The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 18 - 60 |
| Updated: | 5/12/2018 |
| Start Date: | November 2015 |
| End Date: | March 2020 |
| Contact: | Jose M Martinez, MA |
| Email: | jose.martinez@nyumc.org |
| Phone: | 212 263 7225 |
The Safety , Tolerability and Efficacy of Dronabinol, a Synthetic Endocannabinoid Receptor Agonist, for the Treatment of Nausea and Vomiting in Patients With Familial Dysautonomia
This is a pilot clinical trial of dronabinol 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.
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 purpose of this pilot study is to assess the safety, tolerability and efficacy of
dronabinol 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 dronabinol in patients with FD using an open-label dose titration phase
followed by 4-weeks of wash-out period. Part 2 will address the efficacy of dronabinol for
the treatment of nausea in patients with FD using a randomized, placebo controlled, double
blind, 12-week cross over design.
The first specific aim of this proposal is to assess the safety and tolerability of
dronabinol in patients with FD. The second specific aim of this proposal is to determine
whether stimulation of endocannabinoid receptors with dronabinol will improve recurrent
nausea in patients with FD. Secondary aims are to determine whether stimulation of
endocannabinoid receptors with dronabinol will increase weight, and decrease anxiety.
dronabinol 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 dronabinol in patients with FD using an open-label dose titration phase
followed by 4-weeks of wash-out period. Part 2 will address the efficacy of dronabinol for
the treatment of nausea in patients with FD using a randomized, placebo controlled, double
blind, 12-week cross over design.
The first specific aim of this proposal is to assess the safety and tolerability of
dronabinol in patients with FD. The second specific aim of this proposal is to determine
whether stimulation of endocannabinoid receptors with dronabinol will improve recurrent
nausea in patients with FD. Secondary aims are to determine whether stimulation of
endocannabinoid receptors with dronabinol will increase weight, and decrease anxiety.
Inclusion Criteria:
1. Male or female patients aged 18-60.
2. Confirmed diagnosis of familial dysautonomia by genetic testing.
3. Symptoms of severe nausea.
4. Able to swallow the capsules.
5. Written informed consent or ascent to participate in the pilot trial and understanding
that they can withdraw consent or accent at anytime without affecting their future
care.
6. Ability to comply with the requirements of the study procedures, including taking
blood pressure measurements at home
Exclusion Criteria:
1. Patients with a history of hypersensitivity to any cannabinoid or sesame oil.
2. Cannabinoid use in the previous 4 weeks (a urinary cannabinoid test will be performed
before study entry).
3. Patients with a history of substance abuse, including alcohol abuse or dependence, or
marijuana.
4. Seizure disorder with at least one epileptic seizure in the last 3 years or abnormal
epileptic discharge in electroencephalography
5. Patients with history of bipolar disorder, severe depression or schizophrenia.
6. Patients that require driving, operating machinery, or engaging in hazardous
activities.
7. Patients taking medications thought, in the investigator's opinion, to be unsafe when
used with dronabinol.
8. Patients with atrial fibrillation, angina or an electrocardiogram documenting a
significant abnormality that may jeopardize the patient's health.
9. Patients with significant pulmonary, liver, renal (creatinine > 2.0 mg/ml), or cardiac
illness that may, in the investigators opinion jeopardize their health by
participating in this pilot trial.
10. Patients with severe cognitive impairment or pervasive developmental disorders, or
patients who are unable to clearly identify and rate their symptoms of nausea.
11. Women who are pregnant or lactating.
12. Patients who have a significant abnormality on clinical examination that may, in the
investigator's opinion, jeopardize their health by participating in this pilot trial.
We found this trial at
1
site
New York, New York 10016
Principal Investigator: Horacio C Kaufmann, MD
Phone: 212-263-7225
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