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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.

Must not have

Any alpha-1 adrenoreceptor agonist, beta-blocker, DOPA decarboxylase inhibitor, midodrine, ephedrine, or any triptan medication as a concomitant medication.

History of hypertension, anti-hypertensive therapy, heart failure (or decreased ejection fraction), cardiac arrhythmia, or bleeding diatheses.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up six week periods of active drug versus placebo

Awards & highlights

Approved for 10 Other Conditions

All Individual Drugs Already Approved

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.

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.

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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not taking any medication for blood pressure, Parkinson's, or migraines.

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I have a history of high blood pressure, heart issues, or bleeding disorders.

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I have liver or kidney disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ six week periods of active drug versus placebo

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Incidence of Treatment-Emergent Adverse Events as assessed by CTCAE v5 tool

Secondary study objectives

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 study objectives

Change in scores on the Orthostatic Hypotension Symptom Assessment questionnaire after Northera (Droxidopa)

Awards & Highlights

Approved for 10 Other Conditions

This treatment demonstrated efficacy for 10 other conditions.

All Individual Drugs Already Approved

Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

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

0 / 7Eligibility criteria met