Dysautonomia, FamilialPhase 2CarbiFDNCT02553265
Carbidopa for the Treatment of Excessive Blood Pressure Variability
A Phase 2 study testing Carbidopa Low-Dose and Carbidopa High-Dose in people with Dysautonomia, Familial and Baroreflex Failure Syndrome, run by NYU Langone Health at 2 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 10 to 60 years
- Conditions the study is for
- Dysautonomia, Familial, Baroreflex Failure Syndrome
- What is being tested?
- Carbidopa Low-Dose and Carbidopa High-Dose
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 22 participants
- Where
- 2 sitesUnited States
- Study dates
- Sep 2015to 10 May 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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
Show all 9Show fewer
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
NYU Langone Medical Center, Dyautonomia Center, Suite 9Q
New York, New York, 10016
NYU Medical Center
New York, New York, 10016
About this study
Official title: 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.
Read the detailed description
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.
- Being tested
- Carbidopa Low-Dose, Placebo, Carbidopa High-Dose
- Keywords
- Familial dysautonomia (HSAN III), Norepinephrine, Sympathetic nervous system, Autonomic nervous system, Blood pressure variability, Hypertension, Carbidopa/Lodosyn, Afferent baroreflex failure, Clinical trial
- Medical subject headings
- Dysautonomia, Familial, Hypertension
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Carbidopa Low-DoseDrug
Also called Lodosyn ®, DL-α-methyl-α-hydrazino-3, 4-dihydroxyphenyl-propionic acid, HMD, MK-486
300 mg/day
PlaceboOther
Also called Placebo control pill
A placebo containing an inert substance, in capsule form that does not contain an active drug ingredient.
Carbidopa High-DoseDrug
Also called Lodosyn ®, DL-α-methyl-α-hydrazino-3, 4-dihydroxyphenyl-propionic acid, HMD, MK-486
600 mg/day
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Matching placebo Receives: Other: Placebo |
| Low-Dose Carbidopa | Experimental | Not described Receives: Drug: Carbidopa Low-Dose |
| High-Dose Carbidopa | Active comparator | Not described Receives: Drug: Carbidopa High-Dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Who Reported Adverse Events Related to Study Drug
Adverse events defined as: a change in a patient's baseline condition including intercurrent illnesses irrespective of the relationship to carbidopa treatment. This will be monitored primarily with phone calls at weekly intervals. In addition, patients will be asked about adverse events while at the office. Patients will also fill a daily diary with a specific prompts to note any adverse events.
Measured: Up to 90 days
Number of Participants With Significant Changes in Body Mass That Resulted in Discontinuation From the Study.
Body mass measured in kg
Measured: Up to 90 days
Number of Participants With Abnormal Electrocardiographic Interval Patterns
Clinically significant changes in the intervals of characteristic electrocardiographic patterns
Measured: Up to 90 days
Average Systolic Blood Pressure Variability (Daytime)
Patients with FD undergo ambulatory BP monitoring while keeping a detailed log of their activities (sleep/meal-times/medications/posture/symptoms). Variability in blood pressure overtime will be measured by the standard deviation during awake hours
Measured: up to Week 14
Highest Systolic Blood Pressure
Maximum blood pressure captured on 24-h ambulatory monitoring
Measured: Day 1 of treatment period
Systolic Blood Pressure
SBP measured in the seated position
Measured: up to Week 14
Heart Rate
Heart rate in the seated position
Measured: up to Week 14
Number of Participants Who Displayed Clinical Significant Laboratory Values on CBC or Metabolic Panel
Clinically significant laboratory values include complete blood count (CMC) and metabolic panel related to treatment with carbidopa
Measured: Up to 90 days
Number of Participants Who Displayed Clinically Significant Values in Urine Safety Parameters
Clinically significant values on urinalysis, urine safety parameters related to treatment with carbidopa
Measured: Up to 90 days
Secondary outcomes (6)
Severity of Hypotension During an Active Stand Test
Lowest blood pressure captured during 3 minutes of standing
Measured: up to Week 14
Number of Participants Who Reported Worsening of OH Symptoms or Dropped Out Because of Worsening OH While on Active Study Drug
Measured: Up to 90 days
Frequency of Worsening Symptoms Noted in the Patient's Diary
A tailored questionnaire to examine symptoms over the treatment period and the used of as needed medications. Each day will have a designated page. Since nausea/vomiting and hypertension occur together in FD we will use a diary consisting of a simplified version of the Rhodes Index 44 symptoms of nausea/retching, with items addressing vomiting/throwing up omitted, as most participants will have had anti-reflux surgery to prevent vomiting (fundoplication), graded on a 5-point scale (appendix 2). The diary will also include space to write down any adverse events on a daily basis.
Measured: Up to 90 Days
24-h Urinary Norepinephrine Excretion
Norepinephrine concentration determined from a 24-hour urine sample in a bottle shielded from light containing preservative. Patients will be instructed to refrigerate their sample and bring it on the morning of their visit in a cool bag.
Measured: up to Week 14
Coefficient of Systolic BP Variability (Daytime)
The measurement of blood pressure variability based on the standard deviation that also takes into account the underlying level of BP.
Measured: up to Week 14
Morning Surge in Systolic BP on Awakening From Sleep (24-h)
The morning surge will be calculated as the difference between the mean systolic blood pressure during the hour that included the lowest blood pressure during sleep and maximum value detected within 2-h of awakening from sleep
Measured: up to Week 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Sep 2015 (estimated)
- Study startSep 2015
- Primary completion10 May 2019
- Study completion10 May 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Dysautonomia, FamilialStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Horacio C Kaufmann, MD, Principal investigator, NYU School of MedicineLucy J Norcliffe-Kaufmann, PhD, Principal investigator, NYU School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02553265
- Sponsor's protocol ID
- 13-00065
- Other IDs
- FD-R-004772
- Acronym
- CarbiFD
- Status last verified
- Jan 2022
- First submitted
- 14 Sep 2015
- Last updated
- 9 Feb 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Norcliffe-Kaufmann L, Martinez J, Axelrod F, Kaufmann H. Hyperdopaminergic crises in familial dysautonomia: a randomized trial of carbidopa. Neurology. 2013 Apr 23;80(17):1611-7. doi: 10.1212/WNL.0b013e31828f18f0. Epub 2013 Apr 3.
PubMed 23553478Norcliffe-Kaufmann LJ, Axelrod FB, Kaufmann H. Cyclic vomiting associated with excessive dopamine in Riley-day syndrome. J Clin Gastroenterol. 2013 Feb;47(2):136-8. doi: 10.1097/MCG.0b013e3182582cbf.
PubMed 22739220Norcliffe-Kaufmann L, Axelrod FB, Kaufmann H. Developmental abnormalities, blood pressure variability and renal disease in Riley Day syndrome. J Hum Hypertens. 2013 Jan;27(1):51-5. doi: 10.1038/jhh.2011.107. Epub 2011 Dec 1.
PubMed 22129610Kaufmann H, Malamut R, Norcliffe-Kaufmann L, Rosa K, Freeman R. The Orthostatic Hypotension Questionnaire (OHQ): validation of a novel symptom assessment scale. Clin Auton Res. 2012 Apr;22(2):79-90. doi: 10.1007/s10286-011-0146-2. Epub 2011 Nov 2.
PubMed 22045363Norcliffe-Kaufmann L, Axelrod F, Kaufmann H. Afferent baroreflex failure in familial dysautonomia. Neurology. 2010 Nov 23;75(21):1904-11. doi: 10.1212/WNL.0b013e3181feb283.
PubMed 21098405Palma JA, Norcliffe-Kaufmann L, Fuente-Mora C, Percival L, Mendoza-Santiesteban C, Kaufmann H. Current treatments in familial dysautonomia. Expert Opin Pharmacother. 2014 Dec;15(18):2653-71. doi: 10.1517/14656566.2014.970530. Epub 2014 Oct 17.
PubMed 25323828
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Carbidopa in Familial Dysautonomia: Phase-II Study, Investigational New Drug (IND) 117435, Date: 01/07/13
- ClinicalTrials.gov ID
- NCT02553265
- Acronym / sponsor ID
- CarbiFD / 13-00065
- Keywords
- Familial dysautonomia (HSAN III), Norepinephrine, Sympathetic nervous system, Autonomic nervous system, Blood pressure variability, Hypertension, Carbidopa/Lodosyn, Afferent baroreflex failure
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- Sep 2015
- Primary completion
- 10 May 2019 (actual)
- Study completion
- 10 May 2019 (actual)
- First posted
- 17 Sep 2015 (estimated)
- Results
- Posted 9 Feb 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Other: PlaceboMatching placebo |
| Low-Dose Carbidopa | Experimental | Drug: Carbidopa Low-Dose |
| High-Dose Carbidopa | Active comparator | Drug: Carbidopa High-Dose |
Who may qualify
- Ages
- 10 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Male or female patients with familial dysautonomia (FD) age 10 or older
- Unstable blood pressure, defined as:
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Carbidopa Low-DoseAlso: Lodosyn ®, DL-α-methyl-α-hydrazino-3, 4-dihydroxyphenyl-propionic acid, HMD, MK-486300 mg/day | Drug | Low-Dose Carbidopa |
| PlaceboAlso: Placebo control pillA placebo containing an inert substance, in capsule form that does not contain an active drug ingredient. | Other | Placebo |
| Carbidopa High-DoseAlso: Lodosyn ®, DL-α-methyl-α-hydrazino-3, 4-dihydroxyphenyl-propionic acid, HMD, MK-486600 mg/day | Drug | High-Dose Carbidopa |
Primary outcome
Number of Participants Who Reported Adverse Events Related to Study Drug
Time frame: Up to 90 days
Adverse events defined as: a change in a patient's baseline condition including intercurrent illnesses irrespective of the relationship to carbidopa treatment. This will be monitored primarily with phone calls at weekly intervals. In addition, patients will be asked about adverse events while at the office. Patients will also fill a daily diary with a specific prompts to note any adverse events.
Number of Participants With Significant Changes in Body Mass That Resulted in Discontinuation From the Study.
Time frame: Up to 90 days
Body mass measured in kg
Number of Participants With Abnormal Electrocardiographic Interval Patterns
Time frame: Up to 90 days
Clinically significant changes in the intervals of characteristic electrocardiographic patterns
Average Systolic Blood Pressure Variability (Daytime)
Time frame: up to Week 14
Patients with FD undergo ambulatory BP monitoring while keeping a detailed log of their activities (sleep/meal-times/medications/posture/symptoms). Variability in blood pressure overtime will be measured by the standard deviation during awake hours
Highest Systolic Blood Pressure
Time frame: Day 1 of treatment period
Maximum blood pressure captured on 24-h ambulatory monitoring
Systolic Blood Pressure
Time frame: up to Week 14
SBP measured in the seated position
Heart Rate
Time frame: up to Week 14
Heart rate in the seated position
Number of Participants Who Displayed Clinical Significant Laboratory Values on CBC or Metabolic Panel
Time frame: Up to 90 days
Clinically significant laboratory values include complete blood count (CMC) and metabolic panel related to treatment with carbidopa
Number of Participants Who Displayed Clinically Significant Values in Urine Safety Parameters
Time frame: Up to 90 days
Clinically significant values on urinalysis, urine safety parameters related to treatment with carbidopa
6 secondary outcomes
Severity of Hypotension During an Active Stand Test
Time frame: up to Week 14
Number of Participants Who Reported Worsening of OH Symptoms or Dropped Out Because of Worsening OH While on Active Study Drug
Time frame: Up to 90 days
Frequency of Worsening Symptoms Noted in the Patient's Diary
Time frame: Up to 90 Days
24-h Urinary Norepinephrine Excretion
Time frame: up to Week 14
Coefficient of Systolic BP Variability (Daytime)
Time frame: up to Week 14
Morning Surge in Systolic BP on Awakening From Sleep (24-h)
Time frame: up to Week 14
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 23553478Norcliffe-Kaufmann L, Martinez J, Axelrod F, Kaufmann H. Hyperdopaminergic crises in familial dysautonomia: a randomized trial of carbidopa. Neurology. 2013 Apr 23;80(17):1611-7. doi: 10.1212/WNL.0b013e31828f18f0. Epub 2013 Apr 3.
- Background: PubMed 22739220Norcliffe-Kaufmann LJ, Axelrod FB, Kaufmann H. Cyclic vomiting associated with excessive dopamine in Riley-day syndrome. J Clin Gastroenterol. 2013 Feb;47(2):136-8. doi: 10.1097/MCG.0b013e3182582cbf.
- Background: PubMed 22129610Norcliffe-Kaufmann L, Axelrod FB, Kaufmann H. Developmental abnormalities, blood pressure variability and renal disease in Riley Day syndrome. J Hum Hypertens. 2013 Jan;27(1):51-5. doi: 10.1038/jhh.2011.107. Epub 2011 Dec 1.
- Background: PubMed 22045363Kaufmann H, Malamut R, Norcliffe-Kaufmann L, Rosa K, Freeman R. The Orthostatic Hypotension Questionnaire (OHQ): validation of a novel symptom assessment scale. Clin Auton Res. 2012 Apr;22(2):79-90. doi: 10.1007/s10286-011-0146-2. Epub 2011 Nov 2.
- Background: PubMed 21098405Norcliffe-Kaufmann L, Axelrod F, Kaufmann H. Afferent baroreflex failure in familial dysautonomia. Neurology. 2010 Nov 23;75(21):1904-11. doi: 10.1212/WNL.0b013e3181feb283.
- Background: PubMed 25323828Palma JA, Norcliffe-Kaufmann L, Fuente-Mora C, Percival L, Mendoza-Santiesteban C, Kaufmann H. Current treatments in familial dysautonomia. Expert Opin Pharmacother. 2014 Dec;15(18):2653-71. doi: 10.1517/14656566.2014.970530. Epub 2014 Oct 17.
- New York University (NYU) Dysautonomia Center Research SiteLink provided by the sponsor.
- Patient Advocacy GroupLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Horacio C Kaufmann, MD principal investigator
NYU School of Medicine
Lucy J Norcliffe-Kaufmann, PhD principal investigator
NYU School of Medicine
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Carbidopa Low-Dose and Carbidopa High-Dose in people with Dysautonomia, Familial and Baroreflex Failure Syndrome, run by NYU Langone Health at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Who Reported Adverse Events Related to Study Drug (time frame: Up to 90 days). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02553265, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2022; AzTrials last refreshed its copy on 8 Nov 2026.