The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia

• Status: Withdrawn
• Trial end date: 2019-03-22
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: New York University School of Medicine; NYU Langone Health
• Treatments: Dronabinol

Criteria

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.