A Phase 2 interventional study of Dronabinol and Placebo in Nausea, Vomiting and Familial Dysautonomia, sponsored by NYU Langone Health. Withdrawn at 1 site in United States. Open to participants aged 18 Years to 60 Years. Per ClinicalTrials.gov, last updated 2019-06-13.
Sponsored by NYU Langone Health · Phase 2, Interventional, and Treatment
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.
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.
166 studies on the registry are indexed under Autonomic Nervous System Diseases; 36 are open to participants now.
Browse Autonomic Nervous System Diseases studies →NYU Langone Health is the lead sponsor of 1,391 studies on the registry; 254 are open to participants now.
Of its 227 completed or terminated interventional studies of FDA-regulated products, 191 (84%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
The initial dose will be 2.5 mg BID (5 mg/day), and the maximum dose will be 10 mg TID (30 mg/day).
Drug: Dronabinol
placebo
Other: Placebo
Also known as: Marinol
placebo
Number of adverse effects
number of AEs during 4 weeks active drug phase vs. 4 weeks placebo phase
Time frame: 8 weeks
Change in nausea scores
nausea scores during 4 weeks active drug phase vs. 4 weeks placebo phase
Time frame: 8 weeks
Change in weight.
change in weight (kgs) during 4 weeks active drug phase vs. 4 weeks placebo phase
Time frame: 8 weeks
Change in anxiety scores
change in anxiety scale scores during 4 weeks active drug phase vs. 4 weeks placebo
Time frame: 8 Weeks
This study is withdrawn, as verified in Jun 2019. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Autonomic Nervous System Diseases→
NYU Langone Health