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Trial record 5 of 6 for:    "Autonomic Neuropathy" | "Hormones"

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Recruitment Status : Withdrawn (Unable to begin study with drug provider)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
Sponsor:
Information provided by (Responsible Party):
NYU Langone Health

Tracking Information
First Submitted Date  ICMJE November 13, 2015
First Posted Date  ICMJE November 20, 2015
Last Update Posted Date June 13, 2019
Study Start Date  ICMJE November 2015
Actual Primary Completion Date March 22, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: November 18, 2015)
  • 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 Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02608931 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: November 18, 2015)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia
Official Title  ICMJE The Safety , Tolerability and Efficacy of Dronabinol, a Synthetic Endocannabinoid Receptor Agonist, for the Treatment of Nausea and Vomiting in Patients With Familial Dysautonomia
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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE
  • Nausea
  • Vomiting
  • Familial Dysautonomia
Intervention  ICMJE
  • Drug: Dronabinol
    Other Name: Marinol
  • Other: Placebo
    placebo
Study Arms  ICMJE
  • 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).
    Intervention: Drug: Dronabinol
  • Placebo Comparator: Placebo Capsules
    placebo
    Intervention: Other: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: June 11, 2019)
0
Original Estimated Enrollment  ICMJE
 (submitted: November 18, 2015)
25
Actual Study Completion Date  ICMJE March 22, 2019
Actual Primary Completion Date March 22, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02608931
Other Study ID Numbers  ICMJE 14-01577
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party NYU Langone Health
Study Sponsor  ICMJE NYU Langone Health
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Horacio C Kaufmann, MD NYU MEDICAL CENTER
PRS Account NYU Langone Health
Verification Date June 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP