Tag Archive for: ion283

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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.

ION283 Safety Study Approval ASO Therapy for Lafora Disease

ION283 Safety Study Approved

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We just learned from Dr. Minassian that the FDA has approved the ION283 Safety Study!

We are so grateful for your continuous support, for Dr. Minassian and his team at UTSW, Dr. Messahel, IONIS, and everyone involved for getting us here.

Our patient community has been a driving force in supporting reaching this stage and we can celebrate another step forward today. We hope this will lead to treatments for our children.

Berge A. Minassian, M.D

“30 years in the making, we now have FDA approval to start our ION283 clinical trial.

I would like to thank each of your associations for your support over the years…” – Dr. Berge Minassian

We do not yet have the application site on clinicaltrials.gov or the criteria for inclusion in the Safety Study. Those will be coming in a few weeks. In the meantime, we can continue fundraising and ensuring all patient families learn of this opportunity.

Budget

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.

France Lafora is also fundraising HERE.

Donate today!


Please help us share this information! 

If you did not get an email update from us earlier, please ensure you are signed up for our mailing list. Once we learn the criteria from Dr. Minassian, we will send out the information to everyone who has signed up for “research updates.” You can contact christine@chelseashope.org to confirm your email preferences with the subject “Check my Chelsea’s Hope Email Preferences.”

Questions? Please check out our FAQs section on our webpage. 

We answer commonly asked questions about the safety study, including what it is, how much it costs, who can participate, and more! If you don’t see an answer to your question, please email katherine@chelseashope.org with the subject “Safety Study Question.”