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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: The Safety , Tolerability and Efficacy of Dronabinol, a Synthetic Endocannabinoid Receptor Agonist, for the Treatment of Nausea and Vomiting in Patients With Familial Dysautonomi

Brief 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.

Detailed Summary:

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.

Sponsor: New York University School of Medicine

Current Primary Outcome:

• Number of adverse effects [ Time Frame: 8 weeks ]
  number of AEs during 4 weeks active drug phase vs. 4 weeks placebo phase
• Change in nausea scores [ Time Frame: 8 weeks ]
  nausea scores during 4 weeks active drug phase vs. 4 weeks placebo phase

Original Primary Outcome: Same as current

Current Secondary Outcome:

• Change in weight. [ Time Frame: 8 weeks ]
  change in weight (kgs) during 4 weeks active drug phase vs. 4 weeks placebo phase
• Change in anxiety scores [ Time Frame: 8 Weeks ]
  change in anxiety scale scores during 4 weeks active drug phase vs. 4 weeks placebo

Original Secondary Outcome: Same as current

Information By: New York University School of Medicine

Dates:
Date Received: November 13, 2015
Date Started: November 2015
Date Completion: November 2017
Last Updated: December 7, 2016
Last Verified: December 2016