NauseaPhase 2NCT02608931
The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
A Phase 2 study testing Dronabinol in people with Nausea and Vomiting, run by NYU Langone Health at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Nausea, Vomiting, Familial Dysautonomia
- What is being tested?
- Dronabinol
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2015to 22 Mar 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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.
Read the 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.
- Conditions
- Nausea, Vomiting, Familial Dysautonomia
- Being tested
- Dronabinol, Placebo
- Keywords
- delta-9-tetrahydrocannabinol (THC)
- Medical subject headings
- Nausea, Vomiting, Dysautonomia, Familial
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DronabinolDrug
Also called Marinol
PlaceboOther
placebo
| Study group | Role | What they receive |
|---|---|---|
| Dranabinol Capsules | Experimental | The initial dose will be 2.5 mg BID (5 mg/day), and the maximum dose will be 10 mg TID (30 mg/day). Receives: Drug: Dronabinol |
| Placebo Capsules | Placebo comparator | placebo Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse effects
number of AEs during 4 weeks active drug phase vs. 4 weeks placebo phase
Measured: 8 weeks
Change in nausea scores
nausea scores during 4 weeks active drug phase vs. 4 weeks placebo phase
Measured: 8 weeks
Secondary outcomes (2)
Change in weight.
change in weight (kgs) during 4 weeks active drug phase vs. 4 weeks placebo phase
Measured: 8 weeks
Change in anxiety scores
change in anxiety scale scores during 4 weeks active drug phase vs. 4 weeks placebo
Measured: 8 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2015 (estimated)
- Study startNov 2015
- Primary completion22 Mar 2019
- Study completion22 Mar 2019
Why it stopped: Unable to begin study with drug provider
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NauseaStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Horacio C Kaufmann, MD, Principal investigator, NYU MEDICAL CENTER
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02608931
- Sponsor's protocol ID
- 14-01577
- Status last verified
- Jun 2019
- First submitted
- 13 Nov 2015
- Last updated
- 13 Jun 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT02608931
- Acronym / sponsor ID
- 14-01577
- Conditions
- Nausea, Vomiting, Familial Dysautonomia
- Keywords
- delta-9-tetrahydrocannabinol (THC)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Unable to begin study with drug provider
- Enrolment
- 0 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Study start
- Nov 2015
- Primary completion
- 22 Mar 2019 (actual)
- Study completion
- 22 Mar 2019 (actual)
- First posted
- 20 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dranabinol Capsules | Experimental | Drug: DronabinolThe initial dose will be 2.5 mg BID (5 mg/day), and the maximum dose will be 10 mg TID (30 mg/day). |
| Placebo Capsules | Placebo comparator | Other: Placeboplacebo |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DronabinolAlso: Marinol | Drug | Dranabinol Capsules |
| Placeboplacebo | Other | Placebo Capsules |
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
2 secondary outcomes
Change in weight.
Time frame: 8 weeks
Change in anxiety scores
Time frame: 8 Weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jun 2019.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Horacio C Kaufmann, MD principal investigator
NYU MEDICAL CENTER
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Dronabinol in people with Nausea and Vomiting, run by NYU Langone Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 13 Jun 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse effects (time frame: 8 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jun 2019.