated
- 2026-10-05
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence of SAEs | 10 weeks | This is to measure safety and tolerability of study drug in children and adolescents with Menkes disease. The study will determine the adverse event profile of the formulation by comparing the time-to-event of serious adverse events (SAEs) and adverse events (AEs) between treatment and placebo arms as defined in the protocol. |
| Secondary | Change in level of plasma norepinephrine and dihydroxyphenylglycol after Northera (Droxidopa) | Baseline and post intervention at 4 weeks | Plasma L-DOPS levels: Levels of L-threo-3,4-dihydroxyphenylserine (L-DOPS, droxidopa) may be measured for comparison to the known pharmacokinetic (PK) profile of Northera (Droxidopa) (peak level at approximately 3 hrs). The assay for plasma LDOPS and plasma catecholamines (High Performance Liquid Chromatography with electrochemical detection) is the same. Measurements of L-DOPS may permit correlation between plasma L-DOPS and plasma norepinephrine levels. |
| Secondary | Change in systolic blood pressure | Baseline and post intervention at 4 weeks | Blood pressure (BP) will be measured using a standard blood pressure monitor while subjects are standing, and between supine and head-up tilt table positions after Northera (Droxidopa). Unit: mm Hg. Tilt Table testing involves positioning a patient onto the tilt table with feet resting on footplates. Continuous blood pressure monitors are then attached to the subject. The entire table can be tilted to move from 0˚ to 60˚angle in approximately 45s, allowing determination of changes in pulse and blood pressure based on supine (0˚) and standing (60˚) positions. |
| Secondary | Average number of daily bowel movements | Baseline, 4 weeks | Gastrointestinal symptoms will be monitored using irritable bowel syndrome-diarrhea report. Decreased daily bowel movements counts as an improvement. |
| Secondary | Average time standing | Baseline and post intervention at 4 weeks | This is to measure any improvement in physicality using performance of physical exertion tests. The longer the time standing, the greater the improvement. Unit: minutes. |
| Secondary | Average 6-minute walk distance | Baseline and post intervention at 4 weeks | This is to measure any improvement in physicality using performance of physical exertion tests. The further the distance, the greater the improvement. The primary measurement taken from a 6-minute walk test (6MWT) is the final distance someone manages to walk, which we refer to as the 6MWD (6-minute walk distance), which can be used to assess a patient's functional status. For everyday healthy people, the average distance walked during this test is between 400 and 700 meters. |
| Other | Change in scores on the Orthostatic Hypotension Symptom Assessment questionnaire after Northera (Droxidopa) | Baseline and post intervention at 4 weeks | The Orthostatic Hypotension Questionnaire (OHQ) was developed, with two components: the six-item symptoms assessment scale and a four-item daily activity scale to assess the burden of symptoms. Validation analyses were then performed on the two scales and a composite score of the OHQ. Lower the OHQ scores, less severe the symptoms. |
Source ClinicalTrials.gov
Outcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT07398508
GET https://conductscience.org/api/trials/v1/NCT07398508/registered