Tag Archive for: ion283

Text says 'ION283 Safety Study ASO Therapy for Lafora Disease' over a background image of the Dallas, Texas, skyline

Now Recruiting: ION283 Safety Study

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The application packet for the ION283 safety study is now available!

You can view the application as a PDF or download a Word document.

Please read the email below from Dr. Minassian:


“Dear Lafora disease patients and families,

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.edu as a single email containing all the attachments.  Please note that our email system cannot accept files that are larger than 50 Mb total.  Therefore, make the video short and of low quality.  Just enough to show the patient walking ten steps independently.

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
Principal Investigator
University of Texas Southwestern”

Our deepest gratitude goes to Dr. Minassian, Dr. Joshi, Dr. Verma, Dr. Messahel, and the entire team at UTSW for making this possible. We are also thankful for Ionis’ drug donation and our community’s collective fundraising efforts. We can’t stop now!

Please help us share this information, and good luck to everyone applying.

View PDF Application Download Application as a Word Document
BECAUSE WE HAVE HOPE Lafora Holiday Season Auction 2024

Announcing Holiday Season Auction

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This November, we are hosting a holiday season auction to raise money for the upcoming ION283 safety study.

Bidding will open on November 1st and continue through the 30th. 100% of the profits will go toward the safety study.

Each piece has been donated by the Lafora families or affiliates. Check out our page to see what items are already available for bidding. We invite you to bid on an item to purchase or share this auction with your family and friends via this link: https://givebutter.com/c/laforaauction/auction.

How it will work

Some items will have a unique bidding timeline, but the entire online auction will run throughout November. Generous supporters have donated the items from around the world!

You will have to register to bid here: https://givebutter.com/c/laforaauction/auction.

Please review the details and shipping for each item you bid on. Some items have additional shipping costs, while others only ship to certain countries.

Can you help us?

This is an open call for in-kind donations for our holiday season auction! We request artwork, books, clothing, jewelry, tickets, vacations, and passes to events in your country.

Please email us with the subject “November Auction Donation” to get involved. You can donate items until November 24th.

Items available

Custom made Cruella costume
Tuscan Treasures Vacation
Passport to Paradise Vacation
Mexican Moments Vacation
Handmade gifts from Cel-Luz
Pride and Prejudice
Broad Museum Tickets
Painting by Niki
Painting by Angelina
Text says 'ION283 Safety Study ASO Therapy for Lafora Disease' over a background image of the Dallas, Texas, skyline

ION283 Safety Study on ClincialTrials.Gov

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A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease – ClinicalTrials.gov

We are thrilled to announce that the ION283 Safety Study information was posted to clinicaltrials.gov. Read the full inclusion and exclusion criteria HERE.

Please share the protocol with your clinicians to determine if your loved one is eligible. The essential eligibility criteria is:

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)

At this time, the study is not enrolling patients. However, they have posted the details for families to prepare. If you or your neurologist contact the UTSW team through the clinicaltrials.gov website, you will receive an email instructing you to contact your country’s patient organization for an application packet that your neurologist must complete. However, the patient organizations have not yet received this application packet. Dr. Minassian and his team are waiting for approval of the application packet by the university. They think the packet will be approved sometime in October.

Read the protocol
What if my country does not have a patient organization?
If you do not have a patient organization in your country, please contact Chelsea’s Hope at info@chealseashope.org. We will make sure you receive the application packet as soon as it is available.
How will I know when the enrollment process starts?
The enrollment process will open as soon as the patient organizations send out the application packet. The release date and time for the application packet will be announced in advance so that eligible families and their neurologists know when to expect the application.

How will the enrollment process work?
Neurologists must nominate their patient for the study. In order to do that, they will have to complete the application packet distributed by the patient organizations. The release date for this application packet will be announced in advance, and the packet will be sent to all current patient families and neurologists on our contact registry, and to any new neurologists or family members who contact the organization expressing interest in the study.

When the packet of information is released, neurologists must complete the information requested, and email the packet to the team at UTSW (that contact email will be provided in the packet). The team at UTSW will keep a record of the order in which the packets arrive based on the timestamp of the email, and they will use that list to accept patients on a first-come, first-serve basis.

Patients will not know what number they are on the list. If they receive a spot in the study, they will be contacted when there are enough funds to enroll them.

We will continue to update our patient community as new information becomes available. If you have questions about the application process, contact our Science Director, Dr. Kit Donohue.

IONIS Statement for the Lafora Community September 4, 2024

Ionis Statement for the Lafora Community

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IONIS logoIonis shared a statement with Chelsea’s Hope for the Lafora community. They have agreed to license the ION283 program to Noventia Pharma, and Noventia will assume responsibility for developing it. 

The licensing of the ION283 program will not impact the Safety Study. That will continue as planned, and we will update our community as soon as enrollment is open on clinicaltrials.gov.

Read statement

As a reminder, please sign up for our mailing list and indicate you want to receive “research updates” to receive this news as soon as possible.