Carbidopa for the Treatment of Excessive Blood Pressure Variability



Status:Recruiting
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:10 - 60
Updated:7/22/2018
Start Date:September 2015
End Date:June 2019
Contact:Jose M Martinez, MA
Email:jose.martinez@nyumc.org
Phone:212 263 7225

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Carbidopa in Familial Dysautonomia: Phase-II Study, Investigational New Drug (IND) 117435, Date: 01/07/13

The overall study objectives are to determine whether carbidopa (Lodosyn®) is safe and well
tolerated and to assess whether it can inhibit catecholamine-induced paroxysmal hypertension
and normalize or reduce the exaggerated blood pressure variability in patients with familial
dysautonomia (FD, also called hereditary sensory and autonomic neuropathy type III or
Riley-Day syndrome). Funding Source- FDA OOPD.

The investigators propose to perform a double-blind randomized trial with a cross over design
to compare high dose (600 mg/day) and low dose (300 mg per day) carbidopa blockade with
placebo. Patients will be randomly assigned to a high-dose/low-dose/placebo sequence,
lowdose/placebo/high-dose sequence or placebo/high-dose/low-dose sequence. Participants will
remain on each treatment period for 28-days.

Aim 1: To evaluate the safety and tolerability of carbidopa in FD patients with particular
emphasis on the orthostatic fall in blood pressure.

Aim 2: As proof of concept, examine the hemodynamic effects of carbidopa and determine its
effects on norepinephrine production, BP variability and paroxysmal hypertension.

Aim 3: In a dose finding study, compare the effects of low (300 mg/day) and high (600 mg/day)
dose carbidopa blockade vs. placebo on BP variability and paroxysmal hypertension.

Inclusion Criteria:

- Male or female patients with familial dysautonomia (FD) age 10 or older

- Unstable blood pressure, defined as:

- Systolic BP standard deviation >15 mmHg

- Or coefficient of variation >15%

- Or documented episodic hypertensive peaks (>140mmHg)

- Confirmed diagnosis of FD (genetic testing)

- Providing written informed consent (or ascent) to participate in the trial

- Ability to comply with the requirements of the study procedures.

Exclusion Criteria:

- Patients taking monoamine oxidase (MAO)-inhibitors

- Patients taking: metoclopramide, domperidone, risperidone or other dopamine blockers

- Patients taking tricyclic antidepressants

- Patients taking neuroleptic drugs (haloperidol and chlorpromazine)

- Patients with a known hypersensitivity to any component of this drug.

- Patients with atrial fibrillation, angina or significant ECG abnormality

- Patients with significant pulmonary, cardiac, liver, renal (creatinine >2.0 mg/ml)

- Patients who have a significant abnormality on clinical examination that may, in the
investigator's opinion might jeopardize their healthy participating in this trial.

- Women who are pregnant or lactating.
We found this trial at
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sites
New York, New York 10016
Phone: 212-263-7225
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New York, New York 10016
Principal Investigator: Horacio Kaufmann, MD
Phone: 212-263-7225
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