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Trial record 43 of 143 for:    Recruiting, Not yet recruiting, Available Studies | "Vomiting"

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 : Recruiting
First Posted : November 20, 2015
Last Update Posted : May 10, 2018
Sponsor:
Information provided by (Responsible Party):
New York University School of Medicine

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.

Condition or disease Intervention/treatment Phase
Nausea Vomiting Familial Dysautonomia Drug: Dronabinol Other: Placebo Phase 2

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
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
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : March 2020


Arm Intervention/treatment
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).
Drug: Dronabinol
Other Name: Marinol

Placebo Comparator: Placebo Capsules
placebo
Other: Placebo
placebo




Primary Outcome Measures :
  1. Number of adverse effects [ Time Frame: 8 weeks ]
    number of AEs during 4 weeks active drug phase vs. 4 weeks placebo phase

  2. Change in nausea scores [ Time Frame: 8 weeks ]
    nausea scores during 4 weeks active drug phase vs. 4 weeks placebo phase


Secondary Outcome Measures :
  1. Change in weight. [ Time Frame: 8 weeks ]
    change in weight (kgs) during 4 weeks active drug phase vs. 4 weeks placebo phase

  2. Change in anxiety scores [ Time Frame: 8 Weeks ]
    change in anxiety scale scores during 4 weeks active drug phase vs. 4 weeks placebo



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Ages Eligible for Study:   18 Years to 60 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Male or female patients aged 18-60.
  2. Confirmed diagnosis of familial dysautonomia by genetic testing.
  3. Symptoms of severe nausea.
  4. Able to swallow the capsules.
  5. 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.
  6. Ability to comply with the requirements of the study procedures, including taking blood pressure measurements at home

Exclusion Criteria:

  1. Patients with a history of hypersensitivity to any cannabinoid or sesame oil.
  2. Cannabinoid use in the previous 4 weeks (a urinary cannabinoid test will be performed before study entry).
  3. Patients with a history of substance abuse, including alcohol abuse or dependence, or marijuana.
  4. Seizure disorder with at least one epileptic seizure in the last 3 years or abnormal epileptic discharge in electroencephalography
  5. Patients with history of bipolar disorder, severe depression or schizophrenia.
  6. Patients that require driving, operating machinery, or engaging in hazardous activities.
  7. Patients taking medications thought, in the investigator's opinion, to be unsafe when used with dronabinol.
  8. Patients with atrial fibrillation, angina or an electrocardiogram documenting a significant abnormality that may jeopardize the patient's health.
  9. 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.
  10. Patients with severe cognitive impairment or pervasive developmental disorders, or patients who are unable to clearly identify and rate their symptoms of nausea.
  11. Women who are pregnant or lactating.
  12. 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.

Information from the National Library of Medicine

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


Contacts
Contact: Jose M Martinez, MA 212 263 7225 jose.martinez@nyumc.org

Locations
United States, New York
NYU Medical Center Recruiting
New York, New York, United States, 10016
Contact: Jose M Martinez, MA    212-263-7225    jose.martinez@nyumc.org   
Principal Investigator: Horacio C Kaufmann, MD         
Sponsors and Collaborators
New York University School of Medicine
Investigators
Principal Investigator: Horacio C Kaufmann, MD NYU MEDICAL CENTER

Responsible Party: New York University School of Medicine
ClinicalTrials.gov Identifier: NCT02608931     History of Changes
Other Study ID Numbers: 14-01577
First Posted: November 20, 2015    Key Record Dates
Last Update Posted: May 10, 2018
Last Verified: May 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by New York University School of Medicine:
delta-9-tetrahydrocannabinol (THC)

Additional relevant MeSH terms:
Vomiting
Nausea
Primary Dysautonomias
Autonomic Nervous System Diseases
Dysautonomia, Familial
Signs and Symptoms, Digestive
Signs and Symptoms
Nervous System Diseases
Hereditary Sensory and Autonomic Neuropathies
Nervous System Malformations
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Dronabinol
Hallucinogens
Physiological Effects of Drugs
Psychotropic Drugs
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Cannabinoid Receptor Agonists
Cannabinoid Receptor Modulators
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action
Hormones