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Clinical Trial Update for Prion Diseases, August 2024

By Ionis Pharmaceuticals, news, research

August 2024

August 16th, 2024 -We are pleased to share with you the attached Community Statement from Ionis announcing that the pause on recruitment for their PrProfile Prion Disease study has been lifted. In addition, all planned clinical trial locations for PrProfile are now active, including several additional clinical trial sites that were not part of the earlier stage of enrollment – 16 in total.

Please review the attached Community Statement. We remain hopeful with the continued progress toward potential drug developments and clinical trials that may lead to a treatment or cure.


April 2024

April 9th, 2024 -Ionis has released the following community statement on the PrProfile clinical trial recruitment pause:


March 2024

March 26th, 2024 – Ionis has just announced the activation of a site in Gunma, Japan for the PrProfile Study.

You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.


March 25th, 2024 – Ionis has just announced the activation of a site in Tel Aviv, Israel for the PrProfile Study.

You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.


March 19th, 2024 – Ionis has just announced the opening of two enrolment locations in Japan – one in Tokyo and the other in Yamaguchi for the PrProfile Study.

You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.


March 13th, 2024Ionis has just announced the opening of an enrolment site in Montreal, Canada (McGill University Health Centre) for the PrProfile Study.

You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.


February 2024

February 21st, 2024Ionis has just announced the opening of an enrolment site in Boston, USA (Massachusetts General Hospital) for the PrProfile Study.

You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.


February 3rd, 2024Ionis has just announced the opening of an enrolment site in Germany for the PrProfile Study. This is the third site to open outside of the USA.

You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.


January 2024

January 27th, 2024Ionis has just announced the opening of an enrolment site in Barcelona, Spain for the PrProfile Study. This is the second site to open outside of the USA.

You can learn more about this site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

As always, individuals interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.


January 24th, 2024Ionis has announced that they have opened a third enrollment site in Canada (University of Alberta) for the PrProfile Study.

You can learn more about that site here: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717.

Anyone interested in learning more about the trial/site information can contact PrionDisease@clinicaltrialmedia.com and/or call +1- (844) 221-3587.


As of January 20, 2024, the UCLA Neurology Clinic in Los Angeles, California, United States has been added as the next prion disease human clinical trial site.

Please email PrionDisease@clinicaltrialmedia.com or call (844) 221-3587 to obtain more information on the study details and clinical trial site locations.


December 2023

Ionis has provided an update on their prion disease clinical trial –

The status of the Ionis Clinical Trial on clinicaltrials.gov is now “Recruiting“.
One site is listed as active and recruiting at this time and that is in the U.S. at the University Hospitals Cleveland Medical Center in Ohio.

Key points:

  • The clinical trial is for persons in the early stages of active disease.
  • Trial sites will be rolled out in phases (not all sites will be live at the same time).
  • Ionis does not determine patient eligibility to participate in the clinical trial.
  • Refer interested patients to their physician or a clinical trial site to discuss eligibility.
  • It will be the determination of the clinical trial sites if they include patients from other countries.

We encourage you to:

  • Familiarize yourself with the links within the Community Statement so that you can provide support and information to your patients and families.
  • Familiarize yourself with the FAQs included in the Community Statement – we need to provide consistent messaging to our patients and families.
  • Share the community statement with your patients and families.

Remember, this is just a first step in a very long process. Research does take time but each step leads us closer to discovery. Regardless of the outcome, this is a positive step for those impacted by prion disease.


The Ionis Clinical Trial is posted on the clinical trials website.

Here is the link:  A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717

The current status is “not yet recruiting”. Even though no sites are open, this is a normal step in the process. You are able to see some of the study criteria and other information.

We will continue to provide you with updates as things progress.


October 2023

We are please to provide the following community statement that has just been released by Ionis about their upcoming clinical trial.

About Gate Bioscience

By Gate Bioscience, research

Gate Bioscience is creating a new category of medicines called Molecular Gates. Our therapeutics are small molecules that selectively eliminate harmful extracellular proteins at their origin, inside the cell. We are working to develop a small molecule therapeutic to selectively eliminate prion protein (PrP) in the brain. If successful, this work could lead to an orally available PrP-lowering medicine: in effect, a pill to treat or prevent prion disease. Gate Bioscience’s Prion therapeutic is in preclinical development and is not yet ready for clinical trials.

– Science article – Foiling Deadly Prions

– Our website – https://www.gatebio.com/