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NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome

NCT04977388 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2026-04-13

Study results available
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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.

Conditions

Interventions

DRUG

Droxidopa

Subjects will self-administer capsules of Droxidopa by mouth twice daily for six weeks.

OTHER

Placebo

Subjects will self-administer capsules of placebo by mouth twice daily for six weeks.

Sponsors & Collaborators

  • Stephen G. Kaler, MD

    lead OTHER

Principal Investigators

  • Stephen G Kaler, MD · Vagelos College of Physicians & Surgeons, Columbia University, New York, NY

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-07-12
Primary Completion
2023-10-30
Completion
2024-06-29
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04977388 on ClinicalTrials.gov