We are thrilled that Ionis has initiated a clinical trial in Alexander disease! The first site has posted on clinicaltrials.gov and Ionis has released a community statement. Patients and families have waited a long time for this day! While this is an incredibly exciting time, we want to encourage our community members to be cognizant of international travel challenges associated of COVID-19. As mentioned in their community statement, additional sites will open soon and patients interested in participating should reach out to a site close to them when it begins enrolling. Please contact the hospitals directly for more information.

Contact information for the US sites (newly updated!)

Lucile Packard Children’s Hospital Stanford
Palo Alto, California, United States 94304
Contact: Erika Shols 650-272-8986 eschols@standford.edu

Children’s Hospital of Philadelphia
Philadelphia, Pennsylvania, United States 19104
Contact: Geraldine Liu 267-425-2063 liup@chop.edu

Mass General Hospital (Recruiting)
Boston, Massachusetts, United States 02114
Contact: Haley Andonian 617-724-1379 handonian@partners.org

Contact information for the Canadian sites:
Quebec
McGill University Health Centre
Montreal, Quebec, Canada, H3A 0G4
Contact 514-412-4400 ext 23380 bernardlab.neuropediatrics@mcgill.ca

Contact information for the Netherlands:
Amsterdam Universitair Medische Centra - Academisch Medisch Centrum (Recruiting)
Amsterdam, Noord-Holland, Netherlands, 1105 AZ
Contact: Marjo van der Knaap    +31205667508    ms.vanderknaap@amsterdamumc.nl

Drug Development & Clinical Trial Resources from Global Genes:

• Roadmap to Drug Development

• How patients can share their voices through drug development process

• Searching for answers, contacting biopharmaceutical companies effectively

• From Molecules to Medicine, Clinical Research

• NIH clinical trials resource page

See Ionis’s press release and community statement below:

Dear Alexander Disease Patient Community, 

We are writing to share a new update on our progress towards developing a treatment for patients with Alexander disease. Ionis has initiated a clinical trial to evaluate the safety and effectiveness of ION373, an investigational antisense medicine designed to lower the production of Glial Fibrillary Acidic Protein (GFAP), the protein that causes Alexander disease. This update means that the first clinical trial site has opened to begin evaluating patients for study enrollment. This is a global study, and there will be sites opening across several countries over the coming months. When the first site is opened in a country, the locations of all planned sites in that country will be added to the clinicaltrials.gov posting for the trial. Patients who are interested should contact the site closest to them once the site’s information appears on clinicaltrials.gov. 

This multi-center pivotal study of ION373 is a double-blind, placebo-controlled, multiple ascending dose study that will recruit up to 58 Alexander disease patients between 2 and 65 years of age. The study is designed as a dose-escalating study with each dose group evaluated in a sequential manner. This means that the study will enroll slowly as one dose group of patients is enrolled at a time and monitored before the next dose group begins. For more information on this clinical trial, please visit: clinicaltrials.gov [NCT04849741]. Patients who are interested in participating should check this page periodically for updates on study sites near them that are enrolling for this clinical trial. 

The eligibility criteria for this trial were designed to enroll the patients in whom the safety and efficacy of ION373 can be assessed using endpoints that we believe will measure a clinical benefit. We recognize that due to study limitations, not everybody interested will be able to participate. Ultimately, our goal for this program is to enable all patients access to a safe and effective treatment upon regulatory approval, and we are committed to achieving this goal as quickly as possible. 

We are incredibly grateful to the patients, families, clinicians, and researchers who have supported the development of ION373, and we recognize and deeply appreciate the commitment of those who choose to take part in clinical research. It is only with the support of the community that we can advance potential new treatments and realize a better future for those affected by Alexander disease. We look forward to continuing our partnership with the community and providing regular updates on our progress. 

With Hope, 

The Ionis Alexander Disease Team 

To contact the Patient Centric Drug Development team at Ionis, please email: patients@ionisph.com.