Back to resultsThe Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
Primary Purpose
Nausea, Vomiting, Familial Dysautonomia
Status
Withdrawn
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Dronabinol
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Nausea focused on measuring delta-9-tetrahydrocannabinol (THC)
Eligibility Criteria
Inclusion Criteria:
- Male or female patients aged 18-60.
- Confirmed diagnosis of familial dysautonomia by genetic testing.
- Symptoms of severe nausea.
- Able to swallow the capsules.
- 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.
- Ability to comply with the requirements of the study procedures, including taking blood pressure measurements at home
Exclusion Criteria:
- Patients with a history of hypersensitivity to any cannabinoid or sesame oil.
- Cannabinoid use in the previous 4 weeks (a urinary cannabinoid test will be performed before study entry).
- Patients with a history of substance abuse, including alcohol abuse or dependence, or marijuana.
- Seizure disorder with at least one epileptic seizure in the last 3 years or abnormal epileptic discharge in electroencephalography
- Patients with history of bipolar disorder, severe depression or schizophrenia.
- Patients that require driving, operating machinery, or engaging in hazardous activities.
- Patients taking medications thought, in the investigator's opinion, to be unsafe when used with dronabinol.
- Patients with atrial fibrillation, angina or an electrocardiogram documenting a significant abnormality that may jeopardize the patient's health.
- 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.
- Patients with severe cognitive impairment or pervasive developmental disorders, or patients who are unable to clearly identify and rate their symptoms of nausea.
- Women who are pregnant or lactating.
- 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.
Sites / Locations
- NYU Medical Center
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Dranabinol Capsules
Placebo Capsules
Arm Description
The initial dose will be 2.5 mg BID (5 mg/day), and the maximum dose will be 10 mg TID (30 mg/day).
placebo
Outcomes
Primary Outcome Measures
Number of adverse effects
number of AEs during 4 weeks active drug phase vs. 4 weeks placebo phase
Change in nausea scores
nausea scores during 4 weeks active drug phase vs. 4 weeks placebo phase
Secondary Outcome Measures
Change in weight.
change in weight (kgs) during 4 weeks active drug phase vs. 4 weeks placebo phase
Change in anxiety scores
change in anxiety scale scores during 4 weeks active drug phase vs. 4 weeks placebo
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02608931
Brief Title
The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
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 Type
Interventional
2. Study Status
Record Verification Date
June 2019
Overall Recruitment Status
Withdrawn
Why Stopped
Unable to begin study with drug provider
Study Start Date
November 2015 (undefined)
Primary Completion Date
March 22, 2019 (Actual)
Study Completion Date
March 22, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NYU Langone Health
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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 Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Nausea, Vomiting, Familial Dysautonomia
Keywords
delta-9-tetrahydrocannabinol (THC)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Dranabinol Capsules
Arm Type
Experimental
Arm Description
The initial dose will be 2.5 mg BID (5 mg/day), and the maximum dose will be 10 mg TID (30 mg/day).
Arm Title
Placebo Capsules
Arm Type
Placebo Comparator
Arm Description
placebo
Intervention Type
Drug
Intervention Name(s)
Dronabinol
Other Intervention Name(s)
Marinol
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
placebo
Primary Outcome Measure Information:
Title
Number of adverse effects
Description
number of AEs during 4 weeks active drug phase vs. 4 weeks placebo phase
Time Frame
8 weeks
Title
Change in nausea scores
Description
nausea scores during 4 weeks active drug phase vs. 4 weeks placebo phase
Time Frame
8 weeks
Secondary Outcome Measure Information:
Title
Change in weight.
Description
change in weight (kgs) during 4 weeks active drug phase vs. 4 weeks placebo phase
Time Frame
8 weeks
Title
Change in anxiety scores
Description
change in anxiety scale scores during 4 weeks active drug phase vs. 4 weeks placebo
Time Frame
8 Weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or female patients aged 18-60.
Confirmed diagnosis of familial dysautonomia by genetic testing.
Symptoms of severe nausea.
Able to swallow the capsules.
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.
Ability to comply with the requirements of the study procedures, including taking blood pressure measurements at home
Exclusion Criteria:
Patients with a history of hypersensitivity to any cannabinoid or sesame oil.
Cannabinoid use in the previous 4 weeks (a urinary cannabinoid test will be performed before study entry).
Patients with a history of substance abuse, including alcohol abuse or dependence, or marijuana.
Seizure disorder with at least one epileptic seizure in the last 3 years or abnormal epileptic discharge in electroencephalography
Patients with history of bipolar disorder, severe depression or schizophrenia.
Patients that require driving, operating machinery, or engaging in hazardous activities.
Patients taking medications thought, in the investigator's opinion, to be unsafe when used with dronabinol.
Patients with atrial fibrillation, angina or an electrocardiogram documenting a significant abnormality that may jeopardize the patient's health.
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.
Patients with severe cognitive impairment or pervasive developmental disorders, or patients who are unable to clearly identify and rate their symptoms of nausea.
Women who are pregnant or lactating.
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Horacio C Kaufmann, MD
Organizational Affiliation
NYU MEDICAL CENTER
Official's Role
Principal Investigator
Facility Information:
Facility Name
NYU Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
12. IPD Sharing Statement
Learn more about this trial
The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
We'll reach out to this number within 24 hrs