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Norepinephrine Precursor

Droxidopa for Dysautonomia in Menkes Disease

Phase 1 & 2
Recruiting
Led By Stephen G Kaler, MD
Research Sponsored by Stephen G. Kaler, MD
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age ≥ 18 years of age.
History of at least thrice weekly occurrence of dizziness/feeling lightheaded while standing upright and/or thrice weekly episodes of diarrhea or an urgent need to defecate after food ingestion for more than four weeks immediately preceding enrollment.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up six week periods of active drug versus placebo
Awards & highlights

Study Summary

This trial is studying whether a drug called Northera is safe and effective in young adults with Menkes disease or OHS.

Who is the study for?
Adults over 18 with Menkes disease or Occipital Horn Syndrome who have survived severe complications and experience symptoms like low blood pressure when standing or chronic diarrhea. Participants must have a specific genetic mutation, be able to take oral medication, and commit to study visits. Those with liver or kidney diseases, heart issues, hypertension, or on certain medications cannot join.Check my eligibility
What is being tested?
The trial is testing Northera (Droxidopa) against a placebo to see if it can safely improve symptoms of dysautonomia such as intermittent low blood pressure and chronic diarrhea in adult survivors of Menkes disease and those with Occipital Horn Syndrome.See study design
What are the potential side effects?
Possible side effects of Northera (Droxidopa) include high blood pressure (especially when lying down), headache, dizziness, nausea, fatigue and possibly less common effects like fainting or an increased heart rate.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am 18 years old or older.
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I often feel dizzy or urgently need the bathroom after eating, for over a month.
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My tests show a mutation in the ATP7A gene.
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I can follow the Northera medication schedule as prescribed.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~six week periods of active drug versus placebo
This trial's timeline: 3 weeks for screening, Varies for treatment, and six week periods of active drug versus placebo for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of Treatment-Emergent Adverse Events as assessed by CTCAE v5 tool
Secondary outcome measures
Change in plasma catechol levels after Northera (Droxidopa)
Changes in 6 minute walk test performance after Northera (Droxidopa)
Changes in Time standing duration after Northera (Droxidopa)
+3 more
Other outcome measures
Change in scores on the Orthostatic Hypotension Symptom Assessment questionnaire after Northera (Droxidopa)

Trial Design

2Treatment groups
Active Control
Placebo Group
Group I: Northera™ (Droxidopa) (Treatment A)Active Control1 Intervention
Northera (Droxidopa) (Treatment A) will be provided to adult subjects as a capsule with 100mg, 200mg, or 300mg of Northera (Droxidopa) contained within gelatin color capsules (sky blue and white, size 0) based on findings from the dose titration visit. These capsules are physically indistinguishable from the Treatment B (placebo) capsules. Frequency of administration (by mouth) will be twice daily for six weeks.
Group II: Placebo (Treatment B)Placebo Group1 Intervention
Empty gelatin color capsules (sky blue and white, size 0) filled with cellulose microcrystalline and physically indistinguishable from Treatment A capsules. Frequency of administration (by mouth) will be twice daily for six weeks

Find a Location

Who is running the clinical trial?

Stephen G. Kaler, MDLead Sponsor
Stephen G Kaler, MDPrincipal InvestigatorNationwide Children's Hospital

Media Library

Droxidopa (Norepinephrine Precursor) Clinical Trial Eligibility Overview. Trial Name: NCT04977388 — Phase 1 & 2
Occipital Horn Syndrome Research Study Groups: Northera™ (Droxidopa) (Treatment A), Placebo (Treatment B)
Occipital Horn Syndrome Clinical Trial 2023: Droxidopa Highlights & Side Effects. Trial Name: NCT04977388 — Phase 1 & 2
Droxidopa (Norepinephrine Precursor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04977388 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are volunteers still being sought for this trial?

"The study in question, as detailed on clinicaltrials.gov, is looking for enrolment from the public. The listing was created on 7/12/2021 and was updated most recently on 7/22/2021."

Answered by AI

Could you please provide a list of similar clinical trials involving Droxidopa (Treatment A)?

"Currently, there are 3 ongoing clinical trials evaluating the efficacy of Northera™ (Droxidopa) (Treatment A). 0 of those trials are in Phase 3. The majority of research on Northera™ (Droxidopa) (Treatment A) is conducted in Hawthorne, New york; however, there are active studies at 3 other locations."

Answered by AI

Can I join this clinical trial if I meet the requirements?

"This trial is only for menkes disease patients who are between 18 and 50 years old. Up to 6 people can be enrolled in the study."

Answered by AI

What is the most common reason that patients take Northera™ (Droxidopa) (Treatment A)?

"Northera™ (Droxidopa) (Treatment A), commonly used to treat parkinson's disease, can also mitigate the effects of non-diabetic autonomic neuropathy, dopamine-beta-monooxygenase, and multiple system atrophy."

Answered by AI

Does this research project require participants to be above a certain age?

"According to the information available, people aged 18-50 are eligible for this clinical trial. There are 389 other medical studies currently underway for patients that are under 18 and 1054 for senior citizens over the age of 65."

Answered by AI

Have other researchers looked into this topic before?

"There are three active clinical trials for Northera (Droxidopa) (Treatment A), which began in 2015. The Phase 2 trial, sponsored by Lundbeck LLC, had 30 participants and was completed in 2015. Since then, there have been 18297 studies completed."

Answered by AI

How many individuals are currently enrolled in this clinical trial?

"That is accurate. The clinical trial detailed on clinicaltrials.gov is currently looking for enrollees. This particular research project was first announced on 7/12/2021 with the most recent update being made on 7/22/2021. There is 1 location where 6 people can take part in this study."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome
Stephen G. Kaler, MD 2021. "NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04977388.
~2 spots leftby Jun 2025
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