Familial Dysautonomia Clinical Trial
Official title:
Carbidopa for the Treatment of Nausea and Vomiting in Familial Dysautonomia
This is a pilot clinical trial of carbidopa to treat disabling attacks of nausea and
vomiting in patients with familial dysautonomia (FD, also known as Riley Day syndrome or
hereditary sensory and autonomic neuropathy type III). FD is a rare autosomal recessive
disease in which the growth and development of selective nerves is impaired. Patients with
FD suffer recurrent uncontrollable nausea and vomiting crises accompanied by skin flushing,
tachycardia and arterial hypertension. Current treatments of nausea are ineffective or have
intolerable side sides. Our long-term goal is to treat nausea effectively and without side
effects, a therapeutic intervention that would markedly improve the quality of life of
patients with FD.
The investigators have recently found that resting plasma dopamine levels are high in
patients with FD and increase up to 40-fold during nausea and vomiting attacks. This led us
to postulate that stimulation of dopamine receptors in the chemoreceptor trigger zone of the
brainstem is the likely mechanism of vomiting.
Carbidopa is a reversible competitive inhibitor of aromatic L-amino acid decarboxylase (also
known as dopa-decarboxylase) that cannot cross the blood brain barrier. It has been used
successfully for many years to block the extracerebral synthesis of dopamine and avoid
nausea and vomiting in patients with Parkinson's disease taking levodopa. The investigators
reasoned that carbidopa could have a similar antiemetic effect in patients with FD.
The investigators propose to conduct a pilot trial to assess the safety, tolerability and
efficacy of carbidopa for the treatment of nausea in patients with FD. The pilot trial will
recruit 25 patients with FD who complain of severe nausea that affects their quality of
life. The trial will be divided into two consecutive, but independent parts. Part 1, will
address the safety and tolerability of carbidopa in patients with FD using an open-label
dose titration phase followed by 4-weeks of open-label treatment. Part 2, will address the
efficacy of carbidopa for the treatment of nausea in patients with FD using a randomized,
placebo controlled, double blind, 4-week cross over design.
The investigators hope to demonstrate that carbidopa is a safe, well-tolerated drug that
blocks the peripheral formation of dopamine and thus prevents dopamine-induced nausea and
vomiting attacks in patients with FD.
Status | Completed |
Enrollment | 9 |
Est. completion date | October 2012 |
Est. primary completion date | February 2012 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 12 Years and older |
Eligibility |
Inclusion Criteria: - 1. Male of female patients aged 12 and older - 2. Confirmed diagnosis of familial dysautonomia by genetic testing. - 3. Symptoms of severe nausea - 4. Written informed consent or ascent to participate in the pilot trial and understanding that they can withdraw consent at anytime without affecting their future care. - 5. Ability to comply with the requirements of the study procedures, including taking blood pressure measurements at home. Exclusion Criteria: - 1. Patients taking metroclopromide, domperidone, risperidone or other dopamine blockers - 2. Patients taking MAO-inhibitors - 3. Patients taking tricyclic antidepressants - 4. Patients taking neuroleptic drugs (haloperidol and chlorpromazine) - 5. Patients with a known hypersensitivity to any component of this drug. - 6. Patients with atrial fibrillation, angina or an electrocardiogram documenting significant abnormality that may jeopardize the patient's health. - 7. Patients with significant pulmonary, liver, renal (creatinine >2.0 mg/ml) or cardiac illness - 8. Patients who are unable to clearly identify and rate their symptoms of nausea. - 9. Women who are pregnant or lactating - 10. Patients who have a significant abnormality on clinical examination that may, in |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
New York University School of Medicine |
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Number of Adverse Events | 4 months | Yes | |
Secondary | Nausea Scores and Dopamine Levels | 4 months | Yes |
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