Double-blind, Placebo-control, Cross-over Trial of Cannabidiol for Essential Tremor

IETF Funded Research

Principal Investigator: Fatta Nahab, M.D., associate professor, University of California, San Diego

The focus of this study is to collect the necessary data needed to determine whether further research is warranted to develop cannabidiol (CBD) as a viable tremor suppressant for essential tremor.

The central hypothesis is that CBD will reduce tremor amplitude while being well tolerated and safe. The hypothesis has been formulated on the basis of preliminary data and through consultation with Dr. Adrian Handforth’s group actively studying cannabinoids in the Harmaline model of ET.

Results obtained from the study will provide the critical knowledge needed to request National Institutes of Health (NIH) funding for a drug development program to pursue the shorter-term development of CBD for essential tremor and the longer-term development of similar yet more targeted novel agents.

There are two specific aims to this study:
Aim 1: Does CBD have tremor suppression properties in ET?
Aim 2: Is CBD safe and well tolerated for short-term use in an ET population?

With respect to expected outcomes, the findings of aims 1 & 2 are expected to provide the necessary pilot data to justify, or not, the scientific need for further human trials of CBD for the treatment of ET. Furthermore, these findings have the potential to open up alternative basic science lines of targeted testing and drug development with the potential to develop a novel molecule specifically designed for ET and increase excitement and funding within biotechnology companies.

Progress Report: Double-blind, Placebo-control, Cross-over Trial of Cannabidiol for Essential Tremor

Principal Investigator: Fatta Nahab, M.D., associate professor, University of California, San Diego

January 2019 Update

We have made significant progress on our work. We have received our Drug Enforcement Agency (DEA)  import license and our pharmaceutical partner, Tilray, obtained their export license. The drug product and matching placebo capsules were manufactured. The product was shipped to us in mid-December and after a complex series of regulatory hurdles, we received delivery.

We have received an enormous interest in the study from prospective participants. We completed pre-screening of 200 subjects and the first 10 eligible to participate are now being scheduled.

 

January 2020 Update

Since January 2019, the following updates have occurred:

Accrual Log:

Total Screened: 12 subjects
Screen Failures: 3 (2 had Parkinson’s disease and 1 had functional tremor). Note that a large number of subjects were excluded at pre-screening due to presence of a resting tremor suggestive of Parkinson’s.
Enrolled into Study: 4 subjects
Completed Study: 2 subjects
Dropouts: 2 subjects
Subjects Screened, Qualified and Awaiting Randomization: 5

Challenges:

Our industry partner has experienced production challenges and delays in Canadian regulatory approvals to permit export of the product to the U.S. This has led to my needing to renew my DEA import permit twice due to expiration of the permit before receipt of Canadian regulatory approval. As of now, we have a new, active import permit and have received Canadian approval with an anticipated receipt of new drug product by February 2020.

Lastly, we continue to experience major recruiting challenges for this trial. These challenges are multi-factorial, including: high number of misdiagnosed individuals (substantial number exhibiting features of Parkinsonism or functional tremor) and reservation to participate on the part of eligible individuals.

We thank you for your continued support of this work.