Trial · NCT04977388
NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome
NCT04977388, Phase 1, Phase 2, Completed, Stephen G. Kaler, MD
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome. Trials, record NCT04977388. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04977388
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome
- NCT number
- NCT04977388
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 3 participants, actual
- Lead sponsor
- Stephen G. Kaler, MD
- Conditions
- Menkes Disease, Occipital Horn Syndrome
- Interventions
- Droxidopa, Placebo
- First posted
- 2021-07-26
- Last updated
- 2026-04-13
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Treatment Related Adverse Events as Assessed by CTCAE v4.0 | TEAEs in 6 week periods of either active drug (droxidopa) or placebo | Treatment related adverse events as assessed by CTCAE v 4.0 by study arm |
| Secondary | Mean Change in Systolic Blood Pressure in Tilt Position | Change in systolic BP in tilt position during each 6 week treatment arm (droxidopa and placebo) compared to baseline. | Change in systolic BP in tilt position during each treatment arm (droxidopa and placebo) compared to baseline. |
| Secondary | Mean Change in Diastolic Blood Pressure in Tilt Position | The change in diastolic BP in tilt position during each 6 week treatment arm (droxidopa and placebo) compared to baseline. | The change in diastolic BP in tilt position during each treatment arm (droxidopa and placebo) compared to baseline. |
| Secondary | Plasma Catechol Levels | Change in plasma catechols between each 6 week treatment arm (droxidopa and placebo) | Change in plasma catechols between placebo and droxidopa treatment arms |
| Secondary | Change From Baseline in Daily Bowel Movements | Change from baseline in daily bowel movements per day during each 6 week treatment arm (droxidopa and placebo). | Change from baseline in daily bowel movements |
| Secondary | Change From Baseline in Time Standing Duration | Change from baseline in standing time duration during each 6 week treatment arm (droxidopa and placebo). | Change from baseline in Time standing duration |
| Secondary | Change From Baseline in Timed Up and Go (TUG) Test Performance | Change from baseline in TUG test performance during each 6 week treatment arm (droxidopa and placebo) | Change from baseline in Timed Up and Go (TUG) test performance |
| Secondary | Change From Baseline in 6 Minute Walk Test Performance | Change from baseline in the 6MW distance during each 6 week treatment arm (droxidopa and placebo) | Change from baseline in 6 minute walk test performance |
| Secondary | Change From Baseline in Scores on the Orthostatic Hypotension Symptom Assessment (OHSA) Questionnaire | Change from baseline in OHSA score during each 6 week treatment arm (droxidopa and placebo) | Change from baseline in scores on the Orthostatic Hypotension Symptom Assessment questionnaire. The scale rates the severity of OH symptoms from 0 to 10 ; with 10 defined as the worst possible. |
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.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 42016919 | DERIVED |
Source PubMed
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/NCT04977388
GET https://conductscience.org/api/trials/v1/NCT04977388/registered