The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
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|ClinicalTrials.gov Identifier: NCT02608931|
Recruitment Status : Withdrawn (Unable to begin study with drug provider)
First Posted : November 20, 2015
Last Update Posted : June 13, 2019
|Condition or disease||Intervention/treatment||Phase|
|Nausea Vomiting Familial Dysautonomia||Drug: Dronabinol Other: Placebo||Phase 2|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||0 participants|
|Intervention Model:||Crossover Assignment|
|Masking:||Double (Participant, Investigator)|
|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 Dysautonomia|
|Study Start Date :||November 2015|
|Actual Primary Completion Date :||March 22, 2019|
|Actual Study Completion Date :||March 22, 2019|
Experimental: Dranabinol Capsules
The initial dose will be 2.5 mg BID (5 mg/day), and the maximum dose will be 10 mg TID (30 mg/day).
Other Name: Marinol
Placebo Comparator: Placebo Capsules
- 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
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02608931
|United States, New York|
|NYU Medical Center|
|New York, New York, United States, 10016|
|Principal Investigator:||Horacio C Kaufmann, MD||NYU MEDICAL CENTER|