The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia

NCT02608931 | Withdrawn Phase 2 DMC FDA Drug

• 1 other identifier: 14-01577
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Nausea; Vomiting; Familial Dysautonomia

Keywords

delta-9-tetrahydrocannabinol (THC)

Outcome Measures

Primary Outcomes (2)

• Number of adverse effects

  number of AEs during 4 weeks active drug phase vs. 4 weeks placebo phase

  8 weeks

• Change in nausea scores

  nausea scores during 4 weeks active drug phase vs. 4 weeks placebo phase

  8 weeks

Secondary Outcomes (2)

• Change in weight.

  8 weeks

• Change in anxiety scores

  8 Weeks

Study Arms (2)

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).

Drug: Dronabinol

Placebo Capsules

PLACEBO COMPARATOR

placebo

Other: Placebo

Interventions

Dronabinol DRUG

Also known as: Marinol

Dranabinol Capsules

Placebo OTHER

placebo

Placebo Capsules

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

NYU Medical Center

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Nausea; Vomiting; Dysautonomia, Familial

Interventions

Dronabinol

Condition Hierarchy (Ancestors)

Signs and Symptoms, Digestive; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Primary Dysautonomias; Autonomic Nervous System Diseases; 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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Officials

• Horacio C Kaufmann, MD

  NYU MEDICAL CENTER

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 13, 2015
• First Posted: November 20, 2015
• Study Start: November 1, 2015
• Primary Completion: March 22, 2019
• Study Completion: March 22, 2019
• Last Updated: June 13, 2019

Record last verified: 2019-06

Locations

US (1)