ION283 Safety Study
Keep reading for the latest news about the ION283 Safety Study and FAQs. We will continue to update our patient community as new information becomes available.
This is an exciting step forward! It will ensure a brighter future for the entire Lafora community.
NOW RECRUITING: ION283 Safety Study
Please follow the application instructions emailed by Dr. Minassian.
Timeline of Events
The FDA approved the ION283 Safety Study in August!
During the 2024 Lafora Disease Science Symposium, the team at University of Texas Southwestern (UTSW) received the official National Clinical Trial number from the FDA for the ION283 Safety Study: NCT06609889. Families and clinicians can now find official information about the eligibility criteria and the study protocol on clinicaltrials.gov. The essential eligibility criteria are:
1) Patients must be between 10-18 years of age at the time of enrollment (meaning they are at least 10 years old and have not yet turned 19 years old)
2) Patients must have a genetically confirmed diagnosis showing a mutation in either EPM2A or EPM2B/NHLRC1
3) Patients must be able to walk independently (meaning they can walk 10 steps without assistance)
The additional update we have is that Ionis announced on September 4, 2024, that they reached an agreement to license ION283 to Noventia Pharmaceuticals. Read Ionis’s statement to the Lafora community HERE.
The licensing of ION283 will not impact Dr. Minassian’s Safety Study at UT Southwestern. That will continue as planned, and we will update our community as soon as enrollment is open on clinicaltrials.gov.
On October 14, Dr. Souad Messahel kindly joined us for a Q&A. The meeting recording and transcript are available upon request. Requests can be emailed to info@chelseashope.org. View her slides HERE.
Frequently Asked Questions about the ION283 Safety Study
Has the enrollment process started?
Yes! It began on November 17, 2024. Learn more and apply here: https://chelseashope.org/now-recruiting-ion283-safety-study/.
How will the enrollment process work?
We are now ready to receive referrals for consideration for inclusion into the clinical trial: Safety and Efficacy of Intrathecally Administered ION283 in Patients with Lafora Disease
This email relates to the prescreening phase. Below are the requirements for the prescreening. If a patient passes the prescreening, they will be placed on a list of prospective patients to participate in the trial. Each patient invited to participate will still need to meet broader inclusion-exclusion criteria at the time of screening in Dallas. These final inclusion-exclusion criteria are listed in the above website.
Only referrals from neurologists will be accepted, except where it is impossible for the patient to have a neurologist (we are thinking of the patient in Gaza), in which case another doctor or local medic can make the referral.
Attached is a document that contains a prescreening table and the Lafora Disease Performance Scale, both of which should be completed by the referring neurologist. All parts of the document must be completed. All documents and video mentioned in the prescreening table must be provided at the time of referral precisely as described.
Referrals must be sent by the neurologist from his or her email address to LDrecruiting@utsouthwestern.
The list of prospective participants will be generated on a first-come first-served basis, where first-come means that all the above requirements are included in the referral. Any referral that has any component missing will not be accepted and will be returned.
The study will only include 10 participants. Therefore, the first 10 on the first-come list will be invited to Dallas for formal screening toward potentially entering the study. If any patient among the first 10 fails to meet inclusion-exclusion criteria at the time of final screening in Dallas, then this will open a slot for a subsequent patient on the list.
The upper age limit is 18. We understand this to mean any patient before their 19th birthday at the time of final screening in Dallas. In case among the first 10 patients passing prescreening there is a patient whose turn in the list means they will age out of their 19th birthday at the time of the final screen in Dallas, such a patient may be moved up the list as long as this does not affect the age-criterion admissibility of the remaining patients.
Thank you for considering participation in this clinical trial.
Berge A. Minassian, MD
What is the safety study?
This will be a 2-year study to assess the safety and effectiveness of ASO genetic therapy in Lafora disease. Other forms of ASO therapy have been successful in treating other genetic diseases; hence Ionis Pharmaceuticals has developed a new ASO drug called ION283 for treatment of Lafora disease. ION283 has been shown to prevent Lafora Body accumulation in preliminary studies of Lafora disease animal models.
Who is leading it?
Dr. Berge Minassian is the Chief of Child Neurology at UT Southwestern Medical Center. He has been active in neurogenetics throughout his entire career and is a dedicated clinician treating Lafora disease patients. He announced that on April 11th, at the April 2024 Research Roundtable, his team submitted an Investigational New Drug application to the FDA for a safety study of ION283 ASO therapy.
He shared on August 14th that the FDA approved the ION283 Safety Study.
What are the criteria to join?
The inclusion/exclusion criteria are available on clinicaltrials.gov.
Patients will need to be recommended by their neurologists in order to participate in this study.
Where will the study take place?
The safety study will be held at UT Southwestern Medical Center in Dallas, Texas, and participants will have to be present in Dallas for treatment and assessment for about five days every three months. Participants would be responsible for covering travel costs, including travelers’ health insurance and accommodation; however, both Chelsea’s Hope will work to locate resources for families who need financial support to cover travel costs. UT Southwestern has a lot of experience with international patients and has previously partnered with Ronald McDonald House, which provides accommodation and meals for patients and their families.
How long is the study?
The study will last for 2 years. Once the study receives approval, patients will be enrolled on a rolling basis, one per month, as the funds are available.
How many patients will be enrolled?
The plan is that a total of 10 patients will be enrolled in the safety study. The FDA will require a preliminary evaluation after the first two patients are enrolled in the study. If those evaluations show good safety and tolerance of the drug in the first two patents, then the remaining 8 patients can be enrolled.
What is the cost?
The estimate is 1.5 million USD for running the 2-year study; however, this number could change! While Ionis is providing the drug free of charge, this is the budget required to cover the clinical costs associated with the study.
Can the safety study be expanded?
That depends on a number of factors. After the FDA approves the protocol, the investigators at UTSW can submit amendments to expand the study. These amendments could include expanding the age range and the number of patients in the safety study. These amendments will depend on the safety data collected from the initial patients enrolled in the study. It will also depend on how much ION283 drug is available. Enough drug for 10 patients is guaranteed, however, additional drug for more patients would need to be negotiated with the pharmaceutical company.
How can I donate?
Chelsea’s Hope created a fundraising page in anticipation of the Safety Study. Lafora France also has a dedicated fundraising page.
You can make tax-deductible donations in your preferred country. Our organizations will then donate to the UTSW foundation once we have confirmation that the study is active.
You will also be able to donate through a foundation at UT Southwestern to support running the study. Chelsea’s Hope will send out details when the site for receiving funds is launched. If you haven’t already, sign up for emails from Chelsea’s Hope.
If I donate, will my child be guaranteed a spot in the safety study?
No. To keep the process fair, you cannot essentially pay to have your child join the study. Donations to the fund will have no impact on who is selected to participate in the trial!
How can I learn more?
The information about the safety study will be found on clinicaltrials.gov soon, and will have the inclusion/exclusion criteria, protocol, and contact information. Chelsea’s Hope will assist with fundraising for the study, and we will send out details when the site for receiving funds is launched at UT Southwestern. We will continue to communicate any information we learn with you, so please make sure you’ve signed up for our mailing list and indicated your email preferences.
Besides donating, how can I support the study?
Connect with Chelsea’s Hope for updates on the study, and help us spread the word!
What does the licensing of ION283 mean for the Lafora community?
Who is Noventia Pharma?
Noventia Pharma is “a privately owned European specialty pharmaceutical company committed to advancing research to bring hope to those living with serious diseases… In seeking a partner, Ionis was looking for an organization who had the commitment and capability necessary to execute a global development program to evaluate ION283 in Lafora disease.” – Ionis
ASO therapy for Lafora disease
Next Steps
Currently, the Lafora community can fundraise.
The estimated cost of the Safety Study is $1.5 million USD. Please remember, donations do not guarantee a spot for your loved one in the trial. We encourage donations through a patient organization.
Chelsea’s Hope has an online fundraiser where you can donate, and we will transfer the money to UTSW. Read family stories about why they are fundraising and share with your friends!
We have a holiday auction from November 1-30th, too: https://givebutter.com/c/laforaauction/auction.
France Lafora is also fundraising HERE.
Our Collective Fundraising Progress
We are thrilled to share the collective fundraising efforts of our worldwide Lafora organizations: TempoZero, A.I.L.A., Cel-Luz, France Lafora, Chelsea’s Hope, and the Weiss family.
Together, we have raised $710,617 for the safety study.
We are almost to the halfway mark of the estimated budget! We can’t stop now!
Chelsea’s Hope, Post Office Box 348626, Sacramento, CA 95834