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 |
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.
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.
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
2
sites
New York, New York 10016
Principal Investigator: Horacio Kaufmann, MD
Phone: 212-263-7225
Click here to add this to my saved trials
New York, New York 10016
Phone: 212-263-7225
Click here to add this to my saved trials