Phase 2 N=22 Randomized Quadruple-blind Treatment

Carbidopa for the Treatment of Excessive Blood Pressure Variability

Dysautonomia, Familial · Baroreflex Failure Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT02553265 ↗

Enrolled (actual)

Serious AEs

2.1%

Results posted

Feb 2022

Primary outcome: Primary: Number of Participants Who Reported Adverse Events Related to Study Drug — 0; 0; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Carbidopa Low-Dose (Drug); Placebo (Other); Carbidopa High-Dose (Drug)
Age: Pediatric, Adult · 10+ yrs
Sex: All
Sponsor: NYU Langone Health
Primary completion: May 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants Who Reported Adverse Events Related to Study Drug	0; 0; 0	—
PRIMARY Number of Participants With Significant Changes in Body Mass That Resulted in Discontinuation From the Study.	0; 0; 0	—
PRIMARY Number of Participants With Abnormal Electrocardiographic Interval Patterns	0; 0; 0	—
PRIMARY Average Systolic Blood Pressure Variability (Daytime)	22.92; 18.71; 16.92	—
PRIMARY Highest Systolic Blood Pressure	175; 157; 150	—
PRIMARY Systolic Blood Pressure	126; 126; 126	—
PRIMARY Heart Rate	76; 72; 78	—
PRIMARY Number of Participants Who Displayed Clinical Significant Laboratory Values on CBC or Metabolic Panel	0; 0; 0	—
PRIMARY Number of Participants Who Displayed Clinically Significant Values in Urine Safety Parameters	0; 0; 0	—
SECONDARY Severity of Hypotension During an Active Stand Test	96; 91; 96	—
SECONDARY Number of Participants Who Reported Worsening of OH Symptoms or Dropped Out Because of Worsening OH While on Active Study Drug	0; 0; 0	—
SECONDARY Frequency of Worsening Symptoms Noted in the Patient's Diary	0; 0; 0	—
SECONDARY 24-h Urinary Norepinephrine Excretion	16; 6; 8	—
SECONDARY Coefficient of Systolic BP Variability (Daytime)	19; 16; 15	—
SECONDARY Morning Surge in Systolic BP on Awakening From Sleep (24-h)	44; 19; 20	—

Summary

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.

Eligibility Criteria

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.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02553265). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.